Donor transplant for high risk blood disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II trial investigates the use of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with high risk hemoglobinopathies, including sickle cell disease and thalassemia. The study involves a single center and employs an open-label design, utilizing various interventions such as Alemtuzumab, Total Body Irradiation, and Fludarabine. Eligible participants must have a fully matched sibling donor and meet specific health criteria. The trial aims to assess the effectiveness of this transplant approach in improving patient outcomes.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Sickle Cell Disease (SCD)
* SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent
* Transfusion Dependent Alpha- or Beta- Thalassemia
* Diamond Blackfan Anemia
* Other Non-Malignant Hematologic Disorders
* Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation.
* Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.

Exclusion Criteria:

* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment
* HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.
* Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
* Known allergy to any of the study components
* Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study

Where it's running

• Masonic Cancer Center
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Study coordinator: Ashish Gupta, MBBS, MPH
• Email: gupta461@umn.edu
• Phone: 612-626-2961

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Ruxolitinib-enhanced haploidentical transplant for children and young adults with sickle cell disease

  This trial will test whether adding ruxolitinib to a reduced-intensity haploidentical transplant lowers the risk of graft failure in children and young adults with sickle cell disease.

  Recruiting Phase 1/2 Arkansas Children's Hospital Research Institute · Aurora, Colorado and 3 other locations · NCT07252050

• Investigating genetic factors in blood disorders

  This study is trying to find out how genes affect blood disorders like sickle cell disease and thalassemia by looking at the DNA of many people with these conditions.

  Recruiting at 17 sites Cyprus Institute of Neurology and Genetics · Boston, Massachusetts and 25 other locations · NCT05799118

• Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease

  This study is testing a single dose of a new treatment called CTX001 to see if it can help people with transfusion-dependent beta-thalassemia or severe sickle cell disease feel better.

  Recruiting at 6 sites Phase 3 Vertex Pharmaceuticals Incorporated · New York, New York and 5 other locations · NCT05477563

• Cerebrovascular Reactivity Testing with HD-DOT in Sickle Cell Patients

  This study tests whether high-density diffuse optical tomography can measure brain blood flow and oxygen use in people with sickle cell disease, to see how these signals relate to stroke risk.

  Recruiting Washington University School of Medicine · St Louis, Missouri · NCT07776444

• Adapting and testing PEN-Plus in Zambian primary care

  Aiming to see if the PEN-Plus package can be adapted and used in two Zambian hospitals to better manage chronic diseases like heart disease, diabetes, hypertension, and sickle cell in primary care.

  Recruiting Centre for Infectious Disease Research in Zambia · Lusaka, Lusaka Province · NCT07753681

• Newborn Sickle Cell Screening in Mozambique

  We’re testing a three-part plan to help babies in Mozambique get diagnosed early for sickle cell disease and start care quickly.

  Recruiting St. Jude Children's Research Hospital · Nhamatanda, Sofala · NCT07719972

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.