A Long-term Follow-up Study in Participants Who Received CTX001

Part of paid clinical trials in Palo Alto, California.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT04208529
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • CTX001 — BIOLOGICAL
    CTX001 infusion.

Study Details

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).

Key Dates

Start date
Jan 20, 2021
Status verified
Aug 2025
Primary completion
Sep 30, 2039
Completion
Sep 30, 2039

Study Design

Enrollment
160 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER

Arms

  • Experimental: CTX001
    All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151 and CTX001-161) after CTX001 infusion will be asked to participate in this long-term follow-up study.

Primary Outcome Measure

New malignancies [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]

Locations (9)

FacilityCityStateZIPSite coordinators
Lucile Packard Children's HospitalPalo AltoCalifornia94304-
Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyChicagoIllinois60611-
Herbert Irving Pavilion - HematologyNew YorkNew York10032-
New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew YorkNew York10032-
Levine Children's Hospital - HematologyCharlotteNorth Carolina28203-
The Children's Hospital of Philadelphia - HematologyPhiladelphiaPennsylvania19104-
St. Jude Children's Research HospitalMemphisTennessee38105-
TriStar Medical Group Children's Specialists - Pediatric OncologyNashvilleTennessee37203-
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalSan AntonioTexas78229-

Find similar trials in Palo Alto, CA

By condition
Sickle cell disease
By research site
Lucile Packard Children's Hospital· Palo Alto, CAAnn & Robert H. Lurie Children's Hospital of Chicago - Hematology· Chicago, ILHerbert Irving Pavilion - Hematology· New York, NYNew York Presbyterian Hospital - Morgan Stanley Children's Hospital· New York, NYLevine Children's Hospital - Hematology· Charlotte, NCThe Children's Hospital of Philadelphia - Hematology· Philadelphia, PA

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