Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Leber Congenital Amaurosis (LCA)”

24 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 24 results

Not applicableNot Yet RecruitingNCT06024057
What this trial is testing

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

Who this might be right for
To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine 3
Testing effectiveness (Phase 2)Study completedNCT00749957
What this trial is testing

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Who this might be right for
Leber Congenital Amaurosis
Beacon Therapeutics 12
Not applicableStudy completedNCT03396042
What this trial is testing

Natural History Study of CEP290-Related Retinal Degeneration

Who this might be right for
BlindnessLeber Congenital Amaurosis 10Vision Disorders+5 more
Editas Medicine, Inc. 26
Not applicableStudy completedNCT02946879
What this trial is testing

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Who this might be right for
Leber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, Hereditary+1 more
MeiraGTx UK II Ltd 15
Large-scale testing (Phase 3)Looking for participantsNCT06891443
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
Laboratoires Thea 32
Testing effectiveness (Phase 2)Study completedNCT03140969
What this trial is testing

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Who this might be right for
Leber's Congenital Amaurosis
Laboratoires Thea 11
Early research (Phase 1)Study completedNCT00516477
What this trial is testing

Safety Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Leber Congenital Amaurosis
Spark Therapeutics, Inc. 12
Testing effectiveness (Phase 2)Active Not RecruitingNCT01208389
What this trial is testing

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Who this might be right for
Leber Congenital Amaurosis
Spark Therapeutics, Inc. 12
Testing effectiveness (Phase 2)Study completedNCT02781480
What this trial is testing

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

Who this might be right for
Leber Congenital Amaurosis
MeiraGTx UK II Ltd 15
Early research (Phase 1)Active Not RecruitingNCT00481546
What this trial is testing

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

Who this might be right for
Amaurosis of LeberRetinal Diseases
University of Pennsylvania 15
Very early researchUnknownNCT03566147
What this trial is testing

Treatment of RP and LCA by Primary RPE Transplantation

Who this might be right for
Leber Congenital Amaurosis, Retinitis Pigmentosa
Eyecure Therapeutics Inc. 30
Not applicableStudy completedNCT02575430
What this trial is testing

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

Who this might be right for
Leber Congenital Amaurosis (LCA)Retinitis Pigmentosa (RP)
QLT Inc. 59
Testing effectiveness (Phase 2)Looking for participantsNCT05906953
What this trial is testing

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Who this might be right for
Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd. 20
Early research (Phase 1)Study completedNCT00821340
What this trial is testing

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Who this might be right for
Leber Congenital Amaurosis
Hadassah Medical Organization 3
Early research (Phase 1)Study completedNCT01521793
What this trial is testing

Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

Who this might be right for
LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
QLT Inc. 27
Not applicableStudy completedNCT02970266
What this trial is testing

Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.

Who this might be right for
Leber Congenital Amaurosis
Assistance Publique - Hôpitaux de Paris 659
Early research (Phase 1)Study completedNCT01014052
What this trial is testing

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) Mutations

Who this might be right for
LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
QLT Inc. 32
Not applicableNot Yet RecruitingNCT07026565
What this trial is testing

Psychotherapy Group for Parents of Children With LCA

Who this might be right for
Leber Congenital Amaurosis (LCA)
Instituto de Genética Ocular 40
Testing effectiveness (Phase 2)UnknownNCT03872479
What this trial is testing

Single Ascending Dose Study in Participants With LCA10

Who this might be right for
Leber Congenital Amaurosis 10Inherited Retinal DystrophiesEye Diseases, Hereditary+4 more
Editas Medicine, Inc. 34
Large-scale testing (Phase 3)Active Not RecruitingNCT03913143
What this trial is testing

Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
ProQR Therapeutics 36
Load More Results