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New treatment option for Leber Congenital Amaurosis 10
Official title Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
ClinicalTrials.gov ID: NCT06891443
What this study is testing
What is sepofarsen?
sepofarsen is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for leber congenital amaurosis 10.
Also referred to as QR-110.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
- Adults: \>=18 years / Minors: 6 to \<18 years.
- BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP...
- Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
- Detectable ONL in the macular area as determined by the CRC at Screening.
You likely can't join if
- Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
- Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
- Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3...
- Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
- Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Miami, Florida, United States
- Iowa City, Iowa, United States
- Minneapolis, Minnesota, United States
- Philadelphia, Pennsylvania, United States
- Ghent, Belgium
- Belo Horizonte, Minas Gerais, Brazil
- São Paulo, São Paulo, Brazil
- Edmonton, Alberta, Canada
- Toronto, Ontario, Canada
- Paris, France
- Giessen, Germany
- München, Germany
- Tübingen, Germany
- Nijmegen, Netherlands
- Barcelona, Spain
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Miami, Florida, United States; Iowa City, Iowa, United States; Minneapolis, Minnesota, United States; Philadelphia, Pennsylvania, United States; Ghent, Belgium and 11 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.