A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Anemia, Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 18 - 35 |
| Updated: | 1/20/2019 |
| Start Date: | November 27, 2018 |
| End Date: | May 2022 |
| Contact: | Medical Information |
| Email: | medicalinfo@vrtx.com |
| Phone: | 6173416777 |
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12
subjects 18 to 35 years of age 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.
subjects 18 to 35 years of age 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.
The study may be expanded to include up to 45 subjects.
Key Inclusion Criteria:
- Subjects ≥ 18 and ≤ 35 years of age
- Diagnosis of severe sickle cell disease as defined by:
- Documented βS/βS 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
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