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

NCT06891443

RecruitingPhase 3

Sponsor: Laboratoires Thea

Conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder

Interventions: sepofarsen, Placebo IVT

Countries: United States, Belgium, Brazil, Canada, France, Germany, Netherlands, Spain

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.

Eligibility overview

Sex: ALL

Age: 6 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

1. Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
2. Adults: \>=18 years / Minors: 6 to \<18 years.
3. 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.
4. Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
5. Detectable ONL in the macular area as determined by the CRC at Screening.

Exclusion Criteria:

1. Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
2. Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
3. 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).
4. Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
5. Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.
Locations (17)
  • 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