Clinical Trial Results:
A Phase 2a, Open-Label, Single Ascending Dose Study to Evaluate the Pharmacokinetics and Safety of Solriamfetol in Pediatric Participants with Narcolepsy
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Summary
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EudraCT number |
2019-003008-11 |
Trial protocol |
BE FR NL IT |
Global end of trial date |
04 Feb 2022
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Results information
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Results version number |
v1(current) |
This version publication date |
20 Aug 2022
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First version publication date |
20 Aug 2022
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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 |
JZP865-101
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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 |
Jazz Pharmaceuticals, Inc.
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Sponsor organisation address |
3170 Porter Drive, Palo Alto, United States, 94304
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Public contact |
Director, Clinical Trial Disclosure & Transparency, Jazz Pharmaceuticals, Inc., 1 215-832-3750, ClinicalTrialDisclosure@jazzpharma.com
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Scientific contact |
Director, Clinical Trial Disclosure & Transparency, Jazz Pharmaceuticals, Inc., 1 215-832-3750, ClinicalTrialDisclosure@jazzpharma.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-002184-PIP01-17 | ||
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? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
15 Mar 2022
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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 |
04 Feb 2022
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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 |
To evaluate the efficacy in improving wakefulness in pediatric participants with narcolepsy and to characterize the PK of single doses of solriamfetol in pediatric participants with narcolepsy.
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Protection of trial subjects |
The study will be conducted in accordance with applicable local regulations relating to GCP and with the SOPs of the CRO or Jazz Pharmaceuticals, as applicable. These standards respect the following guidelines or laws:
• Guideline for GCP E6 (R2): ICH, November 2016.
• Good Clinical Practice Directive 205/28/EC.
• Clinical Trials Directive (European Medicines Agency [EMA]) Directive 2001/20/EC, as replaced by the EU Clinical Trials Regulation 536/2014 when it comes into application.
Endorsement of the ethical principles embedded in the above guidances and regulations ensures that the rights, safety and well-being of study participants are protected and are consistent with the principles that have their origin in the Declaration of Helsinki, World Medical Association –“Ethical Principles for Medical Research Involving Human Participants”.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
25 Nov 2020
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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 |
Belgium: 3
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Country: Number of subjects enrolled |
France: 6
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Country: Number of subjects enrolled |
Italy: 5
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Worldwide total number of subjects |
14
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EEA total number of subjects |
14
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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) |
7
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Adolescents (12-17 years) |
7
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Adults (18-64 years) |
0
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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 |
A total of 12 pediatric participants who satisfied all eligibility criteria, 6 participants in each age group (Group 1: adolescents 12 to < 18 years old; Group 2: children 6 to < 12 years old), received solriamfetol over three treatment periods. | |||||||||
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Pre-assignment
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Screening details |
The study had a Screening Period for up to 28 days, 3 Treatment Periods during which participants were administered ascending doses of study intervention, and an End of Study Period to collect final safety evaluations. Periods 1, 2, and 3 were separated by a washout of 14 ± 2 days. Dose was escalated only if the previous dose was tolerated. | |||||||||
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Period 1
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Period 1 title |
Period 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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Are arms mutually exclusive |
No
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Arm title
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Group 1: 12 to <18 years old | |||||||||
Arm description |
Participants ages 12 to <18 years old were administered a single oral dose (75 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 1 (12 to < 18 years old) period 1 received a single oral dose of 75 mg solriamfetol.
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Arm title
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Group 2: 6 to <12 years old | |||||||||
Arm description |
Participants ages 6 to <12 years old were administered a single oral dose (37.5 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 2 (6 to < 12 years old) period 1 received a single oral dose of 37.5 mg solriamfetol.
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Period 2
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Period 2 title |
Period 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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Are arms mutually exclusive |
No
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Arm title
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Group 1: 12 to <18 years old | |||||||||
Arm description |
Participants ages 12 to <18 years old were administered a single oral dose (150 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 1 (12 to < 18 years old) period 2 received a single oral dose of 150 mg solriamfetol.
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Arm title
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Group 2: 6 to <12 years old | |||||||||
Arm description |
Participants ages 6 to <12 years old were administered a single oral dose (75 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 2 (6 to < 12 years old) period 2 received a single oral dose of 75 mg solriamfetol.
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Period 3
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Period 3 title |
Period 3
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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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Are arms mutually exclusive |
No
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Arm title
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Group 1: 12 to <18 years old | |||||||||
Arm description |
Participants ages 12 to <18 years old were administered a single oral dose (300 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 1 (12 to < 18 years old) period 3 received a single oral dose of 300 mg solriamfetol.
