Clinical Trial Details

NCT ID: NCT01505062
Date Last Changed: June 1, 2015

Overview

Research Study Summary

Patients are needed to participate in a clinical research study of UshStat to evaluate Usher Syndrome or Retinitis Pigmentosa

Research Study 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

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.

To Learn more

Recruitment Details

Phase
1/2
Gender
Both Male and Female
Age
18 and up
Overall Status
Recruiting
Lead Sponsor
Sanofi
Duration
62 Months
Facility Type
N/A
Compensation

Eligibility

Both Male and Female ages 18 Years and up

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

Site Locations (2)

Country State City Zip Facility and Contact
United States Oregon Portland 97239-3098 Investigational Site Number 840001
France Paris 75012 Investigational Site Number 250001

Contact

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