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HEMLTFU: Late Effects of Gene Therapy and Stem Cell Transplant for Hemoglobin Disorders

About this study

Hemoglobin disorders such as sickle cell disease, alpha thalassemia, and beta thalassemia are types of blood disorders. They may be cured with therapies such as bone marrow (stem cell) transplant) or gene therapy. However, after treatment ends, patients may have life-changing late effects. Since gene therapies are very new, we do not completely understand the long-term effects of these treatments and how they compare to long-term effects after bone marrow transplant.

This study will track the long-term effects of bone marrow transplants and gene therapy in patients with hemoglobin disorders such as sickle cell disease and thalassemia.

Patients who take part in this study will get regular health checkups and other tests, and questionnaires to check for late effects. Patients will also be offered the chance to take part in the TBANK study that stores and uses human tissue samples in the St. Jude biorepository. This study will follow patients for 15 years.

We will use what we learn from this study to try to understand and decrease any long-term effects of these treatments for other patients in the future.

Eligibility overview

  • Hemoglobin disorder (sickle cell disease and thalassemia)
  • Ages 5 and older
  • Have had a bone marrow transplant or gene therapy within the last 15 years 

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The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

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

Study goal:

To study the long-term health of patients with hemoglobin disorders who receive either a stem cell transplant or gene therapy product.

Diagnosis:

Alpha Thalassemia, Beta Thalassemia, Sickle cell disease

Age:

5 years and older

Clinical trials categories:

Alpha Thalassemia Blood Disorders Beta Thalassemia Sickle Cell Disease Genetic Syndromes

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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