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Enrolling by Invitation

NCT Number: NCT04628585

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

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

2 year–53 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Necker, Paris, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Safety and efficacy assessments

Other

Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Primary outcomes

  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 outcomes

  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

    Change from parent study baseline through Year 15 in absolute reticulocyte count, percent reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase hemolysis markers

  12. Change from parent study baseline through Year 15 in markers of iron stores

    Time frame: 15 years post-drug product infusion

    Change from parent study baseline through Year 15 in serum ferritin and liver iron content markers of iron stores

Sponsors and collaborators

Lead sponsor

Genetix Biotherapeutics Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2038
Study completion
2038
First posted
Nov 13, 2020
Registry last updated
Mar 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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