Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-02

Study Completion Date

2026-05-31

Brief Summary

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This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

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Detailed Description

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Conditions

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Sickle Cell Disease Hydroxyurea Failure Hydroxyurea Intolerance Hemoglobinopathies Hematological Diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CTX001

CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.

Group Type EXPERIMENTAL

CTX001

Intervention Type BIOLOGICAL

Administered by intravenous infusion following myeloablative conditioning with busulfan.

Interventions

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CTX001

Administered by intravenous infusion following myeloablative conditioning with busulfan.

Intervention Type BIOLOGICAL

Other Intervention Names

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Exagamglogene autotemcel Exa-cel

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of severe SCD as defined by:
* Documented SCD genotypes
* History of at least two severe VOCs events per year for the previous two years prior to enrollment
* Hydroxyurea (HU) failure unless HU intolerant
* Eligible for autologous stem cell transplant as per investigators judgment

Exclusion Criteria

* A willing and healthy 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

Minimum Eligible Age

2 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No