Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Phase I/IIa Study of StarGen in Patients With Stargardt Macular Degeneration

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2015 by Sanofi
Information provided by (Responsible Party):
Sanofi Identifier:
First received: June 3, 2011
Last updated: January 20, 2015
Last verified: January 2015

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:

Further study details as provided by Sanofi:

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: February 2017
Estimated Primary Completion Date: February 2017 (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 an additional twelve patients.


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

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 within six months.
  • Aphakia or prior vitrectomy to the study eye.
  • Concomitant systemic diseases that can alter visual function.
  • 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 which make the patient unsuitable for the study.
  • 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 or anti-retroviral agents
  • Past history of HIV or hepatitis A, B or C.
  • Inability to comply with the demands of the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01367444

Contact: David Wilson, M.D 503 494 3795

United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239-3098
Principal Investigator: David Wilson, MD         
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
Principal Investigator: David Wilson, MD Oregon Health & Science University, Portland, Oregon
Principal Investigator: Jose-Alain Sahel, MD. Ph.D Hopital Nationale des Quinze-Vingt, Paris France
  More Information

No publications provided

Responsible Party: Sanofi Identifier: NCT01367444     History of Changes
Other Study ID Numbers: TDU13583, SG1/001/10
Study First Received: June 3, 2011
Last Updated: January 20, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Sanofi:
Stargardt Disease

Additional relevant MeSH terms:
Macular Degeneration
Eye Diseases
Retinal Degeneration
Retinal Diseases processed this record on March 01, 2015