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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:
ClinicalTrials.gov Identifier:
NCT02065011
First Posted: February 17, 2014
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. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi
  Purpose

Primary Objective:

To evaluate the long-term safety and tolerability of UshStat® in patients with Usher syndrome type 1B

Secondary Objective:

To assess long-term safety and biological activity of UshStat®


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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
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: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]
    The number and percentage of patients with treatment emergent adverse events.


Secondary Outcome Measures:
  • Change from baseline in ocular safety assessments [ Time Frame: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]
    Best Corrected Visual Acuity (BCVA), Slit-lamp, Funduscopy, Intraocular Pressure, Lab parameters, concomitant meds

  • Delay in retinal degeneration [ Time Frame: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]
    Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, kinetic, static perimetry, microperimetry, autofluorescence, ERG, OCT


Estimated Enrollment: 18
Actual Study Start Date: September 12, 2013
Estimated Study Completion Date: March 20, 2035
Estimated Primary Completion Date: March 20, 2035 (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

Inclusion Criteria:

  • Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA).
  • 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
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): NCT02065011


Locations
United States, Oregon
Investigational Site Number 840001
Portland, Oregon, United States, 97239-3098
France
Investigational Site Number 250001
Paris, France, 75012
Sponsors and Collaborators
Sanofi
Investigators
Principal Investigator: Richard Weleber, MD Casey Eye Institute, Portland Oregon
  More Information

Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02065011     History of Changes
Other Study ID Numbers: LTS13619
US1/002/13
First Submitted: February 14, 2014
First Posted: February 17, 2014
Last Update Posted: October 19, 2017
Last Verified: October 2017

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