Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-004266-18-FR

Enrollment

Registered

2020-04-27

Start date

2020-09-03

Completion date

Unknown

Last updated

2024-10-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Subjects with sickle-cell disease treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety and efficacy of the drug products. MedDRA version: 20.1 Level: LLT Classification code 10040648 Term: Sickle cell SC disease System Organ Class: 100000004850

Interventions

Product Name: LentiGlobin BB305 Drug Product (autologous CD34 cells transduced w/ BB305 lentiviral vector) Pharmaceutical Form: Dispersion for infusion Other descriptive name: AUTOLOGOUS CD34+ CELLS T

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Provision of written informed consent for this study by subject, or as applicable, subject’s parent(s)/ legal guardian(s) 2. Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study 3. Able to comply with study requirements Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 47 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: There are no exclusion criteria for this study.

Design outcomes

Primary

Measure	Time frame
Main Objective: • Evaluate long-term safety of the gene therapy drug product (i.e., the “drug product”) used in bluebird bio-sponsored clinical studies (i.e., the “parent studies”) in treated subjects with sickle cell disease (SCD) • Evaluate long-term efficacy of the drug product;Secondary Objective: Not applicable;Primary end point(s): Primary Efficacy Endpoint: • Non-transfused total hemoglobinover time Pharmacodynamic Endpoints: • Vector copy number (VCN) in peripheral blood over time post-drug product infusion • ßA-T87Q-globin expression in peripheral blood over time post-drug product infusion;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.	—

Measure

Time frame

Main Objective: • Evaluate long-term safety of the gene therapy drug product (i.e., the “drug product”) used in bluebird bio-sponsored clinical studies (i.e., the “parent studies”) in treated subjects with sickle cell disease (SCD) • Evaluate long-term efficacy of the drug product;Secondary Objective: Not applicable;Primary end point(s): Primary Efficacy Endpoint: • Non-transfused total hemoglobinover time Pharmacodynamic Endpoints: • Vector copy number (VCN) in peripheral blood over time post-drug product infusion • ßA-T87Q-globin expression in peripheral blood over time post-drug product infusion;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.

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Secondary

Measure	Time frame
Secondary end point(s): Secondary Safety Endpoint: • Overall survival • All drug product-related adverse events (AEs) through Year 15 post-drug product infusion • All serious adverse events (SAEs) through Year 15 post-drug product infusion (regardless of relatedness to drug product). • Serious or non-serious immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV) and new or worsening hematologic or neurologic disorders or malignancies • Incidence of insertional mutagenesis leading to leukemia Secondary Efficacy Endpoints: • Assessment of the following over time: - non-transfused total Hb =10g/dL - HbS percentage of non-transfused total Hb - HbS percentage of non-transfused total Hb =70%, =60%, =50% - HbAT87Q percentage of non-transfused total Hb - HbAT87Q percentage of non-transfused total Hb =30%, =40%, =50% - non-HbS percentage of non-transfused total Hb • Change from parent study baseline in hemolysis markers • Change from parent study baseline in markers of iron stores • Change from parent study baseline in markers of stress erythropoiesis/anemia • 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 • Change in SCD complications which may include but are not limited to: vaso-occlusive events (VOC, ACS, priapism, hepatic sequestration, splenic sequestration), new or worsening osteonecrosis, or severe leg ulcers; retinopathy, change from baseline in renal function, cardiac-pulmonary function;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.	—

Measure

Time frame

Secondary end point(s): Secondary Safety Endpoint: • Overall survival • All drug product-related adverse events (AEs) through Year 15 post-drug product infusion • All serious adverse events (SAEs) through Year 15 post-drug product infusion (regardless of relatedness to drug product). • Serious or non-serious immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV) and new or worsening hematologic or neurologic disorders or malignancies • Incidence of insertional mutagenesis leading to leukemia Secondary Efficacy Endpoints: • Assessment of the following over time: - non-transfused total Hb =10g/dL - HbS percentage of non-transfused total Hb - HbS percentage of non-transfused total Hb =70%, =60%, =50% - HbAT87Q percentage of non-transfused total Hb - HbAT87Q percentage of non-transfused total Hb =30%, =40%, =50% - non-HbS percentage of non-transfused total Hb • Change from parent study baseline in hemolysis markers • Change from parent study baseline in markers of iron stores • Change from parent study baseline in markers of stress erythropoiesis/anemia • 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 • Change in SCD complications which may include but are not limited to: vaso-occlusive events (VOC, ACS, priapism, hepatic sequestration, splenic sequestration), new or worsening osteonecrosis, or severe leg ulcers; retinopathy, change from baseline in renal function, cardiac-pulmonary function;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.

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Countries

France, United States

Contacts

Public ContactClinical Trial Information

bluebird bio, Inc.

clinicaltrials@bluebirdbio.com+1339 499 9300

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026