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

Part of paid clinical trials in San Francisco, California.

Sponsor
Laboratoires Thea
Study ID
NCT06891443
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • sepofarsen — DRUG
    RNA antisense oligonucleotide for intravitreal injection
  • Placebo IVT — OTHER
    Placebo with identical appearance to sepofarsen

Study Details

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.

Key Dates

Start date
Jun 4, 2025
Status verified
Mar 2026
Primary completion
Nov 30, 2027
Completion
Oct 31, 2028

Study Design

Enrollment
32 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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.

Primary Outcome Measure

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

Central Contacts

  • Sepul Bio Patient Advocacy Director
    +31 617060791
    [email protected]
  • Sepul Bio Chief Medical Officer

Locations (5)

FacilityCityStateZIPSite coordinators
UCSF Wayne and Gladys Valley Center for VisionSan FranciscoCalifornia94158-
University of Miami - Bascom Palmer Eye InstituteMiamiFlorida33156-
University of IowaIowa CityIowa52242-
University of Minnesota Medical SchoolMinneapolisMinnesota55455-
University of Pennsylvania - Center for Advanced Retinal & Ocular TherapeuticsPhiladelphiaPennsylvania19104-

Find similar trials in San Francisco, CA

By research site
UCSF Wayne and Gladys Valley Center for Vision· San Francisco, CAUniversity of Miami - Bascom Palmer Eye Institute· Miami, FLUniversity of Iowa· Iowa City, IAUniversity of Minnesota Medical School· Minneapolis, MNUniversity of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics· Philadelphia, PA

Related Studies