A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
试验速览
- 阶段
- 4 期
- 状态
- Enrolling By Invitation
- 入组人数
- 59
- 试验地点
- 23
- 主要终点
- Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
研究概览
简要总结
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
详细描述
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator.
- •For women of childbearing potential (WOCBP), must be abstinent of sexual activities that may result in pregnancy 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 providing informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method.
- •Written informed consent/assent from the participant (or their legally authorized representative, parent[s], or legal guardian) before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study.
- •Agrees not to participate in other interventional clinical studies, including other studies of mitapivat, during participation in this study.
排除标准
- •Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB).
- •Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are:
- •Participants who are institutionalized by regulatory or court order.
- •Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).
研究组 & 干预措施
Mitapivat
Participants will continue to receive mitapivat at the same dose regimen they were receiving at the final visit of their antecedent study.
干预措施: Mitapivat (Drug)
结局指标
主要结局
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
时间窗: Up to approximately 6.2 years
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug
时间窗: Up to approximately 6.2 years
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Up to approximately 6.2 years
Percentage of Participants With AEs and SAEs, Graded by Severity
时间窗: Up to approximately 6.2 years
The severity of all AEs will be graded by the Investigator according to Version 4.03 of the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) on a 5-point severity scale (Grade 1 through Grade 5) where Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening and Grade 5 is fatal.
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study
时间窗: Not Applicable - Study Completion
次要结局
- Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Up to approximately 6.2 years)
- Percentage of Participants With AEs and SAEs, Graded by Severity(Up to approximately 6.2 years)
- Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug(Up to approximately 6.2 years)
- Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug(Up to approximately 6.2 years)