Leber Congenital Amaurosis 2: Clinical Trials

2 trials for Leber Congenital Amaurosis 2, newest first

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1 active not recruiting, 1 completed. .

Follow Leber Congenital Amaurosis 2: new trials by email Literature Review of Leber Congenital Amaurosis 2 trials Research Report on Leber Congenital Amaurosis 2

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene
Leber Congenital Amaurosis 2, Retinitis Pigmentosa 20
CompletedUniversity of Campania Luigi Vanvitelli602020-05-012023-11-29
Early Check: Expanded Screening in Newborns
Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome
Active not recruitingRTI International300002018-10-152026-07-01

Narrower conditions

Amaurosis congenita of Leber, type 2 (2)  ·  3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1)  ·  3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 1 deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 2 deficiency (1)  ·  Acidemia, isovaleric (1)  ·  Acrodermatitis enteropathica (1)  ·  Adenine phosphoribosyltransferase deficiency (1)  ·  Adrenal hyperplasia, congenital, type 5 (1)  ·  Adrenocorticotropic hormone deficiency (1)  ·  Anemia, Sickle Cell (1)  ·  Angelman Syndrome (1)  ·  Aortic Stenosis, Supravalvular (1)  ·  Arginine-Glycine Amidinotransferase Deficiency (1)  ·  Argininosuccinic Aciduria (1)

Broader conditions

Abnormalities, Multiple (1)  ·  Albinism (1)  ·  Albinism, Oculocutaneous (1)  ·  Amino Acid Metabolism, Inborn Errors (1)  ·  Anemia (1)  ·  Anemia, Hemolytic (1)  ·  Anemia, Hemolytic, Congenital (1)  ·  Aortic Valve Disease (1)  ·  Aortic Valve Stenosis (1)  ·  Arrhythmias, Cardiac (1)  ·  Avitaminosis (1)  ·  Basal Ganglia Diseases (1)  ·  Blood Coagulation Disorders (1)  ·  Blood Coagulation Disorders, Inherited (1)  ·  Blood Platelet Disorders (1)

Sponsors

RTI International (1)  ·  University of Campania Luigi Vanvitelli (1)

Countries

Italy (1)  ·  United States (1)

Top investigators

Curt Scharfe, MD, PhD (1)

Results publications

PMID 31315600, PMID 35142618