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Results for “Meckel-Gruber Syndrome”

11 trials

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Showing 11 of 11 results

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
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
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
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
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
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
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 completedNCT03823014
What this trial is testing

Use of an External Erectile Device in Transgender Man Following Phalloplasty

Who this might be right for
TranssexualismSurgerySex Behavior+1 more
Boston Children's Hospital 15
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
Not applicableLooking for participantsNCT01401998
What this trial is testing

ARPKD Database Study

Who this might be right for
Hepato/Renal Fibrocystic DiseaseAutosomal Recessive Polycystic Kidney DiseaseJoubert Syndrome+7 more
Children's Hospital of Philadelphia 200
Not applicableStudy completedNCT00873678
What this trial is testing

Assessment of the Prevalence of Genes AHI1, NPHP1 and CEP290 in Joubert Syndrome

Who this might be right for
Joubert SyndromeCerebello-oculo-renal Syndromes
Assistance Publique - Hôpitaux de Paris 80