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

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).

Recruiting

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Key information

Conditions

Hemoglobin Disorder Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Sex eligibility

All sexes

Study type

Observational

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location status: Recruiting

Location contact

Sharma Akshay, MD

CONTACT

[email protected]

866-278-5833

About this study

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.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

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 criteria

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

Treatment and study plan

Primary outcomes

To provide ongoing review of long-term clinical and psychosocial outcomes and late effects of hematopoietic stem cell transplant and gene therapy recipients at St. Jude Children's Research Hospital.

Time frame: long term follow up. Can also be stated as "up to 15 years after transplant"

Study contacts

Contact information is provided by the study sponsor or research team.

Akshay Sharma, MD

CONTACT

[email protected]

8662785833

Neha Das Gupta, PhD

CONTACT

[email protected]

8662785833

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Important dates

Study start: 2025
Primary completion: 2035
Study completion: 2050
First posted: Oct 17, 2024
Registry last updated: Aug 7, 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.

Published trials that share one or more normalized conditions with this study.

Recruiting