RecruitingNCT06646640

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


Sponsor

St. Jude Children's Research Hospital

Enrollment

200 participants

Start Date

Jun 26, 2025

Study Type

OBSERVATIONAL

Conditions

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

Inclusion Criteria1

  • Receipt, or planned receipt, of an allogeneic HSCT or infusion of genetically modified autologous cells for hemoglobin disorders within 15 years prior to enrollment

Exclusion Criteria1

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

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Locations(1)

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

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NCT06646640


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