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NCT04639635 · ClinicalTrials.gov registry record
CNGB1 and Allied Disorders
A clinical trial of Retinitis Pigmentosa Associated With CNGB1 Mutations, sponsored by Columbia University.
- Recruiting
- Registry status
- 20
- Enrollment target
- 6
- Study locations
NCT04639635 is a study of Retinitis Pigmentosa Associated With CNGB1 Mutations that is actively recruiting participants, run by Columbia University. The registered enrollment target is 20 participants. The trial reports 6 study locations across 4 states.
The verdict
NCT04639635, a study of Retinitis Pigmentosa Associated With CNGB1 Mutations, is actively recruiting participants, sponsored by Columbia University.
- RECRUITING
- Registry status
- 20 participants
- Enrollment target
- 6
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- OTHER No intervention, this is a natural history progression study
Study Locations (6)
Other
- Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts - Paris
- Eberhard Karls University Tubingen - Tübingen
- Moorfields Eye Hospital NHS Foundation Trust - London
New York
- Dr. Stephen H. Tsang - New York
Pennsylvania
- Wills Eye Hospital - Philadelphia
Bavaria
- Klinikum der Universität München University Eye Hospital, Ludwig-Maximilians-University (LMU) Munich - München
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2019-03-14 |
| Est. Completion | 2026-02 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04639635
The ClinicalTrials.gov registry entry for NCT04639635 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Columbia University, which has 958 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Retinitis Pigmentosa Associated With CNGB1 Mutations appearing as the primary indexed condition, and to 1 intervention - of which No intervention, this is a natural history progression study is the first listed.
NCT04639635 reports 6 study locations spanning 4 distinct geographic areas - top geographies include Other, New York, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT04639635 about?
NCT04639635 is a clinical study titled "CNGB1 and Allied Disorders". 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-...
What is the current status of trial NCT04639635?
This trial is currently recruiting. The enrollment target is 20 participants. The study started on 2019-03-14. Estimated completion is 2026-02.
What conditions does trial NCT04639635 study?
This clinical trial studies the following conditions: Retinitis Pigmentosa Associated With CNGB1 Mutations.
What interventions are being tested in trial NCT04639635?
The interventions under investigation include: No intervention, this is a natural history progression study (OTHER).
Who is sponsoring clinical trial NCT04639635?
This trial is sponsored by Columbia University, which has 958 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04639635 being conducted?
This trial has 6 study locations across New York, Pennsylvania, Bavaria. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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