A study to learn about the safety and efficacy of CTX001 (the study drug product) to treat severe sickle cell disease in pediatric subjects

Phase 1

Conditions: Severe Sickle Cell Disease (SCD)
MedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2021-002173-26-DE

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 15

Inclusion Criteria

• Subjects 2 through 11 years of age, inclusive, on the date of informed consent
• Diagnosis of severe sickle cell disease as defined by:
• Documented severe sickle cell disease genotype
• History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment
• Eligible for autologous stem cell transplant as per investigators judgment

Other protocol defined inclusion criteria may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• An available 10/10 human leukocyte antigen (HLA)-matched related donor
• Prior hematopoietic stem cell transplant (HSCT)
• Clinically significant and active bacterial, viral, fungal, or parasitic infection

Other protocol defined exclusion criteria may apply

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluate the efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001) in pediatric subjects with severe sickle cell disease ;Secondary Objective: • Evaluate the safety and tolerability of a single dose of CTX001<br>• Assess the effects of infusion of CTX001 on disease-specific events and clinical status<br>• Quantify gene editing efficiency;Primary end point(s): Proportion of subjects who do not have any severe VOCs for at least 12 consecutive months (VF12) after CTX001 infusion. The evaluation of VF12 starts 60 days after the last RBC transfusion for post-transplant support or SCD disease management.;Timepoint(s) of evaluation of this end point: Up to 24 months

Secondary Outcome Measures