Trial record 2 of 9 for:    "Usher syndrome"

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 June 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi
ClinicalTrials.gov Identifier:
NCT01505062
First received: January 4, 2012
Last updated: June 24, 2014
Last verified: June 2014
  Purpose

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: January 2012
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: January 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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.
  • 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 ClinicalTrials.gov identifier: NCT01505062

Contacts
Contact: Richard Weleber, MD 503-494-7887

Locations
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239-4197
Principal Investigator: Richard Weleber, MD         
France
Central Hospitalier Nationale d'Opthalmolgie des Quinze-Vingts Recruiting
Paris, France, 75571
Principal Investigator: Jose Sahel, MD         
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

No publications provided by Sanofi

Additional 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
Study First Received: January 4, 2012
Last Updated: June 24, 2014
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
Retinitis Pigmentosa
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

ClinicalTrials.gov processed this record on July 24, 2014