Long-term follow-up for patients with hemoglobin disorders after transplant or gene therapy
Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy
This study looks at how people with hemoglobin disorders are doing long-term after they’ve had a stem cell transplant or gene therapy to see if there are any lasting effects or problems.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Sex | All |
| Sponsor | St. Jude Children's Research Hospital Academic / other |
| Locations | 1 site (Memphis, Tennessee) |
| Trial ID | NCT06646640 on ClinicalTrials.gov |
What this trial studies
This observational study aims to monitor individuals with hemoglobin disorders who have undergone allogeneic hematopoietic stem cell transplantation or received autologous genetically modified cellular products. It focuses on assessing the long-term effects and potential adverse outcomes of these treatments in accordance with FDA guidelines. Participants will be routinely evaluated to gather data on their health status and any complications that may arise over time.
Who should consider this trial
Good fit: Ideal candidates are individuals who have received or are planning to receive an allogeneic hematopoietic stem cell transplant or genetically modified cellular therapy for hemoglobin disorders within the last 15 years.
Not a fit: Patients who have not undergone these specific treatments or are unable to provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and efficacy of treatments for hemoglobin disorders, improving patient care.
How similar studies have performed: While this study follows established FDA guidelines for long-term monitoring, similar studies have shown success in tracking outcomes after hematopoietic stem cell transplants and gene therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Memphis, Tennessee
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
Study contacts
- Principal investigator: Akshay Sharma, MD — St. Jude Children's Research Hospital
- Study coordinator: Akshay Sharma, MD
- Email: referralinfo@stjude.org
- Phone: 8662785833
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.