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
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Leber Congenital Amaurosis”

37 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 37 results

Testing effectiveness (Phase 2)Not Yet RecruitingNCT07681778
What this trial is testing

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

Who this might be right for
Leber Congenital AmaurosisLeber Congenital Amaurosis (LCA)
Opus Genetics, Inc 10
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
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
Testing effectiveness (Phase 2)Ended earlyNCT03913130
What this trial is testing

Extension Study to Study PQ-110-001 (NCT03140969)

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
Laboratoires Thea 9
Not applicableStudy completedNCT03597399
What this trial is testing

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Who this might be right for
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Spark Therapeutics, Inc. 87
Testing effectiveness (Phase 2)Study completedNCT00643747
What this trial is testing

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Who this might be right for
Retinal Degeneration
University College, London 12
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
Not applicableLooking for participantsNCT02435940
What this trial is testing

Inherited Retinal Degenerative Disease Registry

Who this might be right for
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting Blindness 20,000
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
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 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 completedNCT00422721
What this trial is testing

Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy

Who this might be right for
AmaurosisRetinal Diseases
Nantes University Hospital 360
Not applicableActive Not 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)Active Not RecruitingNCT03920007
What this trial is testing

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Who this might be right for
Leber Congenital AmaurosisLCALCA1
Atsena Therapeutics Inc. 15
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
Large-scale testing (Phase 3)UnknownNCT04855045
What this trial is testing

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.

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+8 more
ProQR Therapeutics 15
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
Not applicableStudy completedNCT04525261
What this trial is testing

Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene

Who this might be right for
Leber Congenital Amaurosis 2Retinitis Pigmentosa 20
University of Campania Luigi Vanvitelli 60
Testing effectiveness (Phase 2)Active Not RecruitingNCT05203939
What this trial is testing

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Who this might be right for
Retinitis PigmentosaLeber Congenital Amaurosis
Ocugen 22
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
Next Page