ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    ltf-303
Previous Study | Return to List | Next Study

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 : August 7, 2018
Sponsor:
Information provided by (Responsible Party):
bluebird bio

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.

Condition or disease Intervention/treatment
Beta-Thalassemia Sickle Cell Disease Other: Safety and efficacy assessments

Study Type : Observational
Estimated Enrollment : 94 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
Study Start Date : September 2013
Estimated Primary Completion Date : March 2031
Estimated Study Completion Date : March 2031


Group/Cohort Intervention/treatment
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: 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




Primary Outcome Measures :
  1. 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 ]
  2. All adverse events (AEs) related to drug product [ Time Frame: 15 years post-drug product infusion ]
  3. All serious adverse events (SAEs) [ Time Frame: 15 years post-drug product infusion ]
  4. Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) [ Time Frame: 15 years post-drug product infusion ]
  5. Monitoring of βA-T87Q-globin [ Time Frame: 15 years post-drug product infusion ]
  6. 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 ]
  7. 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 ]
  8. 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 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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:

  • Met the VCN discontinuation criterion in the parent study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02633943


Locations
United States, California
Oakland, California, United States
United States, Illinois
Chicago, Illinois, United States
United States, Maryland
Bethesda, Maryland, United States
United States, Pennsylvania
Philadelphia, Pennsylvania, United States
United States, South Carolina
Charleston, South Carolina, United States
Australia
Sydney, Australia
France
Paris, France
Thailand
Bangkok, Thailand
Sponsors and Collaborators
bluebird bio
Investigators
Study Director: Mohammed Asmal, MD, PhD bluebird bio, Inc.

Additional Information:
Responsible Party: bluebird bio
ClinicalTrials.gov Identifier: NCT02633943     History of Changes
Other Study ID Numbers: LTF-303
First Posted: December 17, 2015    Key Record Dates
Last Update Posted: August 7, 2018
Last Verified: August 2018

Additional relevant MeSH terms:
Anemia, Sickle Cell
Thalassemia
beta-Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn