Clinical Trial Results:
A Phase 3, Open-label, Non-controlled Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of TAK-771 in Japanese Subjects with Primary Immunodeficiency Diseases (PID)
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Summary
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EudraCT number |
2022-003622-45 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
28 Aug 2023
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Results information
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Results version number |
v1(current) |
This version publication date |
06 Mar 2024
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First version publication date |
06 Mar 2024
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
TAK-771-3004
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05150340 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
jRCT: jRCT2031210457 | ||
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Sponsors
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Sponsor organisation name |
Takeda
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Sponsor organisation address |
5 Hayden Avenue , Lexington , United States, MA 02421
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Public contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Scientific contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
28 Aug 2023
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
28 Aug 2023
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The purpose of the trial included Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
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Protection of trial subjects |
All study participants were required to read and sign an Informed Consent Form.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
24 Jan 2022
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Japan: 16
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Worldwide total number of subjects |
16
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
4
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Adolescents (12-17 years) |
1
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Adults (18-64 years) |
11
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Participants with a confirmed diagnosis of primary immunodeficiency diseases (PID) took part in the study at 12 investigative sites in Japan from 24 January 2022 to 28 August 2023. | ||||||||||
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Pre-assignment
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Screening details |
Participants with PID, who had been receiving a consistent dose of intravenous immunoglobulin (IVIG), conventional subcutaneous immunoglobulin (cSCIG) or TAK-664 (immune globulin subcutaneous [human], 20% solution [20%SCIG]) for at least 3 months prior to screening were enrolled in the study to receive TAK-771. | ||||||||||
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Period 1
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Period 1 title |
Epoch 1
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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Epoch 1: TAK-771 Ramp up Period | ||||||||||
Arm description |
TAK-771 included IGI 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI was increased from 1/3 of full dose to full dose in 3 weeks for participants who received TAK-771 once every 3 weeks, or from 1/4 of full dose to full dose in 6 weeks for participants who received TAK-771 once every 4 weeks. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
TAK-771 (10% IGI with rHuPH20)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
80 U/g IgG (rHuPH20 drug product: 160 U/mL)
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Period 2
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Period 2 title |
Epoch 2
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Is this the baseline period? |
No | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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Epoch 2: TAK-771 Full Dose Treatment Period | ||||||||||
Arm description |
TAK-771 included Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 3, or 4 weeks for up to Week 27 for participants with 4-Week dosing interval or Week 25 for participants with 3-Week dosing interval. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
TAK-771 (10% IGI with rHuPH20)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
80 U/g IgG (rHuPH20 drug product: 160 U/mL)
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| Notes [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: The participants who completed Epoch 1 were included in Epoch 2. |
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Baseline characteristics reporting groups
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Reporting group title |
Epoch 1
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Epoch 1: TAK-771 Ramp up Period
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Reporting group description |
TAK-771 included IGI 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI was increased from 1/3 of full dose to full dose in 3 weeks for participants who received TAK-771 once every 3 weeks, or from 1/4 of full dose to full dose in 6 weeks for participants who received TAK-771 once every 4 weeks. | ||
Reporting group title |
Epoch 2: TAK-771 Full Dose Treatment Period
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Reporting group description |
TAK-771 included Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 3, or 4 weeks for up to Week 27 for participants with 4-Week dosing interval or Week 25 for participants with 3-Week dosing interval. | ||
Subject analysis set title |
Epoch 1 and 2
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Pharmacokinetic Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic Analysis Set included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are the number of participants with data available for analysis at given timepoint.
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End point title |
Epoch 2: Serum Trough Levels of Total IgG Antibodies after Administration of TAK-771 [1] | ||||||||||||||||||||||||||||||||||||||||
End point description |
The data was reported at Week 7, 11, 15, 19, 23, 27, and 31 for 4-Week interval and at Week 4, 7, 10, 13, 16, 19, 22, 25 and 28 for 3-Week interval. Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 1 included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. ‘n’ signifies number of participants analyzed at specific time point and '9999' signifies no geometric mean or geometric coefficient of variation were calculated due to 0 participants in that particular arm at specific time point.
