Status:
TERMINATED
Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)
Lead Sponsor:
University of Michigan
Collaborating Sponsors:
Cures Within Reach
Conditions:
Retinitis Pigmentosa
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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 tha...
Eligibility Criteria
Inclusion
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Signed and dated informed consent form
- Early Treatment Diabetic Retinopathy Study Best Corrected Visual Acuity (ETDRS BCVA) of 20 letters (approximately 20/400 Snellen) or better in at least one eye
- Clinical diagnosis of autosomal dominant retinitis pigmentosa
- Confirmed to have one copy of the P23H-RHO pathogenic variant by genetic testing at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
- Clarity of ocular media and adequate pupillary dilation to allow for adequate clinical ocular examination and retinal imaging
- Ability to perform testing required by the study as determined by the investigator
- Ability to take oral medication (medication tablets must be swallowed whole) and be willing to adhere to the daily medication regimen
- For females of reproductive potential: use of highly effective contraception beginning no later than 1 week after the first screening visit, and agreement to use such a method during study participation and through the end of the washout period (6 months after the end of HCQ administration)
- Agreement to adhere to Lifestyle Considerations throughout study duration (take the study drug with meals, avoid taking over-the-counter antacids or kaolin-containing products 4 hours before or after taking the study drug)
Exclusion
- Use of any other drugs which are known to prolong the QT interval
- Concurrent use of any of the following drugs, if the drug cannot be discontinued or substituted: digoxin, antiepileptic medications, cimetidine, methotrexate, cyclosporine, praziquantel, ampicillin
- Current or previous use of tamoxifen
- Pregnancy or lactation
- Known allergy or hypersensitivity to hydroxychloroquine or any other 4-aminoquinoline drugs (chloroquine, amodiaquine, mefloquine, quinacrine, etc.), or known history of glucose-6-phosphate dehydrogenase deficiency
- Treatment with another investigational medical intervention for retinitis pigmentosa within 3 months, or any ever previous treatment with an investigational surgical intervention
- Any pre-existing cardiac, renal, hepatic, or hematologic disease, any prior history of psoriasis or porphyria, or any alcoholism
- Abnormal screening laboratory values including aspartate transaminase (AST) or alanine transaminase (ALT) \> 2.0 x upper limit of normal, subnormal glomerular filtration rate (\< 90 mL/min/1.73m2) or abnormal complete blood count attributable to underlying hematologic disease such as malignancy, aplastic anemia, agranulocytosis, leukopenia, or thrombocytopenia.
Key Trial Info
Start Date :
February 25 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 5 2024
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT04120883
Start Date
February 25 2020
End Date
August 5 2024
Last Update
October 26 2024
Active Locations (1)
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1
University of Michigan
Ann Arbor, Michigan, United States, 48109