NCT05329649Active, not recruitingPhase 3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
Source: ClinicalTrials.gov · Last updated 1 Jul 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 13 (actual)
- First submitted
- 7 Apr 2022
- Start date
- 2 May 2022
- Primary completion date
- 6 Jun 2027
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Diseases
- Sickle cell disease
- NCT number
- NCT05329649
- EU CT number
- 2024-513978-22-00
- EudraCT number
- 2021-002173-26
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 13
- From 2 May 2022 to the primary completion date, 6 Jun 2027
- 7 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior hematopoietic stem cell transplant (HSCT).
Other illness
- Clinically significant and active bacterial, viral, fungal, or parasitic infection
- Other protocol defined Inclusion/Exclusion criteria may apply.
Lab values
- A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor
Who the study is looking for
- 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
Age in the registry: 2 years to 11 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Universitaetsklinikum Duesseldorf AöR Coordinator of this site? Confirm its status | Duesseldorf | Germany | Recruiting |
| St.Mary's Hospital - Haematology Dept Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Recruiting |
| Levine Children's Hospital - Hematology Coordinator of this site? Confirm its status | Charlotte | United States | Unknown status |
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| TriStar Medical Group Children's Specialists - Pediatric Oncology Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| The Children's Hospital of Philadelphia - Hematology Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
Related publications
- Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.N Engl J Med · 2026 · PMID 42274009
This page is information, not medical advice. Discuss any trial with your doctor.