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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02633943




Registration number
NCT02633943
Ethics application status
Date submitted
30/11/2015
Date registered
17/12/2015
Date last updated
26/03/2024

Titles & IDs
Public title
Long-term Follow-up of Subjects With Transfusion-Dependent ß-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
Scientific title
Long-term Follow-up of Subjects With Transfusion-Dependent ß-Thalassemia (TDT) 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:
Transfusion-dependent Beta-Thalassemia 0 0
Condition category
Condition code
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 Transfusion-Dependent ß-Thalassemia - Subjects treated with ex vivo gene therapy product in an applicable bluebird bio-sponsored clinical trial who agree to participate in this long-term follow-up study


Other interventions: Safety and efficacy assessments
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term effects of autologous transplant
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The number of subjects with malignancies
Timepoint [1] 0 0
Up to 15 years post-drug product infusion
Primary outcome [2] 0 0
The number of subjects with immune-related AEs
Timepoint [2] 0 0
Up to 15 years post-drug product infusion
Primary outcome [3] 0 0
The number of subjects with new or worsening hematologic disorders
Timepoint [3] 0 0
Up to 15 years post-drug product infusion
Primary outcome [4] 0 0
The number of subjects with new or worsening neurologic disorders
Timepoint [4] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [1] 0 0
ßA-T87Q-globin expression
Timepoint [1] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [2] 0 0
Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI)
Timepoint [2] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [3] 0 0
Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints
Timepoint [3] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [4] 0 0
Time from drug product infusion to achievement of Transfusion Independence (in parent study or Study LTF-303)
Timepoint [4] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [5] 0 0
Duration of Transfusion Independence
Timepoint [5] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [6] 0 0
Weighted average Hb during Transfusion Independence
Timepoint [6] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [7] 0 0
Change in annualized pRBC transfusion volume (among subjects who achieved TI), from 6 months post-drug product infusion (parent study) through last follow-up
Timepoint [7] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [8] 0 0
Annualized pRBC transfusion volume, from 6 months post-drug product infusion (parent study) through last follow-up
Timepoint [8] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [9] 0 0
pRBC transfusion frequency, from 6 months post-drug product infusion (parent study) through last follow-up
Timepoint [9] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [10] 0 0
Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)
Timepoint [10] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [11] 0 0
Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up
Timepoint [11] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [12] 0 0
Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up
Timepoint [12] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [13] 0 0
Unsupported total Hb levels over time through last follow-up
Timepoint [13] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [14] 0 0
Proportion of subjects with unsupported total Hb levels = 10 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Timepoint [14] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [15] 0 0
Proportion of subjects with unsupported total Hb levels = 11 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Timepoint [15] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [16] 0 0
Proportion of subjects with unsupported total Hb levels = 12 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Timepoint [16] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [17] 0 0
Proportion of subjects with unsupported total Hb levels = 13 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Timepoint [17] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [18] 0 0
Proportion of subjects with unsupported total Hb levels = 14 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Timepoint [18] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [19] 0 0
Liver iron content (LIC) by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) over time at yearly timepoints through last follow-up
Timepoint [19] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [20] 0 0
Change from parent study baseline in LIC by MRI/SQUID over time at yearly timepoints through last follow-up
Timepoint [20] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [21] 0 0
Cardiac T2* by MRI over time at yearly timepoints through last follow-up
Timepoint [21] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [22] 0 0
Change from parent study baseline in cardiac T2* by MRI over time at yearly timepoints through last follow-up
Timepoint [22] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [23] 0 0
Serum ferritin over time at yearly timepoints through last follow-up
Timepoint [23] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [24] 0 0
Change from parent study baseline in serum ferritin over time at yearly timepoints through last follow-up
Timepoint [24] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [25] 0 0
Number of subjects who stopped iron chelation post-DP infusion
Timepoint [25] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [26] 0 0
Number of subjects who stopped iron chelation for at least 6 months post-drug product infusion
Timepoint [26] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [27] 0 0
Time from stopping chelation to last follow-up
Timepoint [27] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [28] 0 0
Proportion of subjects using phlebotomy therapy post-drug product infusion
Timepoint [28] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [29] 0 0
Annualized frequency of phlebotomy therapy usage
Timepoint [29] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [30] 0 0
Reticulocyte counts over time at yearly timepoints through last follow-up
Timepoint [30] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [31] 0 0
Change from Baseline in reticulocyte counts at yearly timepoints through last follow-up
Timepoint [31] 0 0
15 years post-drug product infusion
Secondary outcome [32] 0 0
Proportion of subject with nucleated RBC over time at yearly timepoints through last follow-up
Timepoint [32] 0 0
Up to 15 years post-drug product infusion
Secondary outcome [33] 0 0
Change from Baseline in patient reported outcome (PRO) as assessed by Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score
Timepoint [33] 0 0
5 years post-drug product infusion
Secondary outcome [34] 0 0
Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y)
Timepoint [34] 0 0
5 years post-drug product infusion
Secondary outcome [35] 0 0
Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L)
Timepoint [35] 0 0
5 years post-drug product infusion
Secondary outcome [36] 0 0
Change From Baseline in PRO as assessed by Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score
Timepoint [36] 0 0
5 years post-drug product infusion
Secondary outcome [37] 0 0
Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36)
Timepoint [37] 0 0
5 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 transfusion-dependent ß-thalassemia in a
bluebird bio-sponsored clinical study
Minimum age
0 Years
Maximum age
50 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- There are no exclusion criteria for this study

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2035
Actual
Sample size
Target
Accrual to date
Final
66
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
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
South Carolina
Country [7] 0 0
France
State/province [7] 0 0
Marseille
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
Germany
State/province [9] 0 0
Hannover
Country [10] 0 0
Germany
State/province [10] 0 0
Heidelberg
Country [11] 0 0
Greece
State/province [11] 0 0
Thessaloníki
Country [12] 0 0
Italy
State/province [12] 0 0
Rome
Country [13] 0 0
Thailand
State/province [13] 0 0
Bangkok
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London

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
transfusion-dependent ß-thalassemia (TDT) who have been treated with ex vivo gene therapy
drug product in bluebird bio-sponsored parent 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 this study.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02633943
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Himal L Thakar, MD
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/ct2/show/NCT02633943