Sepofarsen for Leber Congenital Amaurosis

(HYPERION Trial)

Recruiting at 2 trial locations

Overseen By Sepul Bio Patient Advocacy Director

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Laboratoires Thea

Must not be taking: Carbonic anhydrase inhibitors

Disqualifiers: Other IRD mutations, Ocular pathology, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of 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.

Are You a Good Fit for This Trial?

This trial is for individuals with Leber Congenital Amaurosis (LCA) Type 10, specifically those with a certain mutation in the CEP290 gene. Participants must have similar vision impairment in both eyes and some detectable retinal structure. They should also have very limited vision, ranging from roughly 20/50 to light perception only.

Inclusion Criteria

My vision is between 20/50 and nearly complete blindness, but I had better vision before.

My vision in both eyes is nearly the same.

My eye exam shows detectable outer nuclear layer in the macula.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Year 1

One eye receives Sepofarsen and the other eye receives placebo. Injections are administered every 6 months.

12 months

2 visits (in-person)

Treatment Year 2

Continuation of treatment with Sepofarsen in both eyes for some subjects, while others continue with placebo in one eye.

12 months

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Sepofarsen

Trial Overview The study tests Sepofarsen's effectiveness and safety against a placebo in treating LCA type 10. It's a double-masked trial, meaning neither participants nor researchers know who receives the real treatment or placebo. Each eye of a participant gets different treatments for comparison.

How Is the Trial Designed?

Group I: Sepofarsen - Treatment Eye - up to Month 12 Active Control 1 Intervention

Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6.

Group II: Mixed - Treatment Eye - up to Month 24 Active Control 1 Intervention

Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18.

Group III: Continued - Treatment Eye - up to Month 24 Active Control 1 Intervention

Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18.

Group IV: Mixed - Fellow Eye - Month 12 to Month 24 Active Control 1 Intervention

Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18.

Group V: Mixed - Fellow Eye - up to Month 12 Placebo Group 1 Intervention

Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18 Note: up to Month 12 these subjects receive placebo in the Fellow Eye, as all subjects do.

Group VI: Continued - Fellow Eye - up to Month 24 Placebo Group 1 Intervention

Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18.

Group VII: Placebo - Fellow Eye - up to Month 12 Placebo Group 1 Intervention

Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6.

Who Is Running the Clinical Trial?

Laboratoires Thea

Lead Sponsor

Trials

64

Recruited

6,900+

Sepul Bio

Industry Sponsor

Trials

7

Recruited

170+