This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2015 by Sanofi
Sponsor:
Sanofi
Information provided by (Responsible Party):
Sanofi

ClinicalTrials.gov Identifier:
NCT01367444
First received: June 3, 2011
Last updated: January 20, 2015
Last verified: January 2015
History of Changes

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 Stargardt macular degeneration
MedlinePlus related topics: Genes and Gene Therapy Macular Degeneration
Genetic and Rare Diseases Information Center resources: Stargardt Disease
U.S. FDA Resources

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.

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 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.

Find Studies Study Record Detail
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
Sponsor:
Sanofi
Information provided by (Responsible Party):
Sanofi
ClinicalTrials.gov Identifier:
NCT01367444
First received: June 3, 2011
Last updated: January 20, 2015
Last verified: January 2015
History of Changes
Full Text View Tabular View No Study Results PostedDisclaimerHow to Read a Study Record

Tracking Information
First Received Date ICMJE June 3, 2011
Last Updated Date January 20, 2015
Start Date ICMJE June 2011
Estimated Primary Completion Date February 2017 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE
(submitted: June 6, 2011) 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.
Original Primary Outcome Measures ICMJE Same as current
Change History Complete list of historical versions of study NCT01367444 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE
(submitted: June 6, 2011) 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.
Original Secondary Outcome Measures ICMJE Same as current
Current Other Outcome Measures ICMJE Not Provided
Original Other Outcome Measures ICMJE Not Provided

Descriptive Information
Brief Title ICMJE Phase I/IIa Study of StarGen in Patients With Stargardt Macular Degeneration
Official Title ICMJE A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen Administered to Patients With Stargardt Macular Degeneration
Brief Summary
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.

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.

Study Type ICMJE Interventional
Study Phase Phase 1
Phase 2
Study Design ICMJE Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Condition ICMJE Stargardt Disease
Intervention ICMJE Genetic: StarGen
3 dose cohorts, gene therapy
Study Arm (s) Experimental: Subretinal injection StarGen
Dose escalation of subretinally injected StarGen
Intervention: Genetic: StarGen
Publications * Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ICMJE Recruiting
Estimated Enrollment ICMJE 28
Estimated Completion Date February 2017
Estimated Primary Completion Date February 2017 (final data collection date for primary outcome measure)
Eligibility Criteria ICMJE
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.
Gender Both
Ages 18 Years and older
Accepts Healthy Volunteers No
Contacts ICMJE
Contact: David Wilson, M.D 503 494 3795
Listed Location Countries ICMJE United States, France
Removed Location Countries

Administrative Information
NCT Number ICMJE NCT01367444
Other Study ID Numbers ICMJE TDU13583, SG1/001/10
Has Data Monitoring Committee Yes
Responsible Party Sanofi
Study Sponsor ICMJE Sanofi
Collaborators ICMJE Not Provided
Investigators ICMJE
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
Information Provided By Sanofi
Verification Date January 2015

ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

No Study Results Posted on ClinicalTrials.gov for this Study
About Study Results Reporting on ClinicalTrials.gov
Study Status: This study is currently recruiting participants.
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)

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

Contacts
Contact: David Wilson, M.D 503 494 3795

Locations
United States, Oregon
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
Sanofi
Investigators
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

Responsible Party: Sanofi
ClinicalTrials.gov 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

ClinicalTrials.gov processed this record on February 08, 2015