Phase I/IIa Study of StarGen in Patients With Stargardt Macular Degeneration - Full Text View

Sponsor:	Oxford BioMedica
Information provided by (Responsible Party):	Oxford BioMedica
ClinicalTrials.gov Identifier:	NCT01367444

Purpose

The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, StarGen, designed to treat StarGardt Macular Degeneration.

Condition	Intervention	Phase
Stargardt Disease	Genetic: StarGen	Phase 1 Phase 2

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen Administered to Patients With Stargardt Macular Degeneration

Resource links provided by NLM:

Genetics Home Reference related topics: age-related macular degeneration X-linked juvenile retinoschisis

MedlinePlus related topics: Macular Degeneration

U.S. FDA Resources

Further study details as provided by Oxford BioMedica:

Primary Outcome Measures:

The incidence of adverse events [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
The number and percentage of patients with treatment emergent adverse events.

Secondary Outcome Measures:

Delay in retinal degeneration [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
Changes from baseline function relative to the contralateral eye utilising retinal analytical techniques.

Estimated Enrollment:	28
Study Start Date:	June 2011
Estimated Study Completion Date:	October 2013
Estimated Primary Completion Date:	July 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Subretinal injection StarGen Dose escalation of subretinally injected StarGen	Genetic: StarGen 3 dose cohorts, gene therapy

Detailed Description:

There are two parts to the study. A dose-escalation phase looking at three doses of StarGen, eight patients will be recruited at the first dose level, and four each at the next two dose levels. This will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in up to twelve patients.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Clinical diagnosis of Stargardt Disease with at least one pathogenic mutation in the ABCA 4 gene on each chromosome.
Women of childbearing potential must agree to use effective contraception.
Males must agree to use to use two forms of contraception if their study partner is of child-bearing potential for three months after treatment.

Exclusion Criteria:

Pre-existing eye conditions that would preclude surgery.
Cataract surgery.
Aphakia or prior vitrectomy to the study eye.
Concomitant systemic diseases.
any contraindications to pupil dilation.
Any known allergy to any of the components of starGen or diagnostic agents.
Any injectable intravitreal treatment within the six months prior to screen.
Any periocular steroids within four months prior to screen.
Any laboratory abnormality.
Men or women who do not agree to use contraception as specified in the inclusion criteria.
Contraindications to anesthesia.
History of prior gene transfer treatment.
Enrollment in any other clinical study during the 48 week study period.
Treatment with anticoagulants
Past history of HIV or hepatitis A, B or C.
Inability to comply with the demands of the study.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01367444

Contacts

Contact: David Wilson, M.D

503 494 3795

Locations

United States, Oregon
Casey Eye Institute, Oregon Health & Science University	Recruiting
Portland, Oregon, United States, 97239-3098
Principal Investigator: David Wilson, MD
France
Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts	Recruiting
Paris, France, 75571
Contact: Jose Sahel, MD. Ph.D
Principal Investigator: Jose Sahel, MD.Ph.D

Sponsors and Collaborators

Oxford BioMedica

Investigators

Principal Investigator:	David Wilson, MD	Casey Eye Institute, Portland, Oregon
Principal Investigator:	Jose-Alain Sahel, MD. Ph.D	Hopital Nationale des Quinze-Vingt, Paris France

More Information

No publications provided

Responsible Party:	Oxford BioMedica
ClinicalTrials.gov Identifier:	NCT01367444 History of Changes
Other Study ID Numbers:	SG1/001/10
Study First Received:	June 3, 2011
Last Updated:	December 19, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Oxford BioMedica:

Stargardt Disease

Additional relevant MeSH terms:

Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases

ClinicalTrials.gov processed this record on May 23, 2012