Recruiting

Observational Study

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT06646640

Conditions

Hemoglobin Disorder

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

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

Study ID

NCT06646640

Start date

Jun 26, 2025

Status verified date

Aug, 2025

Completion date

Jan, 2050

Anticipated

Primary completion date

Jan, 2035

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • 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" ]

Central Contacts and Locations

Central contacts

Akshay Sharma, MD

8662785833referralinfo@stjude.org

Neha Das Gupta, PhD

8662785833referralinfo@stjude.org

Locations

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Sharma Akshay, MD

866-278-5833referralinfo@stjude.org

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

Aug 7, 2025

Last verified

Aug, 2025

Keywords

  • Transplant
  • Gene Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2025-08-07.