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: 7719188
• 负责人: KARL ELMO ANDERSON
• 金额: $ 28.45万
• 依托单位: UNIVERSITY OF TEXAS MED BR GALVESTON
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-04-01 至 2009-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7719188
• 关键词: 4-aminoquinoline; Alcohol consumption; Antimalarials; Chloroquine; 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; Smoking; Source; Treatment Protocols; United States National Institutes of Health; Urine; Uroporphyrinogen Decarboxylase; Venous blood sampling; Week; 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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 孢子菌tarda（PCT）是最常见的人类卟啉症，对治疗最有反应。 两种截然不同的治疗方法被认为有效。 重复的静脉切开术是最广泛使用的，但缺点包括不适，不便和费用。 我们假设4-氨基喹啉抗菌药物的低剂量方案（羟基氯喹或氯喹）更方便且具有成本效益，但并未被广泛用作第一线治疗。 提出了一项频繁和详细评估功效评估的随机研究，以比较这些治疗方法。 有80例有据可查的PCT的患者将参与这一前瞻性，随机，未盲的2-3期非劣效性研究，比较了通过静脉切开术与低剂量羟基氯喹的治疗的治疗。 患者的特征是PCT的已知危险因素，包括使用乙醇，吸烟，丙型肝炎，HIV感染，使用雌激素，HFE突变和常染色体显性遗传，即由于UROD突变引起的部分尿中缺血蛋白原脱羧酶（UROD）的部分缺乏（如家族性PCT中）。 血浆和尿卟啉浓度将以2周的间隔测量6个月。 这项研究可能会证明对低剂量羟基氯喹的更一般使用是合理的，并最终导致PCT作为批准的指示。 对这种方法的更大接受将降低医疗保健成本，并增加治疗PCT的便利性。