A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Recruiting completed

Phase 2, Phase 3

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

ClinicalTrials.gov Identifier

NCT03745287

Eligibility criteria

Sex

Age

12 Years - 35 Years

Phase

2, 3

Key inclusion criteria

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

Key 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 inclusion/exclusion criteria may apply.

Trial details

Condition

Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies

Study duration

November 2018 - October 2024

Study type

Interventional

Intervention/Treatment

Biological : CTX001

Estimated enrollment

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Recruiting completed

Lucile Packard Children's Hospital of Stanford University

Palo Alto California 94304 United States