Thalassemia Protocol Information

New Clinical Trial Seeks Thalassemia Intermedia Patients for a Study of Butyrate Plus Erythropoietin Sponsored by the National Institutes of Health at Boston University Medical Center, Boston, MA, USA

An experimental medicine, Butyrate, has increased hemoglobin levels in 5 of 6 thalassemia patients by 2-4 grams above baseline (average= 2.8g/dl). Two thalasemia major patients have become free of transfusions for up to 5 years. A new study is now being conducted to evaluate two experimental medicines for thalassemia: Arginine Butyrate and Erythropoietin.


To increase hemoglobin levels in thalassemia intermedia patients by at least 2g/dl above baseline levels using Butyrate and Erythropoietin.

To enroll:

Patients should meet the following criteria:
1. Not require regular transfusions
2. Have a baseline hemoglobin of less than or equal to 10g/dl
3. Have had a splenectomy or have no palpable spleen
4. Be willing to travel to Boston University Medical Center or Mt. Sinai Medical Center in New York City and stay (in a shared apartment or home) for at least 6 weeks (unless your hematologist can devote extra time to collaborate)
5. Be willing to have a port-a-cath placed (if one is not already in place)
6. Be willing to learn to give oneself the medicines at home
7. Be willing to continue treatment for approximately 8 months at home and receive close medical attention with weekly visits from visiting nurses, monthly visits to their hematology physician, and follow-up in Boston 2-3 times.


Dr. Susan P. Perrine at (617)-638-5639
Boston University School of Medicine
Hemoglobinopathy-Thalassemia Research Unit
80 E. Concord St. L-908
Boston, MA 02118 USA


**Some funding is available for shared housing and travel expenses to Boston or New York if a family cannot afford this**

Thalassemia Protocol

Adult Informed Consent Form

Child Informed Consent Form and Assent Form

Protocol Screening Forms

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