PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED ALTU-135 IN PATIENT STUD

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。列出的机构是 研究中心，而研究中心不一定是研究者所在的机构。 这是一项随机、双盲、安慰剂对照临床试验，将评估ALTU-135治疗具有外分泌PI的CF患者的疗效和安全性。 本研究将作为一项多中心研究进行，参与研究中心具有治疗CF患者的专业知识。 研究分为五个阶段： 1.筛选期，在此期间将评估参与研究的资格。 2.住院、无酶基线-B期，在此期间将测量每例患者的外分泌胰腺功能。 3.开放标签治疗期，在此期间所有患者将接受ALTU-135。 4.住院双盲DB治疗期，在此期间，一半患者将随机退出治疗并接受安慰剂，每例患者？的胰腺外分泌功能将进行第二次测量。 5.第二个开放标签R治疗期，在此期间，所有患者将恢复开放标签研究药物治疗。 治疗的总持续时间将不超过44天，研究的总持续时间（不包括筛选访视）将不超过64天。 患者将被要求住院两次，非连续时间约为6至7天。

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. This is a randomized, double-blind, placebo-controlled clinical trial which will evaluate the efficacy and safety of ALTU-135 treatment in CF patients with exocrine PI. It will be conducted as a multicenter study with participating sites having expertise in treating patients with CF. The study is divided into five phases: 1. Screening Period during which eligibility for study participation will be assessed. 2. Inpatient, off-enzyme Baseline -B Period during which each patients exocrine pancreatic function will be measured. 3. Open-Label Treatment Period during which all patients will receive ALTU-135. 4. Inpatient, Double-Blind DB Treatment Period during which half of the patients will be randomly withdrawn from treatment and receive placebo, and each patient?s exocrine pancreatic function will be measured for a second time. 5. Second Open-Label R Treatment Period during which all patients will resume treatment with open-label study drug. The total duration of treatment will not exceed 44 days and the total duration of the study excluding the Screening Visit will not exceed 64 days. Patients will be required to be inpatients for two, non-consecutive periods of approximately six to seven days.