Recruiting
Phase 3

Sepofarsen

Sponsor:

Laboratoires Thea

Code:

NCT06891443

Conditions

Leber Congenital Amaurosis 10

Blindness

Leber Congenital Amaurosis

Sensation Disorders

Vision Disorder

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

sepofarsen

Placebo IVT

Study Details

Brief 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.

Conditions

Leber Congenital Amaurosis 10

Blindness

Leber Congenital Amaurosis

Sensation Disorders

Vision Disorder

Study ID

NCT06891443

Start date

Jun 4, 2025

Status verified date

Mar, 2026

Completion date

Oct, 2028

Anticipated

Primary completion date

Nov, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

32 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Sepofarsen - Treatment Eye - up to Month 12

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

placebo comparator: Placebo - Fellow Eye - up to Month 12

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

active comparator: Continued - Treatment Eye - up to Month 24

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

placebo comparator: Continued - Fellow Eye - up to Month 24

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

active comparator: Mixed - Treatment Eye - up to Month 24

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.

active comparator: Mixed - Fellow Eye - Month 12 to Month 24

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.

placebo comparator: Mixed - Fellow Eye - up to Month 12

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.

Interventions

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

Placebo IVT

Placebo with identical appearance to sepofarsen

Primary outcome measure

  • Change from baseline in Best-Corrected Visual Acuity (BCVA) [ Time Frame: 12 Months ]

Central Contacts and Locations

Central contacts

Sepul Bio Patient Advocacy Director

+31 617060791contact@sepulbio.com

Locations

UCSF Wayne and Gladys Valley Center for Vision

Recruiting

San Francisco, California, United States, 94158

University of Miami - Bascom Palmer Eye Institute

Recruiting

Miami, Florida, United States, 33156

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

University of Minnesota Medical School

Recruiting

Minneapolis, Minnesota, United States, 55455

University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Alberta

Recruiting

Edmonton, Alberta, Canada, T6G 2C8

The Hospital for Sick Children - SickKids

Recruiting

Toronto, Ontario, Canada, M5G 2L3

More Information

Sponsor

Laboratoires Thea

Last update posted

Jun 25, 2026

Last verified

Mar, 2026

Keywords

  • LCA10
  • p.Cys998X
  • Antisense oligonucleotides
  • RNA therapy
  • QR-110
  • sepofarsen
  • CEP290
  • Leber's Congenital Amaurosis
  • c.2991+1655A&gt;G

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Laboratoires Thea on 2026-06-25.