Transplant conditioning for non-malignant diseases using immune suppression
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
This study is testing a new way to prepare children with non-cancer diseases for a stem cell transplant to see if it helps their bodies accept the donor cells while causing fewer side effects.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 220 (estimated) |
| Ages | N/A to 20 Years |
| Sex | All |
| Sponsor | Washington University School of Medicine Academic / other |
| Drugs / interventions | chemotherapy, radiation |
| Locations | 28 sites (Phoenix, Arizona and 27 other locations) |
| Trial ID | NCT00920972 on ClinicalTrials.gov |
What this trial studies
This study investigates a preparative regimen that maximizes host immunosuppression without myeloablation to facilitate the engraftment of donor hematopoietic cells in children with non-malignant disorders. It employs reduced intensity conditioning to minimize early and late toxicities associated with transplant, such as graft versus host disease (GVHD). The trial evaluates the safety and efficacy of this approach by monitoring major toxicities within the first 100 days post-transplantation, utilizing matched and mismatched donor stem cells from both bone marrow and umbilical cord blood sources.
Who should consider this trial
Good fit: Ideal candidates include children under 21 years with specific non-malignant disorders requiring HLA-matched bone marrow or umbilical cord blood transplants.
Not a fit: Patients with HIV, invasive infections, or those who are pregnant or lactating may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly reduce transplant-related toxicities while improving donor cell engraftment in pediatric patients with non-malignant disorders.
How similar studies have performed: Other studies utilizing reduced intensity conditioning for transplant have shown promise, indicating potential for success in this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Stratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8/8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. Stratum 3: Patient must have a hemoglobinopathy receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. All strata: * Recipient age \< 21 years * Lansky/Karnofsky \>/= 40 * Adequate pulmonary, renal, liver, and other organ function as defined in protocol * Negative pregnancy test * Adequate total nucleated cell or CD34+ dose of product as defined in protocol * If sickle cell, Hemoglobin S \<30% Exclusion Criteria: * HIV positive * Invasive infection * Pregnancy/lactating
Where this trial is running
Phoenix, Arizona and 27 other locations
- Phoenix Children's Hospital — Phoenix, Arizona, United States (Recruiting)
- Children's Hospital of Orange County — Orange, California, United States (Recruiting)
- University of California — San Diego, California, United States (Recruiting)
- Yale School of Medicine — New Haven, Connecticut, United States (Recruiting)
- George Washington University School of Medicine — Washington D.C., District of Columbia, United States (Recruiting)
- Nemours Children's Health — Jacksonville, Florida, United States (Completed)
- University of Miami — Miami, Florida, United States (Recruiting)
- Miami Children's Hospital — Miami, Florida, United States (Completed)
- All Children's Hospital — St. Petersburg, Florida, United States (Completed)
- Children's Memorial Hospital — Chicago, Illinois, United States (Completed)
- Indiana University School of Medicine — Indianapolis, Indiana, United States (Recruiting)
- Children's Hospital of New Orleans — New Orleans, Louisiana, United States (Completed)
- Children's Mercy — Kansas City, Missouri, United