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Trial record 1 of 1 for:    ltf-303
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Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02633943
Recruitment Status : Enrolling by invitation
First Posted : December 17, 2015
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
bluebird bio

Tracking Information
First Submitted Date November 30, 2015
First Posted Date December 17, 2015
Last Update Posted Date October 19, 2020
Study Start Date September 2013
Estimated Primary Completion Date March 2031   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 24, 2018)
  • Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study [ Time Frame: 15 years post-drug product infusion ]
  • All adverse events (AEs) related to drug product [ Time Frame: 15 years post-drug product infusion ]
  • All serious adverse events (SAEs) [ Time Frame: 15 years post-drug product infusion ]
  • Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) [ Time Frame: 15 years post-drug product infusion ]
  • Monitoring of βA-T87Q-globin [ Time Frame: 15 years post-drug product infusion ]
  • Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
  • Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
  • 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 [ Time Frame: 15 years post-drug product infusion ]
Original Primary Outcome Measures
 (submitted: December 15, 2015)
  • Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study [ Time Frame: 15 years post-drug product infusion ]
  • All serious adverse events (SAEs) related to drug product [ Time Frame: 15 years post-drug product infusion ]
  • Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) [ Time Frame: 15 years post-drug product infusion ]
  • Monitoring of βA-T87Q-globin analysis by high-performance liquid chromatography [ Time Frame: 15 years post-drug product infusion ]
  • Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
  • Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
  • 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 [ Time Frame: 15 years post-drug product infusion ]
    ACS is defined as an acute event with pneumonia-like symptoms and the presence of a new pulmonary infiltrate. A severe VOC is defined as an episode of pain, lasting more than 2 hours and requiring care at a medical facility.
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy
Official Title Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Brief Summary This is a multi-center, long-term safety and efficacy follow-up study for subjects with hemoglobinopathies (β-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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Subjects with hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
Condition
  • Beta-Thalassemia
  • Sickle Cell Disease
Intervention Other: 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
Study Groups/Cohorts 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
Intervention: Other: Safety and efficacy assessments
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Enrolling by invitation
Estimated Enrollment
 (submitted: August 6, 2018)
94
Original Estimated Enrollment
 (submitted: December 15, 2015)
27
Estimated Study Completion Date March 2031
Estimated Primary Completion Date March 2031   (Final data collection date for primary outcome measure)
Eligibility Criteria

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

Exclusion Criteria:

  • There are no exclusion criteria for this study
Sex/Gender
Sexes Eligible for Study: All
Ages up to 50 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   France,   Germany,   Italy,   Thailand,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02633943
Other Study ID Numbers LTF-303
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party bluebird bio
Study Sponsor bluebird bio
Collaborators Not Provided
Investigators
Study Director: Richard Colvin bluebird bio, Inc.
Study Director: Sunita Goyal bluebird bio, Inc.
PRS Account bluebird bio
Verification Date October 2020