Clinical Trial Results:
A Phase 2, open-label, multi-centre study to assess the efficacy and safety of intravitreal THR-317 for the treatment of macular telangiectasia Type 1 (MacTel 1)
Summary
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EudraCT number |
2017-004010-26 |
Trial protocol |
FR |
Global end of trial date |
22 Nov 2019
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Results information
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Results version number |
v1(current) |
This version publication date |
04 Dec 2020
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First version publication date |
04 Dec 2020
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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 |
THR-317-003
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03669393 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Oxurion NV
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Sponsor organisation address |
Gaston Geenslaan 1, Leuven, Belgium, 3001
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Public contact |
Global Clinical Development, Oxurion NV, 32 16751310, info@oxurion.com
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Scientific contact |
Global Clinical Development, Oxurion NV, 32 16751310, info@oxurion.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 |
16 Jan 2020
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
22 Nov 2019
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Global end of trial reached? |
Yes
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Global end of trial date |
22 Nov 2019
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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 and safety of THR-317 for the treatment of MacTel 1
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Protection of trial subjects |
All study procedures, including the intravitreal injections, were performed by qualified and trained personnel. Only eligible subjects were enrolled in the study and only subjects who did not meet any withdrawal criteria received repeat injections. All subjects were supervised in the immediate post-injection period with appropriate medical treatment readily available. Subjects were followed for 3 months after the last injection. Adverse events were recorded throughout the study period. At each study visit, a full ophthalmic examination, BCVA assessment and SD-OCT imaging were performed.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
18 Sep 2018
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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 |
France: 3
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Country: Number of subjects enrolled |
Switzerland: 5
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Worldwide total number of subjects |
8
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EEA total number of subjects |
3
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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) |
6
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From 65 to 84 years |
2
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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 |
The study included a Screening during which in- and exclusion criteria were checked | ||||||
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 |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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THR-317 | ||||||
Arm description |
Subjects received 3 intravitreal injections of THR-317 8mg, approximately 1 month apart | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
THR-317 8mg
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Investigational medicinal product code |
THR-317
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Intravitreal use
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Dosage and administration details |
intravitreal THR-317 8mg at Day 0, Month 1 and Month 2
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Baseline characteristics reporting groups
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Reporting group title |
THR-317
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Reporting group description |
Subjects received 3 intravitreal injections of THR-317 8mg, approximately 1 month apart | |||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
THR-317
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Reporting group description |
Subjects received 3 intravitreal injections of THR-317 8mg, approximately 1 month apart |
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End point title |
Change from Baseline in CST, based on SD-OCT [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Month 3
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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: This was an open-label, exploratory study with a single treatment arm and a limited number of subjects. This endpoint was evaluated only descriptively. No statistical hypothesis testing was performed. |
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No statistical analyses for this end point |
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End point title |
Change from Baseline in BCVA | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Month 3
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No statistical analyses for this end point |
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End point title |
Incidence of systemic and ocular (serious) adverse events ([S]AEs), from first study treatment up to the end of the study | ||||||
End point description |
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End point type |
Secondary
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End point timeframe |
From first study treatment up to end of the 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 |
From first administration of study treatment up to end of study
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
22.0
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Reporting groups
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Reporting group title |
THR-317
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||
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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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11 Oct 2018 |
Addition of blood sampling for immunogenicity assessment. |
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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 |