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Arm title
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Group 2: 6 to <12 years old | |||||||||
Arm description |
Participants ages 6 to <12 years old were administered a single oral dose (150 mg) of solriamfetol. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
solriamfetol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Each participant in Group 2 (6 to < 12 years old) period 3 received a single oral dose of 150 mg solriamfetol.
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Baseline characteristics reporting groups [1]
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Reporting group title |
Group 1: 12 to <18 years old
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Reporting group description |
Participants ages 12 to <18 years old were administered a single oral dose (75 mg) of solriamfetol. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Group 2: 6 to <12 years old
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Reporting group description |
Participants ages 6 to <12 years old were administered a single oral dose (37.5 mg) of solriamfetol. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Notes [1] - The number of subjects reported to be in the baseline period is not equal to the worldwide number of subjects enrolled in the trial. It is expected that these numbers will be the same. Justification: Fourteen potential participants were screened for this study; 2 failed to meet all entry criteria. A total of 12 participants were enrolled and dosed from 4 centers in the EU (France, Belgium, and Italy), and all 12 participants completed the study. |
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End points reporting groups
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Reporting group title |
Group 1: 12 to <18 years old
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Reporting group description |
Participants ages 12 to <18 years old were administered a single oral dose (75 mg) of solriamfetol. | ||
Reporting group title |
Group 2: 6 to <12 years old
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Reporting group description |
Participants ages 6 to <12 years old were administered a single oral dose (37.5 mg) of solriamfetol. | ||
Reporting group title |
Group 1: 12 to <18 years old
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Reporting group description |
Participants ages 12 to <18 years old were administered a single oral dose (150 mg) of solriamfetol. | ||
Reporting group title |
Group 2: 6 to <12 years old
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Reporting group description |
Participants ages 6 to <12 years old were administered a single oral dose (75 mg) of solriamfetol. | ||
Reporting group title |
Group 1: 12 to <18 years old
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Reporting group description |
Participants ages 12 to <18 years old were administered a single oral dose (300 mg) of solriamfetol. | ||
Reporting group title |
Group 2: 6 to <12 years old
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Reporting group description |
Participants ages 6 to <12 years old were administered a single oral dose (150 mg) of solriamfetol. | ||
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End point title |
Change in Pictorial Sleepiness Scale (PSS) Score from Day 1 Predose to Time of Individual Tmax Postdose for Each Period [1] | ||||||||||||||||||||||||||||
End point description |
The PSS is a pictorial scale used to measure instantaneous perceived sleepiness. The assessment consists of 5 cartoon faces depicting increasing degrees of sleepiness. For each assessment, the faces were presented to the participant in a random 2-dimensional array, and participants chose the face that best describes how sleepy or alert they feel at that time. Each response was converted to a numerical ranked score. PSS is presented on a scale of 0 to 5.58, with 0 corresponding to the most alert, and 5.58 corresponding to the most sleepy. Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3 for the last participant in Group 1.
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End point type |
Primary
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End point timeframe |
Day 1 predose to time of individual Tmax postdose.
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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: No statistical analyses for this end point. |
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| Notes [2] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3 . [3] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Clinical Global Impression of Change (CGIc) Score on Day 1 Relative to Day -1 for Each Period [4] | ||||||||||||||||||||||||||||
End point description |
The CGIc is a 5-point Likert-type rating scale widely used to assess efficacy in clinical studies. For each period, investigators will rate their impression of any change in the participant’s overall sleepiness on dosing day compared to overall baseline (Day 1) sleepiness. The questionnaire is rated on a 5-point scale: 1=Much improved, 2=Minimally improved, 3=No change, 4=Minimally worse, 5=Much worse. Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3 for the last participant in Group 1.
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End point type |
Primary
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End point timeframe |
10 hours postdose.
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| Notes [4] - 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: No statistical analyses for this end point. |
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| Notes [5] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3. [6] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Maximum Plasma Concentration (Cmax) [7] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [7] - 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: No statistical analyses for this end point. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Area Under the Plasma Concentration-Time Curve from Time 0 to the Time t of the Last Quantifiable Concentration (AUC0-t) [8] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [8] - 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: No statistical analyses for this end point. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Area Under the Plasma Concentration-Time Curve from Time 0 to Infinity (AUC0-inf) [9] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [9] - 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: No statistical analyses for this end point. |
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| Notes [10] - Due to limited sampling, estimation of AUC 0-inf was limited across the dose levels. [11] - Due to limited sampling, estimation of AUC 0-inf was limited across the dose levels. [12] - Due to limited sampling, estimation of AUC 0-inf was limited across the dose levels. [13] - Due to limited sampling, estimation of AUC 0-inf was limited across the dose levels. [14] - Due to limited sampling, estimation of AUC 0-inf was limited across the dose levels. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Time to Reach Cmax (tmax) [15] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [15] - 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: No statistical analyses for this end point. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Apparent Terminal Elimination Half-Life (t½) [16] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [16] - 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: No statistical analyses for this end point. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Apparent Oral Clearance (CL/F) [17] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [17] - 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: No statistical analyses for this end point. |
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| Notes [18] - Due to limited sampling, estimation of CL/F was limited across the dose levels. [19] - Due to limited sampling, estimation of CL/F was limited across the dose levels. [20] - Due to limited sampling, estimation of CL/F was limited across the dose levels. [21] - Due to limited sampling, estimation of CL/F was limited across the dose levels. [22] - Due to limited sampling, estimation of CL/F was limited across the dose levels. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Apparent Volume of Distribution (Vd/F) [23] | ||||||||||||||||||||||||||||
End point description |
Descriptive statistics were used to summarize pharmacokinetic (PK) parameters by dose and age group, as appropriate.
