← Back to all trials
Enrolling By Invitation
NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001
Conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Sex: All
Ages: 2 Years – N/A
Healthy volunteers: No
Phase: PHASE3
Enrollment: 160
Sponsor: Vertex Pharmaceuticals Incorporated
Location: Lucile Packard Children's Hospital Palo Alto California
Summary
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).
Eligibility Criteria
Inclusion Criteria:
* Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
* Participants must have received CTX001 infusion in a parent study
Exclusion Criteria:
* There are no exclusion criteria
Source: ClinicalTrials.gov (NCT04208529). StuddyBuddy aggregates publicly available trial information.
