NCT02714816

Brief Summary

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
37

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Apr 2016

Longer than P75 for all trials

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 8, 2016

Completed
14 days until next milestone

First Posted

Study publicly available on registry

March 22, 2016

Completed
10 days until next milestone

Study Start

First participant enrolled

April 1, 2016

Completed
7.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 22, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 22, 2023

Completed
Last Updated

August 14, 2023

Status Verified

August 1, 2023

Enrollment Period

7.3 years

First QC Date

March 8, 2016

Last Update Submit

August 10, 2023

Conditions

Leber Congenital Amaurosis

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (5)

  • Quality of Life Questionnaires

    6 years

  • Retinal Sensitivity

    6 years

  • Retinal Structural analysis

    6 years

  • Fundal Autofluorescence

    6 years

  • Assessment of Visual Fields

    6 years

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with RPE65-LCA condition

You may qualify if:

  • Patients with RPE65 associated retinal dystrophy
  • Minimum subject age of 3 years
  • Able to give consent/parent or guardian able to give consent

You may not qualify if:

  • Patients unable or unwilling to undertake consent or clinical testing
  • Have received a gene therapy treatment in both eyes

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Kellogg Eye Center

Ann Arbor, Michigan, MI 48105, United States

Location

Moorfields Eye Hospital

London, United Kingdom

Location

Related Publications (2)

  • Kumaran N, Georgiou M, Bainbridge JWB, Bertelsen M, Larsen M, Blanco-Kelly F, Ayuso C, Tran HV, Munier FL, Kalitzeos A, Michaelides M. Retinal Structure in RPE65-Associated Retinal Dystrophy. Invest Ophthalmol Vis Sci. 2020 Apr 9;61(4):47. doi: 10.1167/iovs.61.4.47.

    PMID: 32347917DERIVED

  • Kumaran N, Rubin GS, Kalitzeos A, Fujinami K, Bainbridge JWB, Weleber RG, Michaelides M. A Cross-Sectional and Longitudinal Study of Retinal Sensitivity in RPE65-Associated Leber Congenital Amaurosis. Invest Ophthalmol Vis Sci. 2018 Jul 2;59(8):3330-3339. doi: 10.1167/iovs.18-23873.

    PMID: 30025081DERIVED

MeSH Terms

Conditions

Leber Congenital Amaurosis

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Study Officials

  • Michel Michealides, Prof

    UCL/Moorfileds

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 8, 2016

First Posted

March 22, 2016

Study Start

April 1, 2016

Primary Completion

July 22, 2023

Study Completion

July 22, 2023

Last Updated

August 14, 2023

Record last verified: 2023-08

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)US(1)