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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2024
• Main ID: NCT04120883
• Date of registration: 08/10/2019
• Prospective Registration: Yes
• Primary sponsor: University of Michigan
• Public title: Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)
• Scientific title: Oral Hydroxychloroquine for Retinitis Pigmentosa Caused by P23H-RHO (Substitution of Proline to Histidine at Codon 23 of the Rhodopsin Protein)
• Date of first enrolment: February 25, 2020
• Target sample size: 12
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04120883
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Single (Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: David Zacks, MD
• Affiliation: University of Michigan

• Name: Callie Gordon

• Name: Callie Gordon

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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 Criteria:

- 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Drug: Hydroxychloroquine higher dose

Drug: Hydroxychloroquine lower dose

Primary Outcome(s)

Change in Ellipsoid zone area measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) [Time Frame: screening up to 18 months]

Change in Retinal sensitivity (decibels) measured by scotopic and mesopic microperimetry [Time Frame: screening up to 18 months]

Secondary ID(s)

HUM00164470

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cures Within Reach