drugset / Trial / NCT04120883

Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)

NCT04120883

Phase 1/2 Terminated 8 enrolled University of Michigan Cures Within Reach · collab
Non-randomizedSequentialSingle-blindTreatment

Summary

This research study is being done to learn what effect 12 months of treatment with oral hydroxychloroquine (HCQ) will have on the retina in people with retinitis pigmentosa (RP). The hypothesis is that treatment with HCQ is safe and tolerable in patients with autosomal dominant retinitis pigmentosa (adRP) caused by P23H-RHO, and may arrest progression of retinal degeneration by altering the autophagy pathway in photoreceptors. Participants that meet eligibility and agree to the study will be asked to take the study medication (HCQ) for 12 months and have evaluations for up to approximately 18 months from the baseline visit. There will be a total of 6 visits (1 is a phone visit) and will include general examinations, blood work, electrocardiograms, along with special testing of the retina.

Timeline

Start
2020-02-25
Primary completion
2024-08-05
Completion
2024-08-05

Drugs

EvaluationDrugModalityDoseRoute
Subject Hydroxychloroquine Small molecule 4 mg/kg Oral
Subject Hydroxychloroquine Small molecule 5 mg/kg Oral

Indications