An Open-Label Extension Study of Voxelotor Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 162
- 试验地点
- 20
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events (AEs)
研究概览
简要总结
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
详细描述
Open-label extension (OLE) study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Up to approximately 600 participants with sickle cell disease (SCD), will be enrolled at approximately 70 clinical sites globally. All participants will receive voxelotor once daily, administered orally as tablets, dispersible tablets, or powder for oral suspension formulation. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor clinical 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
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study.
- •Note: Participants who discontinued study drug due to an AE, but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.
- •Note: Participants who discontinued Study GBT440-032 as the result of an abnormal transcranial Doppler (TCD) flow velocity assessment (≥ 200 cm/sec) are eligible for treatment in this study.
- •- Participant has provided written consent/assent (for pediatric participants, both the consent of the participant's legal representative or legal guardian and the participant's assent [where applicable] must be obtained).
排除标准
- •Female participant who is breastfeeding or pregnant
- •Participant withdrew consent from a GBT-sponsored voxelotor clinical study
- •Known hypersensitivity to voxelotor or any other components of the study drug
- •Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1
研究组 & 干预措施
Voxelotor
All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a stick pack formulation (powder blend formulation packaged as stick packs). Participants aged ≥ 12 years and/or ≥ 40 kgs will receive a voxelotor dose of 1500 mg QD. Participants aged < 12 years and < 40 kgs will receive weight based dosing of voxelotor. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. Participants may receive study drug as long they continue to receive clinical benefit that outweighs risk as determined by the investigator and/or until the participant has access to voxelotor from an alternative source.
干预措施: Voxelotor (Drug)
结局指标
主要结局
Number of Participants With Treatment Emergent Adverse Events (AEs)
时间窗: From date of informed consent until 28 days after last dose of study drug (maximum up to 4.31 years)
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered to be drug related. A treatment emergent AE was defined as an AE with an onset date on or after the date of informed consent until 28 days after discontinuation of drug. AEs included both serious AEs (SAEs) and all non-SAEs. An SAE was an AE or suspected adverse reaction that, at any dose, in the view of the either the investigator or sponsor, resulted in any of the following outcomes: death, was life-threatening, resulted in persistent disability/incapacity; constituted a congenital anomaly/birth defect; was important medical event; required inpatient hospitalization or prolongation of existing hospitalization.
Number of Participants With SAEs
时间窗: From date of informed consent until 28 days after last dose of study drug (maximum up to 4.31 years)
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered to be drug related. An SAE was an AE or suspected adverse reaction that, at any dose, in the view of the either the investigator or sponsor, resulted in any of the following outcomes: death, was life-threatening, resulted in persistent disability/incapacity; constituted a congenital anomaly/birth defect; was important medical event; required inpatient hospitalization or prolongation of existing hospitalization.
Number of Participants With Sickle Cell Disease (SCD) Related TEAEs and SAEs
时间窗: From date of informed consent until 28 days after last dose of study drug (maximum up to 4.31 years)
SCD-related AEs were common complications associated with the study participant's SCD and were not considered to be related to voxelotor unless judged by the investigator to have worsened in severity and/or frequency or changed in nature during the study. SCD-related complications included the following: sickle cell anemia with crisis, acute chest syndrome (ACS), pneumonia, priapism, and osteonecrosis. A treatment emergent AE was defined as an AE with an onset date on or after the date of informed consent until 28 days after discontinuation of drug. An SAE was an AE or suspected adverse reaction that, at any dose, in the view of either the investigator or sponsor, resulted in any of the following outcomes: death, was life-threatening, resulted in persistent disability/incapacity; constituted a congenital anomaly/birth defect; was important medical event; required inpatient hospitalization or prolongation of existing hospitalization.
次要结局
未报告次要终点