Safety Study of RPE65 Gene Therapy to Treat Leber Congeni... | NCT00643747

Studies
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

NCT00643747

Sponsor

University College, London

Status

Completed

Last Update Posted

Dec 7, 2015Estimated

Enrollment

12Actual

Phase

Phase 1Phase 2

Conditions

Retinal Degeneration

Interventions

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2013

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2007

Primary Completion Date

Dec 2014Actual

Completion Date

Dec 2014Actual

First Submitted Date

Mar 20, 2008

First Submission Date that Met QC Criteria

Mar 25, 2008

First Posted Date

Mar 26, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 4, 2015

Last Update Posted Date

Dec 7, 2015Estimated

Name	Class
Moorfields Eye Hospital NHS Foundation Trust	OTHER
Targeted Genetics Corporation	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.

Detailed Description

The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
A	Experimental	Injection of vector	Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Interventions

Name	Type	Description	Arm Group Labels	Other Names
tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)	Biological	Single subretinal injection of vector suspension; up to 3x10e12 vector particles	A

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
intraocular inflammation		at intervals up to 12 months

Secondary Outcomes

Measure	Description	Time Frame
visual function		intervals up to 12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65

Exclusion Criteria:

Visual acuity in the study eye better than 6/36 Snellen
Hypertension
Diabetes mellitus
Tuberculosis
Renal impairment
Immunocompromise
Osteoporosis
Gastric ulceration
Severe affective disorder)
Pregnancy or lactation

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

5 Years

Maximum Age

30 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Robin R Ali, PhD	University College, London	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Moorfields Eye Hospital NHS Foundation Trust		London		EC1V 2PD	United Kingdom

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
25938638	Background	Bainbridge JW, Mehat MS, Sundaram V, Robbie SJ, Barker SE, Ripamonti C, Georgiadis A, Mowat FM, Beattie SG, Gardner PJ, Feathers KL, Luong VA, Yzer S, Balaggan K, Viswanathan A, de Ravel TJ, Casteels I, Holder GE, Tyler N, Fitzke FW, Weleber RG, Nardini M, Moore AT, Thompson DA, Petersen-Jones SM, Michaelides M, van den Born LI, Stockman A, Smith AJ, Rubin G, Ali RR. Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4.
18441371

ID	Term
D012162	Retinal Degeneration
D058499	Retinal Dystrophies
D057130	Leber Congenital Amaurosis

ID	Term
D015785	Eye Diseases, Hereditary
D005128	Eye Diseases
D012164	Retinal Diseases