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

NCT04208529

Enrolling By InvitationPhase 3

Sponsor: Vertex Pharmaceuticals Incorporated

Conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies

Interventions: CTX001

Countries: United States, Belgium, Canada, Germany, Italy, United Kingdom

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 overview

Sex: ALL

Age: 2 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (20)

Palo Alto, California, United States
Chicago, Illinois, United States
New York, New York, United States
New York, New York, United States
Charlotte, North Carolina, United States
Philadelphia, Pennsylvania, United States
Memphis, Tennessee, United States
Nashville, Tennessee, United States
San Antonio, Texas, United States
Brussels, Belgium
Toronto, Canada
Toronto, Canada
Vancouver, Canada
Düsseldorf, Germany
Klinik Für Kinder- Und Jugendmedizin, Germany
Regensburg, Germany
Rome, Italy
London, United Kingdom
London, United Kingdom
London, United Kingdom