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History of Changes for Study: NCT01505062
Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
Latest version (submitted March 30, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 January 5, 2012 None (earliest Version on record)
2 February 1, 2012 Recruitment Status, Study Status, Contacts/Locations and Oversight
3 August 1, 2012 Study Status
4 August 9, 2012 Study Status
5 May 10, 2013 Contacts/Locations and Study Status
6 February 14, 2014 Study Status and Arms and Interventions
7 May 29, 2014 Contacts/Locations, Study Status and Eligibility
8 June 24, 2014 Contacts/Locations, Study Identification, Sponsor/Collaborators and Study Status
9 January 16, 2015 Study Status
10 January 21, 2015 Contacts/Locations and Study Status
11 February 4, 2015 Study Status
12 June 1, 2015 Study Identification, Arms and Interventions, Study Description, Study Status, Eligibility and Study Design
13 October 12, 2015 Study Status and Eligibility
14 November 12, 2015 Study Status
15 February 24, 2016 Study Status
16 March 29, 2016 Study Status
17 October 14, 2016 Study Status
18 March 15, 2017 Study Status
19 October 18, 2017 Study Status
20 July 9, 2018 Contacts/Locations and Study Status
21 July 13, 2018 Study Status
22 January 28, 2019 Arms and Interventions, Eligibility, Outcome Measures, Study Description, Study Identification, Study Design and Study Status
23 May 3, 2019 Study Status and Contacts/Locations
24 September 26, 2019 Recruitment Status, Study Status, Contacts/Locations and Study Design
25 January 16, 2020 Study Status
Show
Results Submission Events
26 March 16, 2020 Study Status, Arms and Interventions, Outcome Measures, Study Description, Study Identification, Document Section, Results, IPDSharing and Eligibility
27 March 30, 2022 Study Status and IPDSharing
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Study NCT01505062
Submitted Date:  January 5, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: US1/001/10
Brief Title: Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
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
Secondary IDs:
Open or close this module Study Status
Record Verification: January 2012
Overall Status: Not yet recruiting
Study Start: January 2012
Primary Completion: December 2014 [Anticipated]
Study Completion: December 2014 [Anticipated]
First Submitted: January 4, 2012
First Submitted that
Met QC Criteria:
January 5, 2012
First Posted: January 6, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
January 5, 2012
Last Update Posted: January 6, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Oxford BioMedica
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: 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.
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.
Open or close this module Conditions
Conditions: Usher Syndrome
Retinitis Pigmentosa
Keywords: Usher Syndrome Retinitis Pigmentosa
Usher Syndrome associated Retinitis Pigmentosa
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms:
Masking: None (Open Label)
Allocation: N/A
Enrollment: 18 [Anticipated]
Open or close this module Arms and Interventions
Intervention Details:
Genetic: UshStat
Single dose, 3 ascending dose cohorts.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. The incidence of adverse events
[ Time Frame: 48 weeks ]

The number and percentage of patients with treatment emergent adverse events.
Secondary Outcome Measures:
1. To determine delay in retinal degeneration.
[ Time Frame: 48 weeks ]

Changes in function relative to the contralateral eye utilizing retinal analytical techniques.
Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
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
Open or close this module Contacts/Locations
Central Contact Person: Richard G Weleber, MD
Telephone: 503 494 3795
Study Officials: Richard G Weleber, MD
Principal Investigator
Casey Eye Institute, Portland, Oregon
Locations: United States, Oregon
Casey Eye Institute
Portland, Oregon, United States, 97239-4197
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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