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NCT02714816 Clinical Trial

Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

Leber Congenital Amaurosis Clinical Trial

Official title:
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT02714816
Other study ID # MGT005
Secondary ID
Status Completed
Phase
First received
Last updated
Start date April 2016
Est. completion date July 22, 2023

Study information
Verified date August 2023
Source MeiraGTx UK II Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

MGT005 is a natural history study to collect longitudinal prospective data from patients with Leber Congenital Amaurosis associated with defects in RPE65.

Description:

Leber Congenital Amaurosis (LCA) is a diagnosis for a group of severe, autosomal recessively inherited rod - cone dystrophies that typically result in complete visual loss in the third or fourth decade of life. One form, LCA2, is caused by a mutation in the gene encoding RPE56, an RPE-specific 65-kDa isomerase. Non-functional RPE65 results in photoreceptor cells that are unable to respond to light resulting in these patients being visually impaired. In preparation for human clinical trials, a detailed prospective phenotypic study will be undertaken to investigate the natural history of RPE65-LCA. Such a study will help identify suitable patients for therapeutic intervention. Furthermore through greater phenotyping an optimal window for intervention and specific parameters to help quantify effect and identify clinical end points may have been ascertained .

Recruitment information / eligibility
Status Completed
Enrollment 37
Est. completion date July 22, 2023
Est. primary completion date July 22, 2023
Accepts healthy volunteers No
Gender All
Age group 3 Years and older
Eligibility Inclusion Criteria: - Patients with RPE65 associated retinal dystrophy - Minimum subject age of 3 years - Able to give consent/parent or guardian able to give consent Exclusion Criteria: - Patients unable or unwilling to undertake consent or clinical testing - Have received a gene therapy treatment in both eyes

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
United Kingdom Moorfields Eye Hospital London
United States Kellogg Eye Center Ann Arbor Michigan

Sponsors (1)
Lead Sponsor Collaborator
MeiraGTx UK II Ltd

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Analysis of retinal structure and function Retinal structure will be analysed using Adaptive optics and SD-OCT and Fundal autofluorescence. This will be correlated with assessment of visual acuity, psychophysical visual assessment, visual mobility, retinal sensitivity and visual fields 6 years
Secondary Quality of Life Questionnaires Assessment of Visual impairment using appropriate, validated questionnaires 6 years
Secondary Retinal Sensitivity To be assessed in Microperimetry 6 years
Secondary Retinal Structural analysis Retinal Structure analysis with Adaptive Optics 6 years
Secondary Fundal Autofluorescence Presence or Absence 6 years
Secondary Assessment of Visual Fields Assessment of Visual Fields with analysis of hill of vision 6 years
See also
  Status Clinical Trial Phase
Completed NCT01496040 - Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 Phase 1/Phase 2
Completed NCT02970266 - Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. N/A
Completed NCT00516477 - Safety Study in Subjects With Leber Congenital Amaurosis Phase 1
Active, not recruiting NCT03920007 - Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D Phase 1/Phase 2
Recruiting NCT05906953 - Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) Phase 1/Phase 2
Active, not recruiting NCT00999609 - Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Phase 3
Active, not recruiting NCT01208389 - Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Phase 1/Phase 2
Terminated NCT03913130 - Extension Study to Study PQ-110-001 (NCT03140969) Phase 1/Phase 2
Recruiting NCT04855045 - 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. Phase 2/Phase 3
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT00749957 - Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Not yet recruiting NCT04731883 - RPE65 Gene Therapy （LX101）for Leber's Congenital Amaurosis N/A
Recruiting NCT05203939 - Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT06064565 - Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT) Early Phase 1
Recruiting NCT06088992 - Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) Early Phase 1
Completed NCT00821340 - Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Phase 1
Active, not recruiting NCT03913143 - A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) Phase 2/Phase 3
Completed NCT02781480 - Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) Phase 1/Phase 2