Overseen byStephen Tsang, MD, PhD
Age: Any Age
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: N/A
Recruiting
Sponsor: Columbia University
No Placebo Group
Trial Summary
What is the purpose of this trial?Mutations in the rod-expressed gene, cyclic nucleotide-gated channel beta subunit (CNGB1) and associated inborn errors in metabolism are causes of retinal disease that causes progressive loss of vision. Retinitis pigmentosa (RP) is a major cause of untreatable blindness associated with CNGB1 (CNGB1-RP). RP involves the death of photoreceptor cells that can be caused by mutations in a number of different genes. Treatment by gene therapy could prevent blindness in cases of inherited retinal dystrophies including RP. In the future RP due to mutations in CNGB1 may be treatable by gene therapy since this form of photoreceptor degeneration involves a slow loss of rod photoreceptor cells. This provides a wide window of opportunity for the identification of patients and initiation of treatment. Our efforts are directed toward developing gene therapy as a treatment. To this end, our objective is to better understand the disease process of CNGB1-RP and other allied inherited disorders so that we can develop clinical tests to measure the outcomes of treatment.
Eligibility Criteria
Treatment Details
Find a clinic near you
Research locations nearbySelect from list below to view details:
Wills Eye HospitalPhiladelphia, PA
Dr. Stephen H. TsangNew York, NY
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Who is running the clinical trial?
Columbia UniversityLead Sponsor
Michigan State UniversityCollaborator
La Fondation Voir et EntendreCollaborator
Moorfields Eye Hospital NHS Foundation TrustCollaborator
Universität TübingenCollaborator
Wills EyeCollaborator
Ludwig-Maximilians - University of MunichCollaborator