Clinical trial NCT04628585: Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Sickle Cell Disease
Keywords: Hematopoietic Stem Cells
Lead sponsor: bluebird bio
Sponsor class: INDUSTRY
First posted: 2020-11-13
Enrollment: 85
Sex: ALL
Minimum age (years): 2
Maximum age (years): 53
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with sickle cell disease treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
timeFrame: Through 15 years post-drug product infusion
measure: Number of subjects with new or worsening hematologic disorders
timeFrame: Through 15 years post-drug product infusion
measure: Number of subjects with new or worsening neurologic disorders
timeFrame: Through 15 years post-drug product infusion
measure: Number of subjects with malignancies
timeFrame: Through 15 years post-drug product infusion
Secondary outcomes:
measure: Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Annualized number of severe VOEs over time through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Annualized number of VOEs over time through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Change from parent study baseline in annualized number of severe VOEs over time through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Assessment of total Hb over time post-drug product infusion through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Assessment of non-transfused total Hb over time post-drug product infusion through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15
timeFrame: Through 15 years post-drug product infusion
measure: Change from parent study baseline through Year 15 in hemolysis markers
timeFrame: Through 15 years post-drug product infusion
measure: Change from parent study baseline through Year 15 in markers of iron stores
timeFrame: 15 years post-drug product infusion
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Bethesda
country: United States
facility: Warren Grant Magnuson Clinical Center
state: Maryland
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Children's Hospital
state: Minnesota
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Paris
country: France
facility: Hospital Necker
Study officials:
affiliation: bluebird bio, Inc.
name: Anjulika Chawla, MD
role: STUDY_DIRECTOR
References:
citation: Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24.