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Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

NCT ID: NCT00643747

Last Updated: 2015-12-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-01-31

Study Completion Date

2014-12-31

Brief Summary

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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

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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.

Conditions

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Retinal Degeneration

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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A

Injection of vector

Group Type EXPERIMENTAL

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Intervention Type BIOLOGICAL

Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Interventions

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tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Intervention Type BIOLOGICAL

Other Intervention Names

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rAAV 2/2.hRPE65p.hRPE65

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Moorfields Eye Hospital NHS Foundation Trust

OTHER

Sponsor Role collaborator

Targeted Genetics Corporation

INDUSTRY

Sponsor Role collaborator

University College, London

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Robin R Ali, PhD

Role: STUDY_DIRECTOR

University College, London

Locations

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Moorfields Eye Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

Countries

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United Kingdom

References

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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.

Reference Type BACKGROUND
PMID: 25938638 (View on PubMed)

Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2231-9. doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.

Reference Type RESULT
PMID: 18441371 (View on PubMed)

Ripamonti C, Henning GB, Robbie SJ, Sundaram V, van den Born LI, Casteels I, de Ravel TJ, Moore AT, Smith AJ, Bainbridge JW, Ali RR, Stockman A. Spectral sensitivity measurements reveal partial success in restoring missing rod function with gene therapy. J Vis. 2015;15(15):20. doi: 10.1167/15.15.20.

Reference Type DERIVED
PMID: 26605849 (View on PubMed)

Other Identifiers

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06/061

Identifier Type: -

Identifier Source: org_study_id