Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Recruiting now · Phase 3 · Has a placebo group
Conditions studied: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
In brief
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.
Key facts
- Study ID
- NCT06891443
- Run by
- Laboratoires Thea
- People needed
- 32
- Starts
- 2025-06-04
- Expected to finish
- 2028-10-01
- Last updated by the study team
- 2026-06-25
Who can join
Age: 6 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify 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 subjects with documented evidence of prior better vision eligible.
- 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 may not qualify 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 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).
- 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.
Where it is running
- UCSF Wayne and Gladys Valley Center for Vision — San Francisco, California, United States (enrolling)
- University of Miami - Bascom Palmer Eye Institute — Miami, Florida, United States (enrolling)
- University of Iowa — Iowa City, Iowa, United States (enrolling)
- University of Minnesota Medical School — Minneapolis, Minnesota, United States (enrolling)
- University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics — Philadelphia, Pennsylvania, United States (enrolling)
- Universitair Ziekenhuis Gent (UZ) — Ghent, Belgium (enrolling)
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte — Belo Horizonte, Minas Gerais, Brazil (enrolling)
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) — São Paulo, São Paulo, Brazil (enrolling)
- University of Alberta — Edmonton, Alberta, Canada (enrolling)
- The Hospital for Sick Children - SickKids — Toronto, Ontario, Canada (enrolling)
- Centre de maladies rares CHNO des Quinze Vingt — Paris, France (enrolling)
- Justus-Liebig Universität - Department of Ophthalmology — Giessen, Germany (enrolling)
- Klinikum der Ludwig-Maximilian Universität München — München, Germany (enrolling)
- University of Tuebingen - Inst. for Ophthalmic Research — Tübingen, Germany (enrolling)
- Radboud Universitair Medisch Centrum — Nijmegen, Netherlands (enrolling)
- Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital) — Barcelona, Spain (enrolling)
- Moorfields Eye Hospital NHS Foundation Trust — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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