Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2015 by Sanofi
Information provided by (Responsible Party):
Sanofi Identifier:
First received: January 4, 2012
Last updated: February 4, 2015
Last verified: February 2015

The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, UshStat designed to treat retinitis pigmentosa associated with Usher Syndrome Type 1B.

Condition Intervention Phase
Usher Syndrome
Retinitis Pigmentosa
Genetic: UshStat
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy 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 UshStat, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

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:
  • To determine delay in retinal degeneration. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    Changes in function relative to the contralateral eye utilizing retinal analytical techniques.

Estimated Enrollment: 18
Study Start Date: March 2012
Estimated Study Completion Date: May 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Intervention Details:
    Genetic: UshStat
    Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts
Detailed Description:

Following screening procedures the gene transfer agent will be injected once only under one retina by an opthalmic surgeon under anesthesia. Patients will then have regular follow up visits where general health examinations, blood tests and ophthalmic examinations including best corrected visual acuity, slit lamp examination, intraocular pressure, fundoscopy, autofluorescence, Optical Coherence Tomography, perimetry and Electroretinogram will be undertaken.


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

Inclusion Criteria:

  • Clinical and molecular diagnosis of Retinitis Pigmentosa associated with Usher Syndrome type 1B. With at least one pathogenic mutation in the MYO7A gene on each chromosome.
  • Suitable verbal, auditory,written and/or tactile sign language communication as to allow informed consent to be obtained.
  • Women of childbearing potential must agree to use two forms of contraception.
  • Males must agree to use two forms of contraception if their study partner is of child-bearing potential for three months after treatment.
  • Affiliated with the French social security healthcare system (french patients only)

Exclusion Criteria:

  • Presence of significant ocular abnormalities that would preclude surgery, effective study follow up or interfere with study endpoints.
  • Concomitant systemic diseases that can alter visual function.
  • Contraindication to pupil dilation, anesthesia or eye surgery.
  • Periocular steroids in the last four months prior to screen.
  • Known allergies to the components of the delivery vehicle or diagnostic agents such as dilation drops.
  • Life-threatening illness or a history of malignancy within 5 years
  • Laboratory test abnormalities that would make the patient unsuitable for participation in the study
  • Intercurrent illnesses or infections 28 days prior to treatment
  • Concurrent anti-retroviral or immunosuppressant therapy
  • Men or women who do not agree to use contraception as specified in the inclusion criteria.
  • Pregnant or breastfeeding women
  • 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
  • Any ocular surgery in the study eye within 6 months
  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: NCT01505062

Contact: For site information, send an email with site number to

United States, Oregon
Investigational Site Number 840001 Recruiting
Portland, Oregon, United States, 97239-3098
Investigational Site Number 250001 Recruiting
Paris, France, 75012
Sponsors and Collaborators
Principal Investigator: Richard Weleber, MD Casey Eye Institute, Portland, Oregon
Principal Investigator: Jose-Alain Sahel, MD, PhD Hopital Nationale des Quinze-Vingt, Paris France
  More Information

No publications provided by Sanofi

Additional publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Sanofi Identifier: NCT01505062     History of Changes
Other Study ID Numbers: TDU13600, US1/001/10
Study First Received: January 4, 2012
Last Updated: February 4, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Sanofi:
Usher Syndrome Retinitis Pigmentosa
Usher Syndrome associated Retinitis Pigmentosa

Additional relevant MeSH terms:
Usher Syndromes
Retinitis Pigmentosa
Abnormalities, Multiple
Congenital Abnormalities
Deaf-Blind Disorders
Ear Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Hearing Disorders
Hearing Loss
Hearing Loss, Sensorineural
Nervous System Diseases
Neurologic Manifestations
Otorhinolaryngologic Diseases
Pathologic Processes
Retinal Degeneration
Retinal Diseases
Retinal Dystrophies
Sensation Disorders
Signs and Symptoms
Vision Disorders processed this record on May 26, 2015