Trials Sponsors Conditions Drugs Pricing

Conditions

Leber Congenital Amaurosis

Outcome Measures

Primary Outcomes (1)

Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
6 months

Study Arms (3)

Low dose AAV-RPE65

EXPERIMENTAL

Subretinal administration of a single low dose of range AAV-RPE65

Biological: AAV RPE65

Intermediate dose AAV-RPE65

EXPERIMENTAL

Subretinal administration of a single intermediate dose of range AAV-RPE65

Biological: AAV RPE65

High dose AAV-RPE65

EXPERIMENTAL

Subretinal administration of a single high dose of range AAV-RPE65

Biological: AAV RPE65

Interventions

AAV RPE65Biological

Comparison of different dosages of AAV RPE65

High dose AAV-RPE65Intermediate dose AAV-RPE65Low dose AAV-RPE65

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age3 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Aged 3 years or older
Early-onset severe retinal dystrophy consistent with RPE65 deficiency

You may not qualify if:

Sponsors & Collaborators

MeiraGTx UK II Ltd
Industrylead

Study Sites (2)

Kellogg Eye Centre, University of Michigan

Ann Arbor, Michigan, 48105, United States

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

MeSH Terms

Conditions

Leber Congenital Amaurosis

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Results Point of Contact

Title: Chief Medical Officer
Organization: MeiraGTx

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

15 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 28, 2016

First Posted

May 24, 2016

Study Start

April 2016

Date type not reported

This date has not been verified as actual or estimated.

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Study Arms (3)

Low dose AAV-RPE65

Intermediate dose AAV-RPE65

High dose AAV-RPE65

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Study Arms (3)

Low dose AAV-RPE65

Intermediate dose AAV-RPE65

High dose AAV-RPE65

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

Source record

Documents

Locations