Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome.
D Bhatla, S M Davies, S Shenoy...
https://pubmed.ncbi.nlm.nih.gov/18500373Actively Recruiting
Phase 1
Phase 2
Age: 0 - 20Years
All Genders
ID00920972
A Study of Hematopoietic Stem Cell Transplantation Using Reduced-Intensity Preparatory Regimen for Non-Malignant Diseases
Led by Washington University School of Medicine · Updated on 2026-03-25
220
Participants Needed
28
Research Sites
260 weeks
Total Duration
On this page
Sponsors
W
Washington University School of Medicine
Lead Sponsor
S
St. Louis Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a reduced-intensity preparative regimen for hematopoietic stem cell transplantation in children and young adults with non-malignant disorders. This approach aims to maximize immune suppression without using radiation or high-dose chemotherapy, intending to reduce early and late toxicities. The study also focuses on preventing complications like graft-versus-host disease (GVHD) by using specific prophylaxis methods during matched and mismatched donor transplants from bone marrow and cord blood sources. The trial includes different groups based on the type of non-malignant disorder and the donor match. Treatments involve drug regimens such as Campath, Fludarabine, Melphalan, and sometimes Thiotepa, given before transplantation to prepare the body. GVHD prevention drugs like Tacrolimus, Cyclosporine, Mycophenolate mofetil (MMF), Abatacept, and Methotrexate are administered on specific days before and after the stem cell infusion, which occurs on day 0. Participants will be closely monitored for donor cell engraftment and major toxicities within the first 100 days after transplant. Assessments include measuring blood counts for neutrophil and platelet recovery, tracking acute and chronic GVHD occurrences up to two years post-transplant, and evaluating immune recovery one year after transplant. Overall and disease-free survival will also be followed for two years. The study spans from preparative treatment through long-term follow-up, with safety and effectiveness monitored at regular intervals.
CONDITIONS
Brief Title
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
Who Can Participate
Age: 0 - 20Years
All Genders
Eligibility Criteria
You may qualify if you...
- Patient younger than 21 years
- Diagnosed with a non-malignant disorder such as metabolic disorders, hemoglobinopathies, or related conditions
- Receiving a donor stem cell transplant matched as specified by the study (8/8 or 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched umbilical cord blood)
- Lansky or Karnofsky performance status of 40 or higher
- Adequate lung, kidney, liver, and other organ function as defined by the study
- Negative pregnancy test for females of childbearing potential
- Adequate dose of total nucleated cells or CD34+ cells in the transplant product
- For sickle cell disease, Hemoglobin S level less than 30%
You will not qualify if you...
- Positive HIV test
- Current invasive infection
- Pregnancy or lactation
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.
Run-in Period
Duration - Approximately 4 weeks
Participants receive daily Hydroxyurea from Day -50 to -21 as part of preparation for transplant.
Daily medication before Day -21
Treatment
Duration - About 4 weeks from Day -22 to Day 0
Participants receive a reduced-intensity conditioning regimen including Campath-1H, Fludarabine, Melphalan, and in some strata Thiotepa, followed by hematopoietic stem cell infusion on Day 0.
Multiple visits for intravenous or subcutaneous treatments and stem cell infusion
GVHD Prophylaxis and Monitoring
Duration - Up to 1 year post-transplant
Participants receive graft-versus-host disease prophylaxis with medications such as Tacrolimus or cyclosporine, MMF, Abatacept, and Methotrexate, with follow-up extending up to one year after transplant to minimize GVHD risks.
Approximately monthly visits for medication administration and monitoring during the first 100 days; additional visits at 6, 9, and 12 months depending on donor source
Follow-up
Duration - Up to 2 years post-transplant
Participants are monitored for donor engraftment, immune reconstitution, graft-versus-host disease, and overall health outcomes for up to 2 years after transplant.
Periodic visits for assessments over 2 years
Trial Site Locations
Total: 28 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
3
University of California
San Diego, California, United States, 92123
Actively Recruiting
4
Yale School of Medicine
New Haven, Connecticut, United States, 06510
Actively Recruiting
5
George Washington University School of Medicine
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
Nemours Children's Health
Jacksonville, Florida, United States, 32207
Completed
7
University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
8
Miami Children's Hospital
Miami, Florida, United States, 33155
Completed
9
All Children's Hospital
St. Petersburg, Florida, United States, 33701
Completed
10
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Completed
11
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
Actively Recruiting
12
Children's Hospital of New Orleans
New Orleans, Louisiana, United States, 70118
Completed
13
Children's Mercy
Kansas City, Missouri, United States, 64108
Completed
14
St. Louis University
St Louis, Missouri, United States, 63104
Actively Recruiting
15
Washington University School of Medicine (in St. Louis)
St Louis, Missouri, United States, 63110
Actively Recruiting
16
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Completed
17
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
18
University of North Carolina
Chapel Hill, North Carolina, United States, 27514
Actively Recruiting
19
Carolinas Medical Center
Charlotte, North Carolina, United States, 28232
Completed
20
Duke Children's Hospital
Durham, North Carolina, United States, 27705
Completed
21
The University of Oklahoma
Oklahoma City, Oklahoma, United States, 73104
Completed
22
University of Pittsburg
Pittsburgh, Pennsylvania, United States, 15224
Completed
23
Cook Children's Hospital
Forth Worth, Texas, United States, 76104
Completed
24
Texas Transplant Institute
San Antonio, Texas, United States, 78229
Completed
25
University of Utah
Salt Lake City, Utah, United States, 84132
Actively Recruiting
26
BC Children's Hospital
Vancouver, British Columbia, Canada, V6H 3N1
Completed
27
University of Manitoba
Winnipeg, Manitoba, Canada, MB R3E 0T4
Completed
28
University of Calgary
Calgary, Canada
Completed
Loading map...
Research Team
L
Lissy Keller, CCRP
I
Ian Snyder, CCRP
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Allogeneic hematopoietic cell transplantation (HCT) in Hurler's syndrome using a reduced intensity preparative regimen.
M D Hansen, A H Filipovich, S M Davies...
https://pubmed.ncbi.nlm.nih.gov/18026148Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning.
Aarati Rao, Naynesh Kamani, Alexandra Filipovich...
https://pubmed.ncbi.nlm.nih.gov/16990602A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders.
S Shenoy, W J Grossman, J DiPersio...
https://pubmed.ncbi.nlm.nih.gov/15592491Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies.
Allison A King, Naynesh Kamani, Nancy Bunin...
https://pubmed.ncbi.nlm.nih.gov/26348869