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

NCT06646640 | Recruiting

• 1 other identifier: HEMLTFU
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Hemoglobin Disorder

Keywords

Transplant; Gene Therapy

Outcome Measures

Primary Outcomes (1)

• 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.

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

200 patients with hemoglobin disorders that have received, or are planning to receive, either a genetically modified cell product or allogenic HCT will be invited to enroll on this long term follow up study.

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Hemoglobinopathies

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Akshay Sharma, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Akshay Sharma, MD

CONTACT

8662785833; referralinfo@stjude.org

Neha Das Gupta, PhD

CONTACT

8662785833; referralinfo@stjude.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2024
• First Posted: October 17, 2024
• Study Start: June 26, 2025
• Primary Completion (Estimated): January 1, 2035
• Study Completion (Estimated): January 1, 2050
• Last Updated: August 7, 2025

Record last verified: 2025-08

Locations

US (1)