Clinical Trial Results:
PROSPECTIVE, OPEN-LABEL, NON-CONTROLLED, MULTICENTER, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OCTAGAM 10% IN PRIMARY IMMUNE THROMBOCYTOPENIA
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Summary
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EudraCT number |
2012-000796-16 |
Trial protocol |
DE CZ BG PL |
Global end of trial date |
28 Mar 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
20 Jul 2016
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First version publication date |
20 Jul 2016
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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 |
GAMr-30
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Octapharma AG
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Sponsor organisation address |
Seidenstraße 2, Lachen, Switzerland, CH-8853
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Public contact |
Clinical Research Department, Octapharma Pharmazeutika Produktionsgesellschaft m.b. H., clinical.department@octapharma.com
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Scientific contact |
Clinical Research Department, Octapharma Pharmazeutika Produktionsgesellschaft m.b. H., clinical.department@octapharma.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 |
25 Oct 2013
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
28 Mar 2013
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Global end of trial reached? |
Yes
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Global end of trial date |
28 Mar 2013
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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 primary efficacy measure was the platelet count and the increase in platelets to – and the maintenance of – specific thresholds. Number and percentage of patients with response, complete response, no response and loss of response as well as time to response and duration of response are presented.
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Protection of trial subjects |
This trial was conducted in accordance to the principles of GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki.
Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and safety factors associated with the investigational medicinal product. Safety was assessed by the evaluation of AEs, monitoring of vital signs (blood pressure, heart rate, temperature and respiratory rate), physical examinations (to detect relevant somatic or neurological diseases and with attention to signs and symptomes consistent with a thromboembolic event), laboratory parameters and haematology parameters.
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Background therapy |
NA | ||
Evidence for comparator |
NA | ||
Actual start date of recruitment |
30 Aug 2012
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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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 |
Poland: 8
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Country: Number of subjects enrolled |
Bulgaria: 8
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Country: Number of subjects enrolled |
Czech Republic: 9
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Country: Number of subjects enrolled |
Germany: 1
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Country: Number of subjects enrolled |
Romania: 7
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Worldwide total number of subjects |
33
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EEA total number of subjects |
33
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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) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
32
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
33 patients were enrolled - 13 centres recruited patients for the analysis in Poland, Germany, Czech Republik, Bulgaria and Romania. | ||||||||||||
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Pre-assignment
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Screening details |
For the study design chosen, randomisation was not applicable. Patients with confirmed diagnosis of chronic primary ITP (threshold PC less than 100x10^9/L) of at least 12 months, older than 18 years of age, complying to all inclusion criteria and no exclusion criterion were enrolled into this study after having given their written IC. | ||||||||||||
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Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||
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Arms
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Arm title
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OCTAGAM 10% | ||||||||||||
Arm description |
investigation of efficacy and safety of Intravenous immunoglobulin (IVIG) in patients suffering from primary immune thrombocytopenia (ITP) A daily dose of 1 g/kg was administered for 2 consecutive days (Day 1 and Day 2), for a total of 2 g/kg. The initial infusion rate of 0.01 mL/kg/min (60 mg/kg/h) was chosen for safety reasons. The infusion rate was to be increased gradually to a maximum of 0.12 mL/kg/min (720 mg/kg/h) only if tolerated by the patient. The patients received IVIG using an infusion pump for precise infusion rates. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
Octagam 10%
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Investigational medicinal product code |
GAMr-30
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Other name |
Octagam 10%
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Daily dose of 1 g/kg given for two consecutive days, for a total of 2 g/kg.
The initial infusion rate of 0.01 mL/kg/min (60 mg/kg/h) has been chosen for safety reasons. The infusion rate was increased gradually to a maximum of 0.12 mL/kg/min (720 mg/kg/h), only if tolerated by the patient. The patients received IVIG using an infusion pump for precise infusion rates.
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Treated (Safety Set)
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Subject analysis set type |
Safety analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
set of all patients exposed to treatment
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Subject analysis set title |
Full Analysis Set (FAS)
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
consists of all patients of the safety set who satisfied all major eligibility criteria and for whom at least one post-baseline measurement of platelet concentration data is available.
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Subject analysis set title |
Per-Protocol Set (PP)
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Subject analysis set type |
Per protocol | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The PP set consists of all patients of the FA set excluding those who showed major protocol violations which may have an impact on the evaluation of the primary endpoint. This is the set of patients who participated in the study as intended and are available for the primary efficacy evaluation.
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End points reporting groups
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Reporting group title |
OCTAGAM 10%
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Reporting group description |
investigation of efficacy and safety of Intravenous immunoglobulin (IVIG) in patients suffering from primary immune thrombocytopenia (ITP) A daily dose of 1 g/kg was administered for 2 consecutive days (Day 1 and Day 2), for a total of 2 g/kg. The initial infusion rate of 0.01 mL/kg/min (60 mg/kg/h) was chosen for safety reasons. The infusion rate was to be increased gradually to a maximum of 0.12 mL/kg/min (720 mg/kg/h) only if tolerated by the patient. The patients received IVIG using an infusion pump for precise infusion rates. | ||
Subject analysis set title |
Treated (Safety Set)
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
set of all patients exposed to treatment
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Subject analysis set title |
Full Analysis Set (FAS)
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
consists of all patients of the safety set who satisfied all major eligibility criteria and for whom at least one post-baseline measurement of platelet concentration data is available.
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Subject analysis set title |
Per-Protocol Set (PP)
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
The PP set consists of all patients of the FA set excluding those who showed major protocol violations which may have an impact on the evaluation of the primary endpoint. This is the set of patients who participated in the study as intended and are available for the primary efficacy evaluation.
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End point title |
Clinical response [1] | ||||||||||||||||||||||||
End point description |
Efficacy Endpoint:
The primary efficacy measure was the platelet count and the increase in platelets to – and the maintenance of – specific thresholds. Number and percentage of patients with response, complete response, no response and loss of response. The exact definitions of response, complete response and no response were taken from the applicable CHMP Guideline.
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End point type |
Primary
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End point timeframe |
The entire study duration for an individual patient was approximately 3–4 weeks. All patients received 1 g/kg/day Octagam 10% by intravenous infusion for 2 consecutive days.
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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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Time to response [2] | ||||||||||||
End point description |
the time to response and duration of response are presented descriptively to facilitate the comparison of the study results to data from the literature.
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End point type |
Primary
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End point timeframe |
The entire study duration for an individual patient was approximately 3–4 weeks. All patients received 1 g/kg/day Octagam 10% by intravenous infusion for 2 consecutive days.
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| Notes [2] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Duration of response [3] | ||||||||||||||||||||||
End point description |
Duration of response was measured from the achievement of complete response or response to loss of complete response or response.
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End point type |
Primary
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End point timeframe |
The entire study duration for an individual patient was approximately 3–4 weeks. All patients received 1 g/kg/day Octagam 10% by intravenous infusion for 2 consecutive days.
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| Notes [3] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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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 |
24 hours SAE reporting adverse events
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Adverse event reporting additional description |
The condition of the patient was monitored throughout the study. At each visit, whether scheduled or unscheduled, AEs were elicited using a standard non-leading question.
All AEs/SAEs that occured after singing the informed consent but before the first administration of the IMP, were considered “baseline” or “non-treatment-emergent”.
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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 |
14.0
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Reporting groups
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Reporting group title |
all patients exposed to treatment (safety set)
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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? No | |||
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 | |||
