Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration (NCT01367444) | Clinical Trial Compass
TerminatedPhase 1/2
Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration
Stopped: Study stopped not for safety reasons. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.
United States, France27 participantsStarted 2011-06-08
Plain-language summary
Primary Objective:
To assess the safety and tolerability of ascending doses of SAR422459 in participants with Stargardt's Macular Degeneration (SMD).
Secondary Objective:
To evaluate for possible biological activity of SAR422459.
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Signed and dated written informed consent obtained from the participant and/or the participant's legally acceptable representative.
* Diagnosis of SMD, with at least one pathogenic mutant ABCA4 allele on each chromosome.
* Women of childbearing potential must had a negative pregnancy test at Day -1, and agree to use an effective form of contraception for at least three months, or be surgically sterile or postmenopausal, with the last menstrual period being over two years prior to enrollment.
* Males must agree with their partner to use two forms of contraception for at least three months following SAR422459 administration.
* Participants must agree to not donate blood, organs, tissues or cells for at least three months following SAR422459 administration.
* Participants enrolled in France must be affiliated to or benefit from a social security regimen.
Specific Inclusion Criteria Participant Group A:
* Participants (18 years or older) with advanced SMD.
* Visual acuity less than or equal to (\<=) 20/200 in the worst eye.
* Severe cone-rod dysfunction with no detectable or severely abnormal full-field electroretinogram responses.
Specific Inclusion Criteria Participant Group B:
* Participants (18 years or older) with SMD.
* Visual Acuity \<=20/200 in the worst eye.
* Abnormal full-field electroretinogram responses.
Specific Inclusion Criteria Participant Group C:
* Participants (18 years or older) with SMD.
* Visual acuity \<=20/100 in the worst eye…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)