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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02633943




Registration number
NCT02633943
Ethics application status
Date submitted
30/11/2015
Date registered
17/12/2015
Date last updated
31/01/2019

Titles & IDs
Public title
Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy
Scientific title
Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Secondary ID [1] 0 0
LTF-303
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Beta-Thalassemia 0 0
Sickle Cell Disease 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Safety and efficacy assessments

Subjects with hemoglobinopathies - Subjects treated with ex vivo gene therapy product in a bluebird bio-sponsored clinical trial who agree to participate in this study


Other interventions: Safety and efficacy assessments
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study
Timepoint [1] 0 0
15 years post-drug product infusion
Primary outcome [2] 0 0
All adverse events (AEs) related to drug product
Timepoint [2] 0 0
15 years post-drug product infusion
Primary outcome [3] 0 0
All serious adverse events (SAEs)
Timepoint [3] 0 0
15 years post-drug product infusion
Primary outcome [4] 0 0
Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN)
Timepoint [4] 0 0
15 years post-drug product infusion
Primary outcome [5] 0 0
Monitoring of ßA-T87Q-globin
Timepoint [5] 0 0
15 years post-drug product infusion
Primary outcome [6] 0 0
Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with ß-thalassemia
Timepoint [6] 0 0
15 years post-drug product infusion
Primary outcome [7] 0 0
Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with ß-thalassemia
Timepoint [7] 0 0
15 years post-drug product infusion
Primary outcome [8] 0 0
Frequency of AEs from subjects with severe sickle cell disease including acute chest syndrome (ACS), severe vaso-occlusive episodes (VOC), and stroke or ischemic attacks
Timepoint [8] 0 0
15 years post-drug product infusion

Eligibility
Key inclusion criteria
- Provision of written informed consent for this study by subjects, or as applicable,
subject's parent(s)/legal guardian(s)

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

- Able to comply with study requirements
Minimum age
No limit
Maximum age
50 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Met the VCN discontinuation criterion in the parent study

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
South Carolina
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
Thailand
State/province [7] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
bluebird bio
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a multi-center, long-term safety and efficacy follow-up study for subjects with
hemoglobinopathies (ß-thalassemia or severe 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.
Trial website
https://clinicaltrials.gov/show/NCT02633943
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mohammed Asmal, MD, PhD
Address 0 0
bluebird bio, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02633943