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

Conditions

• Hemoglobin Disorder

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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).

Key Dates

Start date

Jun 26, 2025

Status verified

Aug 2025

Primary completion

Jan 31, 2035

Completion

Jan 31, 2050

Study Design

Enrollment

200 participants (estimated)

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

• Akshay Sharma, MD
  8662785833
  [email protected]
• Neha Das Gupta, PhD
  8662785833
  [email protected]

Locations (1)

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