What is this study trying to find out about non-malignant disorders requiring stem cell transplant?

This study is evaluating a reduced-intensity conditioning regimen before stem cell transplant for patients with non-malignant disorders. It aims to see if this approach improves transplant success and reduces side effects compared to standard treatment. The research is led by Paul Szabolcs and focuses on conditions like immune deficiencies, metabolic, and hereditary disorders.

Who may be eligible for this non-malignant disorder clinical trial?

Adults and children aged 2 to 55 with certain non-malignant diseases that can be treated by stem cell transplant may be eligible. Participants must have suitable donor cells available and adequate organ function. They must not have had a recent stem cell transplant or active serious infections. Please review eligibility criteria for more details.

Is there any cost to participate in this non-malignant disorder clinical trial?

Study-related care, including the study drugs and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel or accommodation costs.

Actively Recruiting

Phase 2

Age: 2Months - 55Years

All Genders

ID01962415

Phase II Study of Reduced Intensity Conditioning Before Stem Cell Transplant in Children and Young Adults Up to 55 Years With Non-Malignant Disorders

Led by Paul Szabolcs · Updated on 2025-12-15

100

Participants Needed

Research Sites

52 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating a reduced-intensity conditioning RIC regimen for patients with non-malignant disorders who need a hematopoietic stem cell transplant HSCT. This study focuses on diseases such as thalassemia, sickle cell disease, primary immunodeficiencies, metabolic disorders, and other inherited conditions. The goal is to assess if RIC before transplant can improve outcomes by reducing complications and transplant-related mortality compared to the standard myeloablative conditioning. The treatments being studied include a conditioning regimen with Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa given before stem cell transplants from umbilical cord blood, bone marrow, or peripheral blood stem cells. Two groups are involved one receiving double cord blood transplants for transfusion-dependent anemia or higher rejection risk, and another receiving bone marrow or peripheral blood transplants. The study uses lower chemotherapy doses to prepare patients, aiming to help donor stem cells engraft successfully while decreasing side effects on organs. Participants will be monitored for transplant success and side effects over time, including immune recovery, rates of infections, graft-versus-host disease, organ toxicity, and neurodevelopmental progress for up to one year after transplant. Researchers will also track donor cell engraftment and blood counts. The total participation may last several months to years, with follow-up visits to evaluate long-term outcomes and transplant effectiveness.

CONDITIONS

Brief Title

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who Can Participate

Age: 2Months - 55Years

All Genders

Eligibility Criteria

You may qualify if you...

Age between 2 and 55 years
Availability of a matched related or unrelated umbilical cord blood unit or matched unrelated donor bone marrow or peripheral blood graft
Adequate kidney function (creatinine 4 mg/dL and clearance 5 mL/min/1.73 m2)
Liver enzymes (ALT/AST) less than or equal to 4 times the upper limit of normal
Adequate heart function by echocardiogram or radionuclide scan
Adequate lung function as measured by pulmonary tests or oxygen levels
Written informed consent or assent as per FDA guidelines
Negative pregnancy test if pubertal or menstruating
HIV negative
Diagnosis of a non-malignant disorder suitable for stem cell transplant including specific immune deficiencies, bone marrow failure syndromes, inherited metabolic disorders, hereditary anemias, or inflammatory conditions

You will not qualify if you...

Allogeneic stem cell transplant within the previous 6 months
Active malignancy or myelodysplastic syndrome
Severe acquired aplastic anemia
Uncontrolled infections with progression despite treatment
Pregnancy or nursing
Poorly controlled pulmonary hypertension
Any condition preventing regular follow-up

Research Team

Paul Szabolcs, MD

Shawna McIntyre, RN

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