A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Sanofi
ClinicalTrials.gov Identifier:
NCT02065011
First received: February 14, 2014
Last updated: June 24, 2014
Last verified: June 2014
  Purpose

The purpose of the study is to examine the long-term safety of an experimental gene transfer agent UshStat, designed to treat retinitis pigmentosa associated with Ushers Syndrome Type 1b.


Condition Intervention Phase
Usher Syndrome Type 1b
Drug: UshStat
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients 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: 15 years ] [ Designated as safety issue: Yes ]
    The number and percentage of patients with treatment emergent adverse events.


Secondary Outcome Measures:
  • Determine delay in retinal degeneration [ Time Frame: 15 years ] [ Designated as safety issue: No ]
    Biological Activity: To determine a delay in retinal degeneration following subretinal injection of UshStat®, through changes in function relative to the untreated contralateral eye utilising retinal analytical techniques


Estimated Enrollment: 18
Study Start Date: September 2013
Estimated Study Completion Date: January 2032
Estimated Primary Completion Date: January 2032 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Long-term follow up
Long-term follow up of patients who received UshStat in a previous study
Drug: UshStat
Long-term follow up of patients who received UshStat in a previous study

  Eligibility

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

Inclusion Criteria:

  • Must have been enrolled in Protocol TDU13600
  • Must have received a subretinal injection of UshStat®

Exclusion Criteria:

  • Did not receive UshStat® as part of the TDU13600 protocol
  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: NCT02065011

Locations
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239-4197
Sponsors and Collaborators
Sanofi
Investigators
Principal Investigator: Richard Weleber, MD Casey Eye Institute, Portland Oregon
  More Information

No publications provided

Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02065011     History of Changes
Other Study ID Numbers: LTS13619, US1/002/13
Study First Received: February 14, 2014
Last Updated: June 24, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Usher Syndromes
Syndrome
Disease
Pathologic Processes
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
Retinitis Pigmentosa
Retinal Dystrophies
Retinal Degeneration
Retinal Diseases
Eye Diseases
Abnormalities, Multiple
Congenital Abnormalities
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Signs and Symptoms

ClinicalTrials.gov processed this record on October 19, 2014