EUCTR2018-003459-39-IE进行中（未招募）1 期
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects with Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 116
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Have signed written informed consent prior to participating in this extension study;
- •2. Have completed either antecedent study AG348-C-006 or AG348-C-007 through the Part 2 Week 24 Visit;
- •3. Cohorts 2 and 3: Have demonstrated clinical benefit from AG-348 treatment in the antecedent study, in the opinion of the Investigator;
- •4. For women of reproductive potential:
- •a. In Cohort 1, have a negative local serum (human Chorionic gonadotropin [hCG] pregnancy test during screening of this extension study.
- •b. In Cohort 2 or 3, have a negative local urine pregnancy test during screening of this extension study.
- •5. For women of reproductive potential as well as men with partners who are women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of giving informed consent, during the study, and for 28 days following the last dose of study drug for women and 90 days following the last dose of study drug for men. A highly effective form of contraception is defined as combined (estrogen and progestin containing) hormonal contraceptives (oral, intravaginal, or transdermal) known to be associated with inhibition of ovulation; progestin-only hormonal contraceptives (oral, injectable, or implantable) known to be associated with inhibition of ovulation; intrauterine device; intrauterine hormone releasing system; bilateral tube occlusion; or vasectomized partner. The second form of contraception can include an acceptable barrier method, which includes male or female condoms with or without spermicide, and cervical cap, diaphragm, or sponge with spermicide. Women of reproductive potential using hormonal contraception as a highly
- •effective form of contraception must also utilize an acceptable barrier method while enrolled in the study and for at least 28 days after their
- •last dose of study drug.
- •6. Be willing and able to comply with study visits and procedures.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 116
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Have a significant medical condition (including clinically significant laboratory abnormality) that developed during his/her antecedent AG-348 study that confers an unacceptable risk to participating in this extension study, that could confound the interpretation of the study data, and/or that compromises the ability of the subject to complete study visits and procedures;
- •2. Are currently pregnant or breastfeeding;
- •3. Have a splenectomy scheduled during the study treatment period;
- •4. Meet the withdrawal criteria of his/her antecedent AG-348 study during screening of this extension study.
- •Withdrawal criteria of the antecedent AG-348 studies are as follows:
- •- Withdrawal of consent
- •- Development of an intercurrent medical condition that precludes further participation in the study
- •- Subject requires use of a prohibited concomitant medications
- •- Investigator decision
- •- Persistent nonadherence to protocol requirements
- •- Pregnancy
- •- Lost to follow-up
- •5. Are currently receiving medications that are strong inhibitors of CYP3A4, that have not been stopped for a duration of at least 5 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug; or strong inducers of CYP3A4 that have not been stopped for a duration of at least 28 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug on this extension study.
- •6. Have received anabolic steroids, including testosterone preparations, within 28 days prior to Screening/Day 1 of this extension study;
- •7. Have received hematopoietic stimulating agents (eg, erythropoietins, granulocyte colony stimulating factors, thrombopoietins) within 28 days prior to start of study drug on this extension study;
- •8. Have exposure to any investigational drug other than AG-348, device, or procedure within 3 months prior to Screening/Day 1 of this extension study.
研究者
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