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End point type |
Primary
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End point timeframe |
Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistical analysis was collected for this endpoint. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||
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End point title |
Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. The number of participants analyzed are the number of participants available for analysis. ‘n’ signifies number of subjects analyzed at specific time point
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End point type |
Secondary
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End point timeframe |
Pre-infusion Day at the last dose of TAK-771(Week 27 for participants with 4-Week dosing interval or Week 25 for participants with 3-Week dosing interval) and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. The number of participants analyzed is the number of participant available for analysis. ‘n’ signifies number of subjects analyzed at specific time point. ‘9999’ signifies median and full range was not determined for a single participant. '99999' signifies median and full range was not estimable for two participants.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. The number of participants analyzed is the number of participant available for analysis. 'n' signifies number of subjects analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number participants analyzed are the number of participants available for analysis. ‘n’ signifies number of subjects analyzed at specific time point. 999 indicates that the geometric mean and geometric coefficient of variation was not determined for single participant. 9999 indicates that the geometric mean and geometric coefficient of variation was not determined for two participants.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | ||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. The number of participants analyzed are the number of participants available for analysis. ‘n’ signifies number of subjects analyzed at specific time point. 999 indicates that the geometric mean and geometric coefficient of variation was not estimable for single participant.
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End point type |
Secondary
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End point timeframe |
Pre-infusion at last dose (Week 27 for participants with 4-Week dosing interval (DI) or Week 25 for participants with 3-Week DI) and post infusion at multiple time points up to Week 31 for participants with 4-Week DI and up to Week 28 with 3-Week DI
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number analyzed are the number of participants with data available for analysis at the given time point. ‘n’ signifies number of subjects analyzed at specific time point. 999 indicates that no geometric mean and geometric coefficient of variation were calculated due to 0 subjects in that particular arm at specific time point. 9999 indicates that the geometric coefficient of variation could not determined for single participant.
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End point type |
Secondary
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End point timeframe |
4-Week dosing interval (Week 7, Week 11, Week 15, Week 19, Week 23, Week 27, and Week 31); 3-Week dosing interval (Week 4, week 7, Week 10, Week 16, Week 19, Week 22, Week 25, and Week 28)
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Epoch 1 and 2: Trough Levels of Anti-Clostridium Tetani Toxoid Antibody After Administration of TAK-771 | ||||||||||||
End point description |
Pharmacokinetic Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic Analysis Set included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are the number of participants with data available for analysis at given timepoint. 'n' signifies number of subjects analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval)
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Epoch 1 and 2: Trough Levels of Anti-HBV Antibody After Administration of TAK-771 | ||||||||||||
End point description |
Pharmacokinetic Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic Analysis Set included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are the number of participants with data available for analysis at given timepoint. ‘n’ signifies number of subjects analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval)
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Epoch 1 and 2: Trough Levels of Anti-HIB Antibody After Administration of TAK-771 | ||||||||||||
End point description |
Pharmacokinetic Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic Analysis Set included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are the number of participants with data available for analysis at given timepoint. ‘n’ signifies number of subjects analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval).
