Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy

Last updated: April 22, 2025
Sponsor: St. Jude Children's Research Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Red Blood Cell Disorders

Treatment

N/A

Clinical Study ID

NCT06646640
HEMLTFU
  • All Genders

Study Summary

This is a prospective, longitudinal, non-therapeutic study which includes routine assessment for long-term effects, as per FDA guidelines after receipt of an allogeneic HCT or autologous genetically modified cellular products for hemoglobin disorders.

Primary objective:

  • To provide long term follow up, for individuals with hemoglobin disorders undergoing allogeneic hematopoietic stem cell transplantation (HCT) or receipt of an autologous genetically modified cellular product to treat their hemoglobinopathy. For individuals receiving a genetically modified cellular product, this long term follow up study is in accordance with the guidelines provided by the Food and Drug Administration (FDA).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Receipt, or planned receipt, of an allogeneic HSCT or infusion of geneticallymodified autologous cells for hemoglobin disorders within 15 years prior toenrollment

Exclusion

Exclusion Criteria:

  • Inability or unwillingness of research participant and/or legal guardian/representative to provide written informed consent.

Study Design

Total Participants: 200
Study Start date:
June 01, 2025
Estimated Completion Date:
January 31, 2050

Study Description

This protocol will provide a mechanism to appropriately monitor individuals with hemoglobin disorders that have received an allogenic HCT or an autologous genetically modified cellular product for hemoglobinopathies. Monitoring will include potential long-term adverse effects after receipt of these treatments, as well as long-term monitoring after the receipt of the genetically modified cellular product.

Connect with a study center

  • St. Jude Children's Research Hospital

    Memphis, Tennessee 38105
    United States

    Active - Recruiting

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