Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
This study is currently recruiting participants.
Verified May 2013 by Oxford BioMedica
Sponsor:
Oxford BioMedica
Information provided by (Responsible Party):
Oxford BioMedica
ClinicalTrials.gov Identifier:
NCT01505062
First received: January 4, 2012
Last updated: May 10, 2013
Last verified: May 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Usher Syndrome Retinitis Pigmentosa |
Genetic: UshStat |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Lenz microphthalmia syndrome
nonsyndromic deafness
oculofaciocardiodental syndrome
Pendred syndrome
Peters plus syndrome
retinitis pigmentosa
Usher syndrome
X-linked juvenile retinoschisis
MedlinePlus related topics:
Usher Syndrome
U.S. FDA Resources
Further study details as provided by Oxford BioMedica:
Primary Outcome Measures:
- The incidence of adverse events [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]The number and percentage of patients with treatment emergent adverse events.
Secondary Outcome Measures:
- To determine delay in retinal degeneration. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]Changes in function relative to the contralateral eye utilizing retinal analytical techniques.
| Estimated Enrollment: | 18 |
| Study Start Date: | January 2012 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | December 2014 (Final data collection date for primary outcome measure) |
Intervention Details:
Detailed Description:
-
Genetic: UshStat
Single dose, 3 ascending dose cohorts.
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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| 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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01505062
Contacts
| Contact: Richard G Weleber, MD | 503 494 3795 |
Locations
| United States, Oregon | |
| Casey Eye Institute | Recruiting |
| Portland, Oregon, United States, 97239-4197 | |
| France | |
| Central Hospitalier Nationale d'Opthalmolgie des Quinze-Vingts | Not yet recruiting |
| Paris, France, 75571 | |
Sponsors and Collaborators
Oxford BioMedica
Investigators
| Principal Investigator: | Richard G Weleber, MD | Casey Eye Institute, Portland, Oregon |
| Principal Investigator: | Jose-Alain Sahel, MD | Hospital Nationale des Quinze-Vingt, Paris, France |
More Information
No publications provided
| Responsible Party: | Oxford BioMedica |
| ClinicalTrials.gov Identifier: | NCT01505062 History of Changes |
| Other Study ID Numbers: | US1/001/10 |
| Study First Received: | January 4, 2012 |
| Last Updated: | May 10, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Oxford BioMedica:
|
Usher Syndrome Retinitis Pigmentosa Usher Syndrome associated Retinitis Pigmentosa |
Additional relevant MeSH terms:
|
Retinitis Retinitis Pigmentosa Usher Syndromes Retinal Diseases Eye Diseases Eye Diseases, Hereditary Retinal Dystrophies Retinal Degeneration Genetic Diseases, Inborn 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 Abnormalities, Multiple Congenital Abnormalities Signs and Symptoms |
ClinicalTrials.gov processed this record on June 17, 2013