Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Stem Cell Transplant or Gene Therapy
Not yet recruiting
- Conditions
- Registration Number
- NCT06646640
- Lead Sponsor
- St. Jude Children's Research Hospital
- 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 HSCT or autologous genetically modified cellular products for hemoglobin disorders.
Primary objective:
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- Detailed Description
This protocol will provide a mechanism to appropriately monitor individuals with hemoglobin disorders that have received an allogenic HSCT 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 recei...
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 200
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
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Exclusion Criteria
- Inability or unwillingness of research participant and/or legal guardian/ representative to provide written informed consent.
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Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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"
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States