This Phase 2 randomized controlled trial will study the safety, tolerability, and efficacy of
Hydroxychloroquine in qualified patients with Alport syndrome. The trial will be open-label,
randomized, controlled and will enroll up to 50 patients.
Patients in the Phase 2 cohort will be randomized 1:1 to either Hydroxychloroquine Cohort or
Comparator Cohort.
All patients in the study will follow the same visit and assessment schedule. Following
randomization on Day 1, patients will be scheduled to be assessed during treatment at Weeks
4, 12, and 24. Patients will not receive study drug during a 24-week withdrawal period
between Weeks 25 and 48. Patients will also be scheduled to be assessed at an in person
follow up visit at Week 36, and 48.
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