N/A N=53 Natural History Study in Subjects With Usher Syndrome
Usher Syndrome, Type 1B
Primary: BCVA - Better Seeing Eyes — -2.26 ETDRS score — p=<0.01
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=44 Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
Retinitis Pigmentosa · Usher Syndrome Type 2 · Usher Syndrome Type 3
Primary: Mean Change in Cone Spacing in Arcminutes (Z Score) of 2 Baseline Values Were Compared With Measurements Obtained at Post-op Month 36 — 1.13; 0.85 Difference in change…
Phase 2 N=5 Nitisinone for Type 1B Oculocutaneous Albinism
Albinism · Vision Loss
Primary: Absolute Mean Change in Iris Pigmentation on an 8-point Iris Transillumination Scale at 12 Months as Compared to Baseline. Participants Left and Right Eyes Will be…
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Phase 2 N=23 Gene Therapy for Achromatopsia (CNGB3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
N/A N=11 Audiovestibular Function in Infratentorial Superficial Siderosis
Siderosis · Neurological Disorder · Rare Diseases
Primary: Hearing Evaluation - Pure Tone Audiometry — 57.3 decibel hearing level
N/A N=998 Multicenter, Non-interventional Study, Describing Patients With Inherited Retinal Disease (IRD) in France
Inherited Retinal Disease
Primary: Proportion of Patients by Type of IRD — 720; 174; 30; 58 Participants
Phase 2 N=18 L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Phase 2 N=15 Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Leber Congenital Amaurosis
Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Phase 2 N=45 Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=11 Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
Phase 2 N=12 Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Retinoschisis · X-Linked
Primary: Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS — 3; 3; 12; 6 events
Phase 2 N=11 Gene Therapy for Achromatopsia (CNGA3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
N/A N=301 Vestibular Infants Screening-Flanders
Vestibular Disorder · Hearing Impaired Children
Primary: Number of Hearing-impaired Infants in Flanders With Abnormal Vestibular Screening Results. — 35 Participants
Phase 2 N=27 Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked Retinoschisis
Primary: Number of Participants Experiencing Adverse Events — 6; 7; 12 Participants