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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02633943
Ethics application status
Date submitted
30/11/2015
Date registered
17/12/2015

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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Bluebird bio is committed to transparency and appropriately de-identified subject-level datasets and supporting documents may be shared after all participants have completed study participation and following attainment of applicable marketing approvals associated with a given study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at [email protected].
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02633943