At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- sepofarsen (drug), Placebo IVT (other)
- How long the study runs
- Study runs about 40 months (dates as stated)
- About the drug or intervention
- sepofarsen — drug: RNA antisense oligonucleotide for intravitreal injection · Placebo IVT — other: Placebo with identical appearance to sepofarsen
- Patient visit burden
- Not specified by the sponsor
In plain English
This study, called HYPERION, is testing a treatment called sepofarsen in people with Leber Congenital Amaurosis (LCA) type 10, an inherited eye condition that causes severe vision loss or blindness from birth or early childhood. The sponsor is Laboratoires Thea. The study includes adults aged 18 and over and children aged 6 to under 18.
Who can take part
- A confirmed diagnosis of LCA type 10, with a specific change (mutation) in the CEP290 gene, confirmed by genetic testing
- Age 18 or over, or a child aged 6 to under 18
- Vision that is equal to or worse than a set level (roughly 20/50 on a Snellen chart) in both eyes; people with light perception only may still join if they have proof they previously had better vision
- Similar levels of vision in both eyes, checked at the start of the study
- A layer of light-sensing cells in the centre of the retina (the macula) that can still be seen on scans
Who may not be able to
- Gene changes in other genes that cause other inherited eye conditions or syndromes
- Any eye problem that would make comparing the two eyes difficult
- Unstable swelling in the centre of the retina (called CME), or starting or changing certain eye-drop or tablet treatments for it in the 3 months before joining; stable CME for 3 months is allowed
- Significant clouding of the lens or cataracts
- Any previous genetic therapy (RNA or DNA treatment) or stem cell therapy for any disease, including sepofarsen
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 6 Years and over
- Who
- All
- Number of participants
- 32
- Started
- 2025-06-04
- Last checked
- 2026-03
Plain English Summary
What is this study?
- • Testing a new treatment for leber congenital amaurosis 10
- • Phase3 - 32 participants
- • 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
Who can take part?
- • Ages 6 Years and over
- • Diagnosed with leber congenital amaurosis 10
Where?
- • London - Moorfields Eye Hospital NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
This is a double-masked, randomized, placebo-controlled, paired-eye study in which one eye of each subject will serve as a control. At the start of the study the two eyes of each subject will be randomized such that one eye receives sepofarsen and the other eye receives placebo for the first year. In the second year, for all subjects, the eye that was randomized to receive sepofarsen will continue to receive sepofarsen. For the eye that was randomized to placebo in the first year, treatment in the second year will be allocated, as follows: 50% of the eyes will continue to receive placebo, and 50% of the eyes will receive sepofarsen. Sepofarsen and placebo will be administered via intravitreal injection every 6 months.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 6 Years and over
- Who can join: All genders
What the study is looking for
- ✓Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the...
- ✓Adults: \>=18 years / Minors: 6 to \<18 years.
- ✓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.
Who cannot take part
- ✗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 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...
See the full criteria
Where Is This Study? (1 UK site)
Moorfields Eye Hospital NHS Foundation Trust
London EC1V 2PD, United Kingdom
Research Portfolio Managers- Daniela Narvaez, Anika Kadchha, Abi Chandrakumar, Xin Liu
moorfields.resadmin@nhs.net0207 566 2036How to Get in Touch
Sepul Bio Patient Advocacy Director
Sponsor contactCONTACT
Sepul Bio Chief Medical Officer
Sponsor contactCONTACT