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临床试验/NCT04853576
NCT04853576进行中（未招募）1 期

A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

Editas Medicine, Inc.48 个研究点 分布在 2 个国家目标入组 45 人开始时间: 2021年5月4日最近更新:
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适应症

试验速览

阶段
1 期
状态
进行中（未招募）
入组人数
45
试验地点
48
主要终点
Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)
研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).

详细描述

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive.

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 50 Years（Child, Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Diagnosis of severe sickle cell disease as defined by:
  • •Documented SCD genotype (βS/βS, βS/β0, βS/β+, or others) and
  • •History of at least two severe vaso-occlusive events per year requiring medical attention despite hydroxyurea or other supportive care measures in the two year-period prior to provision of informed consent or assent, as applicable
  • •Karnofsky (for subjects >16 years of age) or Lansky (for subjects ≤ 16 years of age) Performance Status ≥ 80%
  • •Normal transcranial doppler velocity in subjects 16 years of age or younger

排除标准

  • •Available 10/10 HLA-matched related donor
  • •Prior HSCT or contraindications to autologous HSCT
  • •Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells (HSCs) and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients
  • •Unable to receive red blood cell (RBC) transfusion for any reason
  • •Unable or unwilling to comply with standard of care changes in background medical treatment in preparation of, during, or following HSCT, including and not limited to discontinuation of hydroxyurea, voxelotor, crizanlizumab, or L-glutamine
  • •Any history of severe cerebral vasculopathy
  • •Inadequate end organ function
  • •Advanced liver disease
  • •Any prior or current malignancy or immunodeficiency disorder
  • •Immediate family member with a known or suspected Familial Cancer Syndrome
  • •Clinically significant and active bacterial, viral, fungal, or parasitic infection
  • •Other protocol defined inclusion/exclusion criteria may apply

结局指标

主要结局

Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)

时间窗: from Month 6 through Month 18 post EDIT-301 infusion

次要结局

  • Proportion of subjects with 90% reduction in annualized rate of severe VOE compared to pre-treatment period(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects with 75% reduction in annualized rate of severe VOE compared to pre-treatment period(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects achieving complete resolution of VOEs(from Month 6 through Month 18 post EDIT-301 infusion)
  • Proportion of subjects with 50% reduction in annualized rate of severe VOE compared to pre-treatment period(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Difference (pre-treatment vs. post-treatment) in annualized rates of severe VOEs(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Difference (pre-treatment vs. post-treatment) in annualized rate of hospitalization for severe VOEs(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects with sustained HbF ≥ 20% (HbF/Hb) compared with baseline(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects with mean HbF ≥ 30% (HbF/Hb) compared with baseline(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects with mean total Hb ≥ 10 g/dL compared with baseline(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Proportion of subjects with mean total Hb increase from baseline of ≥ 2 g/dL(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Difference (pre-treatment versus post-treatment) in annualized number of units of pRBC transfused for SCD-related indications(starting from 6 months up to 2 years post EDIT-301 infusion)
  • Change from baseline in HbF concentration (g/dL)(up to 2 years post EDIT-301 infusion)
  • Change from baseline in total Hb concentration (g/dL)(up to 2 years post EDIT-301 infusion)
  • Change from baseline in markers of hemolysis (absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, haptoglobin)(up to 2 years post EDIT-301 infusion)
  • Time to neutrophil engraftment (the first day in which 3 consecutive absolute neutrophil count (ANC) ≥ 0.5 x 109/L laboratory values obtained on different days)(up to 24 months after EDIT-301 infusion)
  • Time to platelet engraftment (the first day in which 3 consecutive platelets ≥ 50 x 109/L laboratory values obtained for at least 7 days following the last platelet transfusion and 10 days following any administration of thrombopoietin (TPO) mimetics)(up to 24 months after EDIT-301 infusion)
  • Frequency and severity of adverse events (AEs)(up to 24 months post EDIT-301 infusion)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (48)

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