BETIBEGLOGENE AUTOTEMCEL for Beta-thalassaemia | Transfusion-dependent beta-thalassaemia

Inclusion criteria

• {"criterion_text":"- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)"}
• {"criterion_text":"- Treated with drug product for therapy of transfusion-dependent β- thalassemia in a bluebird bio-sponsored clinical study"}

Exclusion criteria

• {"criterion_text":"- There are no exclusion criteria for this study."}

Primary endpoints

• {"endpoint_text":"- The number of subjects with malignancies","definition_or_measurement_approach":""}
• {"endpoint_text":"- The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)","definition_or_measurement_approach":""}
• {"endpoint_text":"- The number of subjects with new or worsening hematologic disorders","definition_or_measurement_approach":""}
• {"endpoint_text":"- The number of subjects with new or worsening neurologic disorders","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- βA-T87Q-globin expression in peripheral blood over time post-drug product infusion through last follow-up, including Year 5, Year 10, and Year 15","definition_or_measurement_approach":""}
• {"endpoint_text":"- Proportion of subjects who meet the definition of transfusion independence (TI), defined as a weighted average Hb ≥ 9 g/dL without any pRBC transfusions for a continuous period of ≥ 12 months at any time after drug product infusion in parent study and/or Study LTF-303","definition_or_measurement_approach":""}
• {"endpoint_text":"- Proportion of subjects who meet the definition of TI at yearly timepoints including Year 5, Year 10, and Year 15 post-drug product infusion, and at last follow-up","definition_or_measurement_approach":""}
• {"endpoint_text":"- Characterization of TI: Time from drug product infusion to achievement of TI (in parent study or Study LTF-303); Duration of TI; Weighted average Hb during T","definition_or_measurement_approach":""}
• {"endpoint_text":"- Characterization of transfusion reduction (TR): Reduction in annualized pRBC transfusion volume (mL/kg/year) from 6 months post-drug product infusion (parent study) through last followup of at least 50%, 60%, 75%, 90%, or 100% as compared to the annualized pRBC transfusion volume during the 2 years prior to parent study enrollment","definition_or_measurement_approach":""}
• {"endpoint_text":"- Characterization of transfusion reduction (TR):Annualized pRBC transfusion volume (mL/kg/year) and frequency (number/year) from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment","definition_or_measurement_approach":""}
• {"endpoint_text":"- Characterization of transfusion reduction (TR): Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Characterization of transfusion reduction (TR): Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up","definition_or_measurement_approach":""}
• {"endpoint_text":"- Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up as compared to the weighted average nadir Hb during the 2 years prior to parent study enrollment","definition_or_measurement_approach":""}
• {"endpoint_text":"- Unsupported total Hb levels over time through last follow-up, including Year 5, Year 10, and Year 15","definition_or_measurement_approach":""}
• {"endpoint_text":"- Unsupported total Hb levels ≥ 10 g/dL, ≥ 11 g/dL, ≥ 12 g/dL, ≥ 13 g/dL, and ≥ 14 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15","definition_or_measurement_approach":""}
• {"endpoint_text":"- Iron burden over time and change from parent study baseline in iron burden at yearly timepoints through last follow-up, as measured by: Liver iron content by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) as available ; Cardiac T2* by MRI as available ; Serum ferritin","definition_or_measurement_approach":"Measured by liver iron content by MRI/SQUID (if available); cardiac T2* by MRI (if available); serum ferritin"}
• {"endpoint_text":"- Chelation therapy use, including time from last use of chelation therapy to last follow-up and absence of chelation use for at least 6 months post-drug product infusion in parent study and/or Study LTF-303","definition_or_measurement_approach":""}
• {"endpoint_text":"- Therapeutic phlebotomy use, including annualized frequency","definition_or_measurement_approach":""}
• {"endpoint_text":"- Measures of dyserythropoiesis over time as compared to parent study baseline, assessed by the following parameters: Reticulocytes ; Nucleated RBCs","definition_or_measurement_approach":"Assessed by reticulocytes and nucleated RBCs"}
• {"endpoint_text":"- Health-related quality of life (HRQoL) over time as compared to parent study baseline, using the following validated tools as available and as appropriate: Pediatric Quality of Life Inventory (PedsQL) ; EuroQol-5D (EQ-5D; youth version, EQ-5D-Y) ; Short Form-36 (SF-36) v2 ; Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT)","definition_or_measurement_approach":"Assessed using validated HRQoL tools where available: PedsQL, EQ-5D/EQ-5D-Y, SF-36 v2, FACT-BMT"}

France

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

12-09-2024

Processing Time Days

27

Number Of Sites

2

Number Of Participants

9

Germany

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

17-09-2024

Processing Time Days

32

Number Of Sites

2

Number Of Participants

3

Greece

Earliest CTIS Part Ii Submission Date

10-10-2024

Latest Decision Or Authorization Date

13-11-2024

Processing Time Days

34

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

14-10-2024

Processing Time Days

59

Number Of Sites

1

Number Of Participants

10

Primary sponsor

Full Name

Genetix Biotherapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

sponsorDuties codes: [{"id":880019,"code":"5"}]

Name

PPD Development LP

Responsibilities

sponsorDuties codes: [{"id":880017,"code":"4"}]

Name

Primevigilance Zagreb d.o.o.

Responsibilities

sponsorDuties codes: [{"id":880020,"code":"8"}]

Name

Labconnect LLC

Responsibilities

sponsorDuties codes: [{"id":880025,"code":"4"}]

Name

Voisin Consulting CH SARL

Responsibilities

sponsorDuties codes: [{"id":880018,"code":"12"}]

Name

ProtaGene CGT GmbH

Responsibilities

Insertion site analysis

Name

Unisphere Travel Ltd. Inc.

Responsibilities

Patient Reimbursement Services for Travel and Stipends

Name

Multi-Regional Clinical Trials Center Of Brigham And Women's Hospital And Harvard

Responsibilities

sponsorDuties codes: [{"id":880021,"code":"4"}]

Name

Merative US LP

Responsibilities

sponsorDuties codes: [{"id":880024,"code":"7"}]

Name

Genezen Laboratories Inc.

Responsibilities

sponsorDuties codes: [{"id":880023,"code":"4"}]

Third parties

• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"sponsorDuties codes: [{\"id\":880019,\"code\":\"5\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"Insertion site analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"Patient Reimbursement Services for Travel and Stipends","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Multi-Regional Clinical Trials Center Of Brigham And Women's Hospital And Harvard","duties_or_roles":"sponsorDuties codes: [{\"id\":880021,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labconnect LLC","duties_or_roles":"sponsorDuties codes: [{\"id\":880025,\"code\":\"4\"}]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Croatia","full_name":"Primevigilance Zagreb d.o.o.","duties_or_roles":"sponsorDuties codes: [{\"id\":880020,\"code\":\"8\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: [{\"id\":880017,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Merative US LP","duties_or_roles":"sponsorDuties codes: [{\"id\":880024,\"code\":\"7\"}]","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Genezen Laboratories Inc.","duties_or_roles":"sponsorDuties codes: [{\"id\":880023,\"code\":\"4\"}]","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Switzerland","full_name":"Voisin Consulting CH SARL","duties_or_roles":"sponsorDuties codes: [{\"id\":880018,\"code\":\"12\"}]","organisation_type":"Pharmaceutical company"}