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

Laboratoires Thea · with Sepul Bio · NCT06891443

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 6+ · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
32
US sites recruiting
4
Study start
June 4, 2025
Record last updated
October 21, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: sepofarsen; Placebo IVT

Conditions: Leber Congenital Amaurosis 10; Blindness; Leber Congenital Amaurosis; Sensation Disorders; Vision Disorder; Neurological Manifestations; Eye Diseases, Hereditary; Eye Diseases; Eye Disorders Congenital; Retinal Disease

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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.

Recruiting US locations

Study contacts (from the official record)

  • Sepul Bio Patient Advocacy Director · +31 617060791 · contact@sepulbio.com
  • Sepul Bio Chief Medical Officer
Study to Evaluate Sepofarsen in Subjects With Leber Congenital… (NCT06891443) | Bored Studies