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Anti-malarial drug

Hydroxychloroquine for Retinitis Pigmentosa

Phase 1 & 2

Waitlist Available

Led By David Zacks, MD

Research Sponsored by University of Michigan

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed to have one copy of the P23H-RHO pathogenic variant by genetic testing at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory

Clinical diagnosis of autosomal dominant retinitis pigmentosa

Must not have

Use of any other drugs which are known to prolong the QT interval

Treatment with another investigational medical intervention for retinitis pigmentosa within 3 months, or any ever previous treatment with an investigational surgical intervention

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening up to 18 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether the drug hydroxychloroquine can help people with retinitis pigmentosa by altering the autophagy pathway in photoreceptors.

Who is the study for?

This trial is for individuals with autosomal dominant retinitis pigmentosa caused by P23H-RHO. Participants must have a certain level of visual acuity, agree to use effective contraception if applicable, and be able to swallow pills whole. They should not have used specific drugs that affect the retina or have allergies to hydroxychloroquine or similar medications.

What is being tested?

The study tests two doses of oral hydroxychloroquine (HCQ) over 12 months on people with retinitis pigmentosa to see if it can stop the progression of retinal degeneration. It involves six visits including one phone visit, where patients will undergo general exams, blood work, electrocardiograms, and special retina testing.

What are the potential side effects?

Potential side effects may include digestive issues, changes in blood counts leading to anemia or bleeding problems, eye toxicity affecting vision, muscle weakness or nerve pain. There's also a risk of heart-related side effects like abnormal heart rhythms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My genetic test shows I have the P23H-RHO variant.

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I have been diagnosed with a genetic form of retinitis pigmentosa.

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I can take pills as required and follow the medication schedule.

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I can swallow pills whole and will follow the daily medication plan.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not taking any medications known to affect heart rhythm.

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I haven't had any experimental treatments for retinitis pigmentosa in the last 3 months.

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I have no history of heart, kidney, liver, blood diseases, psoriasis, porphyria, or alcoholism.

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My blood tests show liver issues or kidney problems, or I have a blood disorder.

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I am not taking drugs like digoxin, antiepileptics, or methotrexate that can't be stopped.

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I am not allergic to hydroxychloroquine or similar medications, and I don't have a glucose-6-phosphate dehydrogenase deficiency.

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I have used tamoxifen before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening up to 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening up to 18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening up to 18 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in Ellipsoid zone area measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)

Change in Retinal sensitivity (decibels) measured by scotopic and mesopic microperimetry

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: HCQ treatment 2Experimental Treatment1 Intervention

In treatment arm 2, the dose of the study drug will be 5 mg/kg/day. The daily dose will not exceed 400 mg. In both groups, the dose will be rounded down to 100, 200, 300, or 400 mg/day.

Group II: HCQ treatment 1Experimental Treatment1 Intervention

In treatment arm 1, the dose of study drug will be 4 mg/kg/day. The daily dose will not exceed 400 mg. In both groups, the dose will be rounded down to 100, 200, 300, or 400 mg/day.

0 / 11Eligibility criteria met