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Trial record 1 of 4 for:    Recruiting, Not yet recruiting, Available Studies | "Usher Syndromes"
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Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

This study is currently recruiting participants.
Verified October 2017 by Sanofi
Sponsor:
ClinicalTrials.gov Identifier:
NCT01505062
First Posted: January 6, 2012
Last Update Posted: October 19, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi
  Purpose

To evaluate the safety and tolerability of ascending doses of subretinal injections of UshStat in patients with Usher syndrome type 1B.

To evaluate for possible biological activity of UshStat.


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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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 ]
    The number and percentage of patients with treatment emergent adverse events.


Secondary Outcome Measures:
  • To determine delay in retinal degeneration. [ Time Frame: 48 weeks ]
    Changes in function relative to the contralateral eye utilizing retinal analytical techniques.


Estimated Enrollment: 18
Actual Study Start Date: March 20, 2012
Estimated Study Completion Date: February 24, 2020
Estimated Primary Completion Date: February 24, 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SAR421869 (Cohort 1)
Starting dose of UshStat given through subretinal injection
Genetic: UshStat
Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts
Experimental: SAR421869 (Cohort 2)
Escalating dose of UshStat given through subretinal injection
Genetic: UshStat
Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts
Experimental: SAR421869 (Cohort 3)
Escalating dose of UshStat given through subretinal injection
Genetic: UshStat
Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts
Experimental: SAR421869 (Cohort 4)
Maximum tolerated dose (MTD)/eye of UshStat given through subretinal injection
Genetic: UshStat
Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts
Experimental: SAR421869 (Cohort 5)
MTD/eye of UshStat given through subretinal injection
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.

At the end of the study, the patient will be invited to enter in an open-label safety study and long-term follow-up visits (at least once every six months) including ophthalmological examinations and recording of adverse events will continue for 5 years; then the Investigator will follow the patient for a subsequent 10 years at a minimum interval of once a year to monitor delayed adverse events.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01505062


Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com

Locations
United States, Oregon
Investigational Site Number 840001 Recruiting
Portland, Oregon, United States, 97239-3098
France
Investigational Site Number 250001 Recruiting
Paris, France, 75012
Sponsors and Collaborators
Sanofi
Investigators
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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT01505062     History of Changes
Other Study ID Numbers: TDU13600
US1/001/10 ( Other Identifier: Oxford Biomedica )
First Submitted: January 4, 2012
First Posted: January 6, 2012
Last Update Posted: October 19, 2017
Last Verified: October 2017

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

Additional relevant MeSH terms:
Usher Syndromes
Syndrome
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Disease
Pathologic Processes
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn
Deaf-Blind Disorders
Deafness
Hearing Loss
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Hearing Loss, Sensorineural
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Blindness
Vision Disorders
Abnormalities, Multiple
Congenital Abnormalities
Signs and Symptoms