Leber Congenital Amaurosis clinical trials
The registry lists 32 studies for Leber Congenital Amaurosis across more than 10 countries. 4 are recruiting or about to start. 22 of them are mainly about Leber Congenital Amaurosis (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-30.
Get an email when these change. Save the Leber Congenital Amaurosis studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Who runs them
Spark Therapeutics, Inc. (4) · QLT Inc. (3) · MeiraGTx UK II Ltd (3) · ProQR Therapeutics (2) · Editas Medicine, Inc. (2) · Laboratoires Thea (2) · HuidaGene Therapeutics Co., Ltd. (1) · Assistance Publique - Hôpitaux de Paris (1)
What is being tested here
Drugs under test in Leber Congenital Amaurosis studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Sepofarsen | 1 | 3 | 4 |
| Voretigene neparvovec | 0 | 1 | 3 |
| Retinol acetate | 0 | 0 | 2 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3, phase 3 first
- Nov 2027Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) · Phase 3 · Laboratoires Thea
- Mar 2027Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis · Ocugen
- Oct 2027Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 · Spark Therapeutics, Inc.
Who these studies take
Of the 4 Leber Congenital Amaurosis studies recruiting now, 4 (100%) set no upper age limit and 0 (0%) stop at 75 or younger. 3 (75%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (25%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (75%) | 3 (75%) |
| 18 | 4 (100%) | 4 (100%) |
| 30 | 4 (100%) | 4 (100%) |
| 40 | 4 (100%) | 4 (100%) |
| 50 | 4 (100%) | 4 (100%) |
| 65 | 4 (100%) | 4 (100%) |
| 75 | 4 (100%) | 4 (100%) |
| 85 | 4 (100%) | 4 (100%) |
Across all 32 studies, recruiting or not: 25% stop at 75 or younger and 22% at 65; 91% take children; 0% are for women only and 0% for men only; 9% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 15, 2026.
Started recruiting1
Studies
Recruiting first, then most recently updated. Showing 32 of 32. search them.
- Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)RecruitingNCT07681778Phase 1/21 country
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)RecruitingNCT06891443Phase 310 countries
- Inherited Retinal Degenerative Disease RegistryRecruitingNCT024359401 country
- Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65CompletedNCT027148162 countries
- Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65CompletedNCT01496040Phase 1/21 country
- Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)Active, not recruitingNCT03602820
- Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2DActive, not recruitingNCT03920007Phase 1/21 country
- Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital AmaurosisActive, not recruitingNCT05203939Phase 1/21 country
- Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 MutationsCompletedNCT00481546Phase 11 country
- Psychotherapy Group for Parents of Children With LCAActive, not recruitingNCT07026565Not applicable1 country
- A Patient Registry Study for Patients Treated With Voretigene Neparvovec in USCompletedNCT035973991 country
- Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2Active, not recruitingNCT01208389Phase 1/21 country
- Safety and Efficacy Study in Subjects With Leber Congenital AmaurosisActive, not recruitingNCT00999609Phase 31 country
- Extension Study to Study PQ-110-001 (NCT03140969)TerminatedNCT03913130Phase 1/22 countries
- Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)Active, not recruitingNCT06088992Early Phase 11 country
- Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)Status unknownNCT05906953Phase 1/22 countries
- Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)CompletedNCT029468792 countries
- Single Ascending Dose Study in Participants With LCA10Status unknownNCT03872479Phase 1/21 country
- Natural History Study of CEP290-Related Retinal DegenerationCompletedNCT033960424 countries
- An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.Status unknownNCT04855045Phase 2/37 countries
- A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)Active, not recruitingNCT03913143Phase 2/39 countries
- Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)CompletedNCT02781480Phase 1/22 countries
- Safety Study in Subjects With Leber Congenital AmaurosisCompletedNCT00516477Phase 11 country
- Treatment of RP and LCA by Primary RPE TransplantationStatus unknownNCT03566147Early Phase 11 country
- Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 MutationsCompletedNCT00821340Phase 11 country
- Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital AmaurosisCompletedNCT00749957Phase 1/21 country
- Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.CompletedNCT029702661 country
- Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRATCompletedNCT025754307 countries
- Safety Study of RPE65 Gene Therapy to Treat Leber Congenital AmaurosisCompletedNCT00643747Phase 1/21 country
- Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)CompletedNCT01521793Phase 15 countries
- Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) MutationsCompletedNCT01014052Phase 15 countries