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Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy

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ClinicalTrials.gov Identifier: NCT04628585
Recruitment Status : Enrolling by invitation
First Posted : November 13, 2020
Last Update Posted : August 19, 2021
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 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
Sickle Cell Disease Other: Safety and efficacy assessments

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Study Type : Observational
Estimated Enrollment : 85 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Actual Study Start Date : October 21, 2020
Estimated Primary Completion Date : January 2038
Estimated Study Completion Date : January 2038

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Subjects with sickle-cell disease
Subjects treated with ex vivo gene therapy drug products in a bluebird bio-sponsored study who agree to participate in this long-term follow-up study
Other: Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant




Primary Outcome Measures :
  1. Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) [ Time Frame: Through 15 years post-drug product infusion ]
  2. Number of subjects with new or worsening hematologic disorders [ Time Frame: Through 15 years post-drug product infusion ]
  3. Number of subjects with new or worsening neurologic disorders [ Time Frame: Through 15 years post-drug product infusion ]
  4. Number of subjects with malignancies [ Time Frame: Through 15 years post-drug product infusion ]

Secondary Outcome Measures :
  1. Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  2. Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  3. Annualized number of severe VOEs over time through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  4. Annualized number of VOEs over time through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  5. Change from parent study baseline in annualized number of severe VOEs over time through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  6. Assessment of total Hb over time post-drug product infusion through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
  7. Assessment of non-transfused total Hb over time post-drug product infusion through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
    Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q

  8. Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
    Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q

  9. Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
    Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q

  10. Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [ Time Frame: Through 15 years post-drug product infusion ]
    Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q. Non-HbS is the total g/dL of HbF + HbA2 + HbAT87Q

  11. Change from parent study baseline through Year 15 in hemolysis markers [ Time Frame: Through 15 years post-drug product infusion ]
  12. Change from parent study baseline through Year 15 in markers of iron stores [ Time Frame: 15 years post-drug product infusion ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 53 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects with sickle cell disease 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 subject, or as applicable, subject's parent(s)/legal guardian(s)
  • Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study

Exclusion Criteria:

  • There are no exclusion criteria for this 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): NCT04628585


Locations
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United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611-2991
United States, Maryland
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, United States, 20892
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
France
Hospital Necker
Paris, France, 75015
Sponsors and Collaborators
bluebird bio
Investigators
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Study Director: Richard Colvin bluebird bio, Inc.
Additional Information:
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Responsible Party: bluebird bio
ClinicalTrials.gov Identifier: NCT04628585    
Other Study ID Numbers: LTF-307
2019-004266-18 ( EudraCT Number )
First Posted: November 13, 2020    Key Record Dates
Last Update Posted: August 19, 2021
Last Verified: July 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by bluebird bio:
Hematopoietic Stem Cells
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn