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Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype

NCT05951205 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2026-03-25

No results posted yet for this study

Summary

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).

Conditions

Interventions

BIOLOGICAL

Exa-cel

Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2027-07-31
Primary Completion
2033-12-31
Completion
2033-12-31
FDA Drug
Yes

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05951205 on ClinicalTrials.gov