Actively Recruiting
Phase 2
Age: 2Months - 55Years
All Genders
ID01962415
A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults 55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
Led by Paul Szabolcs · Updated on 2025-12-15
100
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Approximately 1 to 2 weeks
Participants receive a reduced intensity conditioning regimen including Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa prior to stem cell transplantation.
Multiple visits for drug administration and monitoring during conditioning and transplantation
Follow-up
Duration - Up to 1 year post-transplant
Participants are monitored for transplant-related outcomes including treatment-related mortality, immune reconstitution, graft versus host disease, and organ toxicity.
Regular visits during the first year post-transplant for clinical assessments and laboratory tests
Trial Site Locations
Total: 1 location
1
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
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Research Team
P
Paul Szabolcs, MD
S
Shawna McIntyre, RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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Published Research Related To This Trial
Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.
Mark T Vander Lugt, Xiaohua Chen, Maria L Escolar...
https://pubmed.ncbi.nlm.nih.gov/32634238