Clinical Trial Results:
A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease
Summary
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EudraCT number |
2018-003498-82 |
Trial protocol |
GB FR DK DE ES NL IT |
Global end of trial date |
23 Jun 2022
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Results information
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Results version number |
v1(current) |
This version publication date |
05 Jul 2023
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First version publication date |
05 Jul 2023
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Other versions |
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 |
4429-301
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03772665 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Acucela dba Kubota Vision
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Sponsor organisation address |
600 University Street, Seattle, United States, 98101
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Public contact |
Clinical Trial Helpdesk, Acucela , +1 2068058310, ClinicalTrials@acucela.com
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Scientific contact |
Clinical Trial Helpdesk, Acucela , +1 2068058310, ClinicalTrials@acucela.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 |
23 Jun 2022
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
23 Jun 2022
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Global end of trial reached? |
Yes
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Global end of trial date |
23 Jun 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 determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)
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Protection of trial subjects |
The study was conducted in accordance with the Declaration of Helsinki and its most recent update, and the International Council for Harmonisation (ICH) E6 Good Clinical Practice (GCP) guideline. The study was also conducted in accordance with local legal and regulatory requirements of the countries involved, and with standard operating procedures in place.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
07 Nov 2018
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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 |
Netherlands: 5
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Country: Number of subjects enrolled |
Spain: 7
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Country: Number of subjects enrolled |
United Kingdom: 9
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Country: Number of subjects enrolled |
Denmark: 10
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Country: Number of subjects enrolled |
France: 12
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Country: Number of subjects enrolled |
Germany: 13
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Country: Number of subjects enrolled |
Italy: 33
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Country: Number of subjects enrolled |
Brazil: 29
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Country: Number of subjects enrolled |
United States: 53
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Country: Number of subjects enrolled |
Canada: 5
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Country: Number of subjects enrolled |
South Africa: 18
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Worldwide total number of subjects |
194
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EEA total number of subjects |
80
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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) |
7
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Adults (18-64 years) |
186
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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 |
- | |||||||||||||||
Pre-assignment
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Screening details |
Subjects who met all of the inclusion criteria and none of the exclusions criteria at Screening were included in the study | |||||||||||||||
Pre-assignment period milestones
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Number of subjects started |
194 | |||||||||||||||
Number of subjects completed |
194 | |||||||||||||||
Period 1
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Period 1 title |
Baseline (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | |||||||||||||||
Roles blinded |
Subject, Investigator, Monitor, Data analyst, Carer, Assessor | |||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Treated | |||||||||||||||
Arm description |
- | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
Emixustat hydrocholoride
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule, hard
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Routes of administration |
Oral use
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Dosage and administration details |
10 mg QD
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Arm title
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Placebo | |||||||||||||||
Arm description |
- | |||||||||||||||
Arm type |
Placebo | |||||||||||||||
Investigational medicinal product name |
Placebo
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule, hard
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Routes of administration |
Oral use
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Dosage and administration details |
QD
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Baseline characteristics reporting groups
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Reporting group title |
Treated
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Treated
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Reporting group description |
- | ||
Reporting group title |
Placebo
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Reporting group description |
- |
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End point title |
Primary Efficacy Endpoint | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
24M
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Statistical analysis title |
Slope estimate | ||||||||||||
Comparison groups |
Treated v Placebo
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Number of subjects included in analysis |
194
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Analysis specification |
Pre-specified
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Analysis type |
superiority | ||||||||||||
P-value |
= 0.8091 | ||||||||||||
Method |
slope estimate | ||||||||||||
Confidence interval |
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Adverse events information
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Timeframe for reporting adverse events |
24M
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Assessment type |
Systematic | ||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||
Dictionary version |
25.0
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Reporting groups
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Reporting group title |
Safety Analysis Set
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Reporting group description |
- | ||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 1% | |||||||||||||||||||||
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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 |