Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

Phase 2 Completed 48 enrolled The University of Texas Medical Branch, Galveston

RandomizedParallel-groupOpen-labelTreatment

Summary

Porphyria cutanea tarda (PCT) is an iron-related disorder that responds to treatment by phlebotomy or low-dose hydroxychloroquine, but comparative data on these treatments are limited. The hypothesis is that hydroxychloroquine is noninferior to phlebotomy in terms of time to remission. Patients with well documented PCT are assigned to treatment by randomization if specific criteria are met. All patients are followed until remission - defined as achieving a normal plasma porphyrin concentration.

Timeline

Start: 2006-03-21
Primary completion: 2021-07-06
Completion: 2021-07-06

Outcome

Missed primary endpoint

paper a difference that was not significant (log-rank, P = .06 and P = .95, respectively). PMID 22985607 ↗

paper The sample size was insufficient to confirm noninferiority of hydroxychloroquine treatment PMID 22985607 ↗

Publications

Singal AK, Kormos-Hallberg C, Lee C, Sadagoparamanujam VM, Grady JJ, Freeman DH Jr, Anderson KE. Low-dose hydroxychloroquine is as effective as phlebotomy in treatment of patients with porphyria cutanea tarda. Clin Gastroenterol Hepatol. 2012 Dec;10(12):1402-9. doi: 10.1016/j.cgh.2012.08.038. Epub 2012 Sep 14.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Hydroxychloroquine	Small molecule	100 mg	Oral