Actively Recruiting
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Led by Paul Szabolcs · Updated on 2026-01-13
50
Participants Needed
1
Research Sites
410 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
CONDITIONS
Official Title
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient, parent, or legal guardian must have given written informed consent.
- Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
- Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to primary immunodeficiency syndromes, congenital bone marrow failure syndromes, inherited metabolic disorders, hereditary anemias, and inflammatory conditions.
- Subjects receive either umbilical cord blood, bone marrow, or peripheral blood stem cell transplant with an alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen, according to clinical practice at UPMC Children's Hospital of Pittsburgh.
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
Research Team
P
Paul Szabolcs, MD
CONTACT
S
Shawna McIntyre, RN
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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