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

Phase 3

Recruiting

Conditions: Sickle-cell disease

Registration Number: 2024-513901-30-00

Lead Sponsor: Bluebird Bio Inc.

Brief Summary: - Evaluate long-term safety of treatment with lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD)

- Evaluate long-term efficacy of treatment with bb1111 in subjects with SCD

Detailed Description: Not available

Recruitment & Eligibility

Status: Ongoing, recruiting

Sex: Not specified

Target Recruitment: 3

Inclusion Criteria

Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)

Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study

Exclusion Criteria

There are no exclusion criteria for this study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)		The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
The number of subjects with new or worsening hematologic disorders		The number of subjects with new or worsening hematologic disorders
The number of subjects with new or worsening neurologic disorders		The number of subjects with new or worsening neurologic disorders
The number of subjects with malignancies		The number of subjects with malignancies

Secondary Outcome Measures

Name	Time	Method
Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15		Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15		The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
Annualized number of severe VOEs over time through Year 15		Annualized number of severe VOEs over time through Year 15
Annualized number of VOEs over time through Year 15		Annualized number of VOEs over time through Year 15
Change from parent study baseline in annualized number of severe VOEs over time through Year 15		Change from parent study baseline in annualized number of severe VOEs over time through Year 15
Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb		Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb
Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase		Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase
Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content		Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content

Trial Locations

Locations (1): Hopital Necker Enfants Malades
🇫🇷
Paris, France
Hopital Necker Enfants Malades
🇫🇷Paris, France
Marina Cavazzana
Site contact
+33144495068
m.cavazzana@aphp.fr