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | ||||||||||||
End point description |
TEAEs are defined as Adverse events (AEs) with onset after date-time of first dose of Investigational product (IP), or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
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End point type |
Secondary
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End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | ||||||||||||||||||
End point description |
TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
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End point type |
Secondary
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End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||||||||
|
|||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||
|
|||||||||||||||||||
End point title |
Percentage of Participants With Serious and Non-serious TEAEs | ||||||||||||||||||
End point description |
TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Serious TEAE=any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) at any dose: results in death, was life-threatening, requires inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI=investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable AESI. Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||||||||
|
|||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||
|
|||||||||||||
End point title |
Percentage of Participants With Severe TEAEs | ||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants with severe TEAEs was 0.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||
End point title |
Percentage of Participants With Local and Systemic TEAEs | ||||||||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||||||||
|
|||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||
|
|||||||||||||
End point title |
Percentage of Participants With TEAEs Leading to Premature Discontinuation From Study | ||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants with TEAEs leading to discontinuation from the study were 0.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Percentage of Participants With Infusion-associated TEAEs | ||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Percentage of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs | ||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants with clinically significant changes in clinical laboratory parameters recorded as TEAEs were 0
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Percentage of Participants With Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs | ||||||||||||
End point description |
Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants with clinically significant changes in vital signs and body weight recorded as TEAEs was 0.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||
End point title |
Epoch 2: Percentage of Participants Who Develop Anti-rHuPH20 Binding Antibody Titers of Greater Than or Equal to 1:160 | ||||||||
End point description |
Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants who develop Anti-rHuPH20 Binding Antibody titers of greater than or equal to 1:160 was 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28)
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Epoch 2: Percentage of Participants Who Develop Neutralizing Antibodies to rHuPH20 | ||||||||
End point description |
Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of participants who develop neutralizing antibodies to rHuPH20 was 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28)
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||||||
End point title |
Percentage of Participants Who Experienced Tolerability Events Related to the Infusion of TAK-771 | ||||||||||||
End point description |
Tolerability events is defined as a case that the infusion rate is reduced, or that the infusion is interrupted or stopped, due to a TEAE related to TAK-771 infusion. Safety Analysis Set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2. 999 indicates that the percentage of who experienced tolerability events related to the infusion of TAK-771 was 0.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||
End point title |
Epoch 1: Number of Weeks to Reach Final Dose Interval (3 Weeks or 4 Weeks) | ||||||||
End point description |
The number of weeks to reach final dose interval is defined as treatment duration of Epoch 1. Safety Analysis Set included all enrolled participants who received investigational drug at least once.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
Up to Week 4 for Participants with 4-Week Dosing Interval or Up to Week 3 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Epoch 2: Percentage of Participants Who Achieve a Treatment Interval of 3 or 4 Weeks | ||||||||
End point description |
Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Epoch 2: Percentage of Participants Who Maintain a Treatment Interval of 3 or 4 Weeks | ||||||||
End point description |
Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Annual Rate of Validated Acute Serious Bacterial Infections (ASBIs) | ||||||||
End point description |
The annual rate of validated ASBIs is calculated as the mean number of ASBIs per participant per year. The data was collected using Poisson estimate. Point-estimate and 99% confidence bound (i.e., the upper bound of two-sided 98% confidence interval) were calculated using a Poisson model with subject-year in study as the offset variable. Full Analysis Set included all enrolled participants who received investigational drug at least once. 999 indicates that the annual rate of validated ASBIs was 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Annual Rate of All Infections Per Participant | ||||||||
End point description |
The annual rate of all infections is calculated as the mean number of infections per participant per year. Full Analysis Set included all enrolled participants who received investigational drug at least once.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Healthcare Resource Utilization: Days Not Able To Attend School/Work or To Perform Normal Daily Activities Due to Illness/Infection | ||||||||
End point description |
