Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04628585
• Other study ID #: LTF-307
• Secondary ID: 2019-004266-18
• Status: Enrolling by invitation
• Start date: October 21, 2020
• Est. completion date: January 2038

Study information

• Verified date: January 2024
• Source: bluebird bio
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 85
• Est. completion date: January 2038
• Est. primary completion date: January 2038
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 53 Years

Eligibility

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 sickle cell disease in a bluebird bio-sponsored clinical study

Exclusion Criteria:

• There are no exclusion criteria for this study

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Other:
• Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Locations

Country	Name	City	State
France	Hospital Necker	Paris
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Warren Grant Magnuson Clinical Center	Bethesda	Maryland
United States	University of Alabama	Birmingham	Alabama
United States	The University of North Carolina	Chapel Hill	North Carolina
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Duke University Medical Center	Durham	North Carolina
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	Baylor College of Medicine	Houston	Texas
United States	Cohen Children's Medical Center	New Hyde Park	New York
United States	Columbia University Medical Center	New York	New York
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
bluebird bio

Countries where clinical trial is conducted

United States,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)		Through 15 years post-drug product infusion
Primary	Number of subjects with new or worsening hematologic disorders		Through 15 years post-drug product infusion
Primary	Number of subjects with new or worsening neurologic disorders		Through 15 years post-drug product infusion
Primary	Number of subjects with malignancies		Through 15 years post-drug product infusion
Secondary	Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15		Through 15 years post-drug product infusion
Secondary	Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15		Through 15 years post-drug product infusion
Secondary	Annualized number of severe VOEs over time through Year 15		Through 15 years post-drug product infusion
Secondary	Annualized number of VOEs over time through Year 15		Through 15 years post-drug product infusion
Secondary	Change from parent study baseline in annualized number of severe VOEs over time through Year 15		Through 15 years post-drug product infusion
Secondary	Assessment of total Hb over time post-drug product infusion through Year 15		Through 15 years post-drug product infusion
Secondary	Assessment of non-transfused total Hb over time post-drug product infusion through Year 15	Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q	Through 15 years post-drug product infusion
Secondary	Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15	Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q	Through 15 years post-drug product infusion
Secondary	Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15	Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q	Through 15 years post-drug product infusion
Secondary	Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15	Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q. Non-HbS is the total g/dL of HbF + HbA2 + HbAT87Q	Through 15 years post-drug product infusion
Secondary	Change from parent study baseline through Year 15 in hemolysis markers	Change from parent study baseline through Year 15 in absolute reticulocyte count, percent reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase hemolysis markers	Through 15 years post-drug product infusion
Secondary	Change from parent study baseline through Year 15 in markers of iron stores	Change from parent study baseline through Year 15 in serum ferritin and liver iron content markers of iron stores	15 years post-drug product infusion

External Links

•   HGB-205 Clinicaltrials.gov
•   HGB-206 Clinicaltrials.gov
•   HGB-210 Clinicaltrials.gov