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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

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ClinicalTrials.gov Identifier: NCT02065011
Recruitment Status : Enrolling by invitation
First Posted : February 17, 2014
Last Update Posted : July 16, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi

Brief Summary:

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 or disease Intervention/treatment Phase
Usher Syndrome Drug: UshStat Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
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
Actual Study Start Date : September 12, 2013
Estimated Primary Completion Date : January 15, 2036
Estimated Study Completion Date : January 15, 2036


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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 :
  1. 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

  2. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
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

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

Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02065011     History of Changes
Other Study ID Numbers: LTS13619
US1/002/13
First Posted: February 17, 2014    Key Record Dates
Last Update Posted: July 16, 2018
Last Verified: July 2018

Additional relevant MeSH terms:
Syndrome
Usher Syndromes
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