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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 February 2014 by Oxford BioMedica
Sponsor:
Information provided by (Responsible Party):
Oxford BioMedica
ClinicalTrials.gov Identifier:
NCT01505062
First received: January 4, 2012
Last updated: February 14, 2014
Last verified: February 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 Oxford BioMedica:

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: July 2016
Estimated Primary Completion Date: July 2016 (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 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.
  • 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.
  • Laboratory test abnormalities
  • Intercurrent illnesses or infections 28 days prior to treatment
  • Concurrent anti-retroviral therapy
  • Men or women who do not agree to use contraception as specified in the inclusion criteria.
  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01505062

Contacts
Contact: Richard G Weleber, MD 503 494 3795

Locations
United States, Oregon
Casey Eye Institute Recruiting
Portland, Oregon, United States, 97239-4197
France
Central Hospitalier Nationale d'Opthalmolgie des Quinze-Vingts Not yet recruiting
Paris, France, 75571
Sponsors and Collaborators
Oxford BioMedica
Investigators
Principal Investigator: Richard G Weleber, MD Casey Eye Institute, Portland, Oregon
Principal Investigator: Jose-Alain Sahel, MD Hospital Nationale des Quinze-Vingt, Paris, France
  More Information

No publications provided

Responsible Party: Oxford BioMedica
ClinicalTrials.gov Identifier: NCT01505062     History of Changes
Other Study ID Numbers: US1/001/10
Study First Received: January 4, 2012
Last Updated: February 14, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Oxford BioMedica:
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 April 17, 2014