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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 : December 28, 2020
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 : May 2037
Estimated Study Completion Date : May 2037

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. Non-transfused total hemoglobin (Hb) over time [ Time Frame: 15 years post-drug product infusion ]

Secondary Outcome Measures :
  1. Overall survival [ Time Frame: 15 years post-drug product infusion ]
  2. All drug product-related AEs [ Time Frame: 15 years post-drug product infusion ]
  3. All serious adverse events (SAEs) [ Time Frame: 15 years post-drug product infusion ]
  4. Serious or non-serious immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) and new or worsening hematologic or neurologic disorders or malignancies [ Time Frame: 15 years post-drug product infusion ]
  5. Incidence of insertional mutagenesis leading to leukemia [ Time Frame: 15 years post-drug product infusion ]
  6. Assessment of the following over time: [ Time Frame: 15 years post-drug product infusion ]
    • non-transfused total Hb ≥10g/dL
    • HbS percentage of non-transfused total Hb
    • HbS percentage of non-transfused total Hb ≤70%, ≤60%, ≤50%
    • HbAT87Q percentage of non-transfused total Hb
    • HbAT87Q percentage of non-transfused total Hb ≥30%, ≥40%, ≥50%
    • non-HbS percentage of non-transfused total Hb

    Total Hb refers to non-transfused total Hb, which is equivalent to the total g/dL of HbS + HbF + HbA2 + HbAT87Q for subjects without a β+ allele. For subjects with a β+ allele, HbA will also be included in the calculation of "non-transfused total Hb" for samples taken ≥60 days after last pRBC transfusion.

    Non-HbS is the total g/dL of HbF + HbA2 + HbAT87Q


  7. Change from parent study baseline in hemolysis markers [ Time Frame: 15 years post-drug product infusion ]
  8. Change from parent study baseline in markers of iron stores [ Time Frame: 15 years post-drug product infusion ]
  9. Change from parent study baseline in markers of stress erythropoiesis/anemia [ Time Frame: Month 36, Month 60, and Year 15 post-drug product infusion ]
  10. Annualized pRBC transfusion volume [ Time Frame: 6-months post-drug product infusion through 15 years ]
    Annualized pRBC transfusion volume (mL/kg/year) from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment.

  11. Annualized pRBC transfusion frequency [ Time Frame: 6-months post-drug product infusion through 15 years ]
    Annualized pRBC transfusion frequency (number/year) from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment.

  12. Change in SCD complications [ Time Frame: 15 years post-drug product infusion ]
    Change in SCD complications which may include but are not limited to: vaso-occlusive events, new or worsening osteonecrosis, or severe leg ulcers; retinopathy, change from baseline in renal function, cardiac-pulmonary function



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
  • Able to comply with study requirements

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, 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: Sunita Goyal 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: December 28, 2020
Last Verified: December 2020

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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