RecruitingPhase 3

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

United States32 participantsStarted 2025-06-04

Plain-language summary

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.

Who can participate

Age range6 Years

SexALL

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Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Change from baseline in Best-Corrected Visual Acuity (BCVA)

Timeframe: 12 Months

Trial details

NCT IDNCT06891443

SponsorLaboratoires Thea

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-11

Contact for this trial

Sepul Bio Patient Advocacy Director

+31 617060791 contact@sepulbio.com

View on ClinicalTrials.gov More Leber Congenital Amaurosis 10 trials