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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).
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.
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| Measure | Description | Time Frame |
|---|---|---|
| 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" |
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Inclusion Criteria:
Exclusion Criteria:
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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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Akshay Sharma, MD | Contact | 8662785833 | referralinfo@stjude.org | |
| Neha Das Gupta, PhD | Contact | 8662785833 | referralinfo@stjude.org |
| Name | Affiliation | Role |
|---|---|---|
| Akshay Sharma, MD | St. Jude Children's Research Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Jude Children's Research Hospital | Recruiting | Memphis | Tennessee | 38105 | United States |
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| Label | URL |
|---|---|
| St. Jude Children's Research Hospital | View source |
| Clinical Trials Open at St. Jude | View source |
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| ID | Term |
|---|---|
| D006453 | Hemoglobinopathies |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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