NCT05951205撤回3 期
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 12
- 主要终点
- Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months
研究概览
简要总结
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
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
- 12 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with documented βS/βC (HbSC) genotype
- •Participants must be eligible for autologous stem cell transplant as per investigator's judgment
排除标准
- •A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
- •Participants with prior hematopoietic stem cell transplant (HSCT)
- •Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Exa-cel
Experimental
Participants will receive a single infusion of exa-cel (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene) through a central venous catheter.
干预措施: Exa-cel (Biological)
结局指标
主要结局
Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months
时间窗: From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion
次要结局
- Duration of Severe VOC Free in Participants who Have Achieved VF12(From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion)
- Proportion of Participants With No Severe Vaso-Occlusive Crises (VOCs) for At least 12 Months (VF12)(From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion)
- Time to First Detectable Haptoglobin(Up to 24 Months After exa-cel Infusion)
- Time to Neutrophil Engraftment(Up to 24 months After exa-cel Infusion)
- Relative Reduction in Annualized Volume of RBC Transfusions(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in Haptoglobin Over Time(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in Pain Scale (11-point numerical rating scale (NRS)) Assessment Over Time In Adults (>=18 Years)(From Baseline Up To 24 Months After exa-cel Infusion)
- Proportion of Participants With Sustained HbF >= 20 % for At least 3, 6, or 12 months(From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion)
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(From Signing of Informed Consent up to 24 Months After exa-cel Infusion)
- Incidence of All-cause Mortality(From Signing of Informed Consent up to 24 Months After exa-cel Infusion)
- Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs(From Baseline up to 24 Months After exa-cel Infusion)
- Total Hemoglobin (Hb) Concentration Over Time(Up To 24 Months After exa-cel Infusion)
- Change in Lactate dehydrogenase (LDH) Over Time(From Baseline Up To 24 Months After exa-cel Infusion)
- Time to First Normalized LDH(Up to 24 Months After exa-cel Infusion)
- Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time(Up To 24 Months After exa-cel Infusion)
- Proportion of Participants With Neutrophil Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)(Within 42 Days After exa-cel Infusion)
- Incidence of Transplant-Related Mortality (TRM)(Within 12 Months After exa-cel Infusion)
- Proportion of Participants Free from Inpatient Hospitalization For Severe VOCs Sustained for At least 12 Months (HF12)(From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion)
- Relative Reduction in Annualized Rate of Severe VOCs(From Baseline up to 24 Months After exa-cel Infusion)
- Relative Reduction in Rate of Inpatient Hospitalizations for Severe VOCs(From Baseline up to 24 Months After exa-cel Infusion)
- HbF Concentration Over Time(Up To 24 Months After exa-cel Infusion)
- Change in Indirect Bilirubin Over Time(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Over Time In Adults (>=18 Years)(From Baseline Up To 24 Months After exa-cel Infusion)
- Time to Platelet Engraftment(Up to 24 months After exa-cel Infusion)
- Change In Reticulocyte Count Over Time(From Baseline Up To 24 Months After exa-cel Infusion)
- Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time(Up To 24 Months After exa-cel Infusion)
- Change in Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me)(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in Pain Scale (11-point NRS) Assessment Over Time In Adolescents (12 to <18 years of age)(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in Pediatric Quality of Life Inventory (PedsQL; self-report and parent proxy versions) Generic Core In Adolescents (12 to <18 years of age)(From Baseline Up To 24 Months After exa-cel Infusion)
- Change in PedsQL SCD module (self-report and parent proxy versions) In Adolescents (12 to <18 years of age)(From Baseline Up To 24 Months After exa-cel Infusion)
- Incidence of Transplant-Related Mortality (TRM)(Up to 100 Days After exa-cel Infusion)
研究者
相似试验
暂停
3 期
Phase 3 Efficacy and Safety Laboratory Classroom Study in Pediatrics (6-12) With ADHD Using CTx-1301ADHDADHD - Combined TypeAttention Deficit Hyper ActivityAttention Deficit Disorder With HyperactivityAttention Deficit Hyperactivity Disorder CombinedAttention-deficit HyperactivityAttention Deficit Hyperactivity DisorderNCT05924594Cingulate Therapeutics45
终止
2 期
Acceleration of Platelet Recovery Following Autologous Peripheral Blood Stem Cell Transplant (PBSC) in Hodgkin, Non-Hodgkin Lymphoma or Multiple Myeloma PatientsLymphoma, Non-HodgkinHodgkin DiseaseMultiple MyelomaNCT01121120Tarix Pharmaceuticals75
终止
2 期
ATX-101 in Advanced Dedifferentiated Liposarcoma and LeiomyosarcomaLeiomyosarcomaLiposarcomaNCT05116683Benjamin Izar4
终止
1 期
Safety Study of TXA127 to Elevate CD4+ T-Lymphocyte Counts in HIV-Infected Patients on Stable HAART TherapyHIV InfectionsNCT00757250US Biotest, Inc.13
招募中
3 期
A Study to Evaluate KarXT as a Treatment for Psychosis Associated With Alzheimer's Disease (ADEPT-4)Alzheimer DiseaseNCT06585787Karuna Therapeutics, Inc., a Bristol Myers Squibb company406