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Results for “Retinitis Pigmentosa 11”

8 trials

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Showing 8 of 8 results

Early research (Phase 1)Study completedNCT06455826
What this trial is testing

MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)

Who this might be right for
Retinitis Pigmentosa 11Retinal DegenerationEye Diseases+2 more
PYC Therapeutics 6
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Testing effectiveness (Phase 2)Looking for participantsNCT06852963
What this trial is testing

A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001

Who this might be right for
Retinitis Pigmentosa 11Retinal DegenerationRetinal Disease+2 more
PYC Therapeutics 16
Early research (Phase 1)Study completedNCT05902962
What this trial is testing

SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects

Who this might be right for
Retinal DystrophyPRPF31 Mutationassociated Retinal DystrophyRP11
PYC Therapeutics 17
Not applicableActive Not RecruitingNCT04805658
What this trial is testing

Natural History Study of Retinitis Pigmentosa Type 11

Who this might be right for
Retinitis Pigmentosa 11Retinitis Pigmentosa
Oslo University Hospital 30
Not applicableLooking for participantsNCT04463316
What this trial is testing

GROWing Up With Rare GENEtic Syndromes

Who this might be right for
Prader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome+30 more
dr. Laura C. G. de Graaff-Herder 600
Not applicableActive Not RecruitingNCT05573984
What this trial is testing

Natural History of PRPF31 Mutation-Associated Retinal Dystrophy

Who this might be right for
Retinitis PigmentosaEye Diseases, HereditaryRetinal Dystrophies+2 more
PYC Therapeutics 50
Not applicableLooking for participantsNCT06092346
What this trial is testing

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Who this might be right for
AMPD3, OMIM*102772, AMP Deaminase DeficiencyAK1, OMIM *103000, Adenylate Kinase DeficiencyAMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency+25 more
National Human Genome Research Institute (NHGRI)
999