Full Analysis Set included all enrolled participants who received investigational drug at least once. 99999 indicates that the median and minimum (full range) were 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Healthcare Resource Utilization: Days on Antibiotics | ||||||||
End point description |
Full Analysis Set included all enrolled participants who received investigational drug at least once. 99999 indicates that the median and minimum (full range) were 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Healthcare Resource Utilization: Number of Hospitalizations Due to Illness/Infection | ||||||||
End point description |
Full Analysis Set included all enrolled participants who received investigational drug at least once.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Healthcare Resource Utilization: Length of Stay in Days of Hospitalizations Due to Illness/Infection | ||||||||
End point description |
Full Analysis Set included all enrolled participants who received investigational drug at least once. 99999 indicates that the median and the minimum (full range) were 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Healthcare Resource Utilization: Number of Acute (Urgent or Unscheduled) Physician Visits Due to Illness/Infection | ||||||||
End point description |
Full Analysis Set included all enrolled participants who received investigational drug at least once. 99999 indicates that the minimum (full range) was 0.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Infusion Parameters in Epoch 2: Number of Infusion Sites Per Infusion | ||||||||
End point description |
Total number of infusion sites injected in Epoch 2 / Total number of infusions administered in Epoch 2. Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Infusion Parameters in Epoch 2: Number of Infusion Sites Per Month | ||||||||
End point description |
Total number of infusion sites injected in Epoch 2 / (duration of Epoch 2 / 30.4375), where duration of Epoch 2 is calculated as the end date of the Epoch 2 – the start date of the Epoch 2 + 1. Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Infusion Parameters in Epoch 2: Duration of Individual Infusions | ||||||||
End point description |
End date and time of infusion in Epoch 2 – Start date and time of infusion in Epoch 2, for each infusion per participant. Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Infusion Parameters in Epoch 2: Maximum Infusion Rate Per Site | ||||||||
End point description |
Maximum Infusion Rate results from CRF / number of infusion sites/body.. Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Infusion Parameters in Epoch 2: Infusion Volume Per Site | ||||||||
End point description |
Infusion Volume per Site is scheduled Dose results from CRF / number of infusion sites/body. Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2. Number analyzed are the number of participants with data available for analysis at given timepoint.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||||||||||||||
End point title |
Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | ||||||||||||||||||||
End point description |
PEDS-QL is generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures following domains: general health/activities, feelings/emotional, social functioning, school functioning. In this study, 2-7 years (parent as observer), 8-13 years (participant as observer) for PEDS-QL health questionnaire was analyzed. Higher scores indicate better quality of life (QOL) for all domains of the PEDS-QL. This modular instrument uses 5-point scale: 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Four dimensions (physical, emotional, social, & school functioning) are scored. Full Analysis Set: all enrolled participants who received investigational drug at least once. ‘n’ signifies number of participants analyzed at specific time point and ‘9999’ signifies SD was not estimable for single participant. CFB signifies change from baseline.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
4-Week Dosing Interval (Week 1, Week 19, and Week 31); 3-Week Dosing Interval (Week 1, Week 16, and Week 28)
|
||||||||||||||||||||
|
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||
End point title |
QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | ||||||||||||||||||||||||||||||||
End point description |
SF-36 is a generic quality-of-life instrument used to assess Health-Related Quality of Life (HR QoL) of participants (pts). In study, ≥14 years (yrs) (pt as observer) for SF-36 health questionnaire was analyzed. Generic instruments are used in general populations to assess a wide range of domains applicable to variety of health states, conditions, and diseases. SF-36 consists of 36 items that are aggregated into 8 multi-item scales (physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health), with scores ranging from 0-100. Higher scores=better HR QoL. Epoch 2 Full Analysis Set: all pts who received investigational drug at least once in Epoch 2. Number analyzed: number of pts with data available for analysis at given timepoint. PCS: Physical component summary, MCS: Mental component summary, RCS: Role/social component summary. Change from baseline (CFB). Week (wk). Interval (int). (-) = decline
|
||||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||||
End point timeframe |
4-Week Dosing Interval (Week 1, Week 19, and Week 31); 3-Week Dosing Interval (Week 1, Week 16, and Week 28)
|
||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||
End point title |
QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire | ||||||||||||||||||||||||||||||||||||||||
End point description |
EQ-5D-3L health questionnaire is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. In this study, 2-11 years (parent as observer),12 years and older (participant as observer) for EQ-5D-3L health questionnaire will be analyzed. The EQ-5D-3L total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. Full Analysis Set included all enrolled participants who received investigational drug at least once. 9999 signifies SD was not estimable for single participant. Week (wk), Interval (int), Change from Baseline (CFB), Years (yrs), Index (In), Score (sc). 999 signifies the mean and/or SD was 0. Negative indicates worsening health outcome.
