St. Jude Children's Research Hospital
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: - To provide long term follow up, for individuals with hemoglobin disorders undergoing allogeneic hematopoietic stem cell transplantation (HSCT) 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).
Hemoglobin Disorder
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 receipt of the genetically modified cellular product.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 200 participants |
| Official Title : | Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Stem Cell Transplant or Gene Therapy |
| Actual Study Start Date : | 2024-12 |
| Estimated Primary Completion Date : | 2035-01 |
| Estimated Study Completion Date : | 2050-01 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
Not yet recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105