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End point type |
Primary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [23] - 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: No statistical analyses for this end point. |
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| Notes [24] - Due to limited sampling, estimation of Vz/F was limited across the dose levels. [25] - Due to limited sampling, estimation of Vz/F was limited across the dose levels. [26] - Due to limited sampling, estimation of Vz/F was limited across the dose levels. [27] - Due to limited sampling, estimation of Vz/F was limited across the dose levels. [28] - Due to limited sampling, estimation of Vz/F was limited across the dose levels. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Change in Pictorial Sleepiness Scale (PSS) Score from Day 1 Predose to 1, 2, 3, 4, 6, 8, and 10 Hours Postdose for Each Period [29] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
The PSS is a pictorial scale used to measure instantaneous perceived sleepiness. The assessment consists of 5 cartoon faces depicting increasing degrees of sleepiness. For each assessment, the faces were presented to the participant in a random 2-dimensional array, and participants chose the face that best describes how sleepy or alert they feel at that time. Each response was converted to a numerical ranked score. PSS is presented on a scale of 0 to 5.58, with 0 corresponding to the most alert, and 5.58 corresponding to the most sleepy. Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3 for the last participant in Group 1.
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End point type |
Secondary
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End point timeframe |
Day 1 predose to 1, 2, 3, 4, 6, 8, and 10 hours postdose.
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| Notes [29] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: No statistical analyses for this end point. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Percentage of Participants with a CGIc of “Much Improved” or “Minimally Improved” on Day 1 Relative to Day -1 for Each Period | |||||||||||||||||||||||||||||||||||
End point description |
The CGIc is a 5-point Likert-type rating scale widely used to assess efficacy in clinical studies. For each period, investigators will rate their impression of any change in the participant’s overall sleepiness on dosing day compared to overall baseline (Day 1) sleepiness. The questionnaire is rated on a 5-point scale: 1=Much improved, 2=Minimally improved, 3=No change, 4=Minimally worse, 5=Much worse. Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3 for the last participant in Group 1.
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End point type |
Secondary
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End point timeframe |
Day 1 predose up to 10 hours postdose.
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| Notes [30] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3. [31] - Data for Group 1 are not shown, as efficacy assessments were not implemented until Period 3. |
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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 |
Adverse events were collected from the time written informed consent is obtained until the End of Study Visit.
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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 |
24.0
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Reporting groups
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Reporting group title |
Group 1: 12 to <18 years old
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Reporting group description |
Participants ages 12 to <18 years old were administered 3 single oral doses (75 mg, 150 mg, 300 mg) of solriamfetol (if tolerated) during 3 separate periods, in an ascending manner. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Group 2: 6 to <12 years old
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Reporting group description |
Participants ages 6 to <12 years old were administered 3 single oral doses (75 mg, 150 mg, 300 mg) of solriamfetol (if tolerated) during 3 separate periods, in an ascending manner. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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06 Mar 2020 |
The primary reason for Amendment 1 was to correct/clarify the clinical laboratory tests to be performed and update the list of analytes evaluated at the urine drug screen. Changes also were made for clarity regarding timing of standard meals/snacks. An editorial change was made to reflect the approval status for solriamfetol in the European Union. |
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10 Jan 2021 |
The primary reason for this global amendment was to add a primary efficacy objective and corresponding endpoints, which elevates the study from phase 1 to phase 2a. To reflect the change in study phase, the study title was also updated.
A primary efficacy objective and associated primary and secondary efficacy endpoints were added to enable preliminary efficacy evaluation; added all endpoints to this section for clarity. |
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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. | |||
| One participant had a single dose delayed (out-of-window Visit 3) due to COVID-19 resulting in an extended washout between Periods 2 and 3. This did not negatively impact the safety of the participant, or the integrity of the PK or safety data. | |||