|
||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||||||||||||
End point timeframe |
4-Week dosing interval (Week 1, Week 19, and Week 31); 3-Week dosing interval (Week 1, Week 16, and Week 28)
|
||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Treatment Preference | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Treatment preference questionnaire (q) is a self-administered q developed to assess participants’ (pt) preference (p) towards the administration (adm) of new subcutaneous immunoglobulin (SCIG) therapy. There are 4-items on the q, which investigate a pt's p on the clinic(cl)/hospital(hosp)/home(h) setting of receiving (rec) the IG therapy, the pt's rating on the frequency (freq) and method of adm, and the pt's p to continue rec the IGSC treatment (trt). Full Analysis Set included all enrolled pts who received investigational drug at least once. ‘n’= number of pts analyzed at specific time point, Before (bf), Where (wr), Prefer (pf), Like very much (LVM), Needlesticks (ns), Total (tl), Month (m), Potential (ptl), Dislike very much (DVM), Schedule (skd), Convenience (conv), Amount (amt), Complexity (Cplx), Without (w/o), Medical supervision (ms), Dislike (DL) Immune Globulin Infusion 10%(10% IGI), Recombinant Human Hyaluronidase (rHuPH20), Experience (exp), Different (d), Would (w), With
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Treatment Satisfaction Questionnaire for Medication (TSQM) is a global satisfaction scale used to assess the overall level of participant’s (pt) satisfaction or dissatisfaction with their medications (med). In this study, 2-12 years (yrs) (parent as observer), >13 yrs (pt as observer) for TSQM will be analyzed. TSQM-9 is a 9-item, validated, self-administered instrument used to assess pt's satisfaction with med. The 3 domains assessed: effectiveness (E), convenience (C), and global satisfaction (GS). The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score=greater satisfaction (sat) in that domain. Full Analysis Set: all enrolled pts who received investigational drug at least once. Number (no) analyzed is the no of pts with data available for analysis at given timepoint. ‘n’= no of pts analyzed at specific time point. '999'= mean or SD were 0 and ‘9999’= SD was not estimable for 1 pt. Change from baseline (CFB), Week (wk). (-)=lesser sat
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
4-Week dosing interval (Week 1, Week 19, and Week 31); 3-Week dosing interval (Week 1, Week 16, and Week 28)
|
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|
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From the time the informed (e)Consent document is signed until the EOS/Early termination visit (Up to 21 days after the last dose for the 3-week dosing intervals and 28 days after the last dose for 4-week dosing intervals)
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Adverse event reporting additional description |
All AEs/SAEs which had been reported until EOS/Early termination visit must be followed to closure (the subject’s health has returned to his/her baseline status or all variables have returned to baseline) or until 30 days after EOS/Early termination visit, whichever comes first.
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Assessment type |
Systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
26
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Reporting groups
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Reporting group title |
Epoch 1: TAK-771 Ramp up Period
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Reporting group description |
TAK-771 included IGI 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI was increased from 1/3 of full dose to full dose in 3 weeks for participants who received TAK-771 once every 3 weeks, or from 1/4 of full dose to full dose in 6 weeks for participants who received TAK-771 once every 4 weeks. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Epoch 2: TAK-771 Full Dose Treatment Period
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Reporting group description |
TAK-771 included IGI 10% and rHuPH20. Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 3, or 4 weeks for up to Week 24. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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19 May 2022 |
Amendment 1 included the following: - Descriptions of ‘Device used in clinical trial’ were added. - Infusion rate of 10%IGI was added. - Inclusion criteria 4 and relative description were modified. - Exclusion criteria 9 was modified. - Description in endpoints and analysis for Safety were modified. - HRQoL index for Treatment satisfaction (Life Quality Index) was removed. - Additional items to be summarized descriptively were included. - Analysis for treatment preference was corrected. - Screening visits column for subjects switching from IVIG/cSCIG treatment shown in schedule of activities and clinical laboratory tests were modified. The description in the body was also modified accordingly. - An instruction for dose modification based on weight changes was added. - The timing of specific antibody testing described in the body text was corrected, and it was added that the testing at PK troughs is not required for those who are not in the PK assessment. - Information regarding analysis of antibody titer was added. - Information regarding QoL/treatment satisfaction data capturing was added. The description of SAE reporting was modified. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
