CLINICAL TRIAL: HYDROXYCHLOROQUINE VS PHLEBOTOMY FOR PORPHYRIA CUTANEA TARDA

临床试验：羟氯喹与放血疗法治疗迟发性皮肤卟啉症

• 批准号: 7952153
• 负责人: KARL ELMO ANDERSON
• 金额: $ 5.95万
• 依托单位: UNIVERSITY OF TEXAS MED BR GALVESTON
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-04-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7952153
• 关键词: 4-aminoquinoline; Alcohol consumption; Antimalarials; Chloroquine; Clinical Research; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Disadvantaged; Dose; Enrollment; Estrogens; Funding; Grant; HIV Infections; Health Care Costs; Hepatitis C; Human; Hydroxychloroquine; Institution; Lead; Measures; Mutation; Patients; Phase; Plasma; Porphyria Cutanea Tarda; Porphyrias; Porphyrins; Randomized; Research; Research Personnel; Resources; Risk Factors; Smoke; Source; Treatment Protocols; United States National Institutes of Health; Urine; Uroporphyrinogen Decarboxylase; Venous blood sampling; cost; prospective

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Porphyria cutanea tarda (PCT) is the most common human porphyria and the most responsive to treatment. Two very different approaches to therapy are considered effective. Repeated phlebotomy is most widely used, but has disadvantages that include discomfort, inconvenience and expense. We hypothesize that a low-dose regimen of the 4-aminoquinoline antimalarial drugs, either hydroxychloroquine or chloroquine, is more convenient and cost-effective but is not widely used as first line therapy. A randomized study with frequent and detailed assessment of efficacy is proposed to compare these treatments. Eighty patients with well-documented PCT will be enrolled in this prospective, randomized, unblinded phase 2-3 noninferiority study comparing treatment by phlebotomy with treatment by low-dose hydroxychloroquine. Patients are characterized in terms of known risk factors for PCT, including ethanol use, smoking, hepatitis C, HIV infection, estrogen use, HFE mutations and autosomal dominant inheritance of a partial deficiency of uroporphyrinogen decarboxylase(UROD) due to UROD mutations (as in familial PCT). Plasma and urine porphyrin concentrations will be measured at 2-week intervals for 6 months. It is likely that this study will justify more general use of low-dose hydroxychloroquine and eventually lead to treatment of PCT as an approved indication. Greater acceptance of this approach will lower health care costs and increase the convenience of treating PCT.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 迟发性皮肤卟啉病（PCT）是最常见的人类卟啉病，也是对治疗最敏感的疾病。 两种截然不同的治疗方法被认为是有效的。 重复放血是最广泛使用的方法，但也有一些缺点，包括不适、不便和费用。 我们假设低剂量的4-氨基喹啉类抗疟药，无论是羟氯喹还是氯喹，都是更方便和成本效益更高的，但并没有被广泛用作一线治疗。 建议进行一项随机化研究，对这些治疗进行频繁和详细的疗效评估。80例记录良好的PCT患者将入组这项前瞻性、随机化、非盲的2-3期非劣效性研究，比较静脉切开术治疗与低剂量羟氯喹治疗。 患者的特征在于已知的PCT风险因素，包括乙醇使用、吸烟、丙型肝炎、HIV感染、雌激素使用、HFE突变和由于尿卟啉原脱羧酶（UROD）突变导致的UROD部分缺陷的常染色体显性遗传（如家族性PCT）。 将在6个月内每2周测量一次血浆和尿液卟啉浓度。 这项研究可能会证明更普遍使用低剂量羟氯喹是合理的，并最终导致PCT治疗作为批准的适应症。 更广泛地接受这种方法将降低医疗保健成本，并增加治疗PCT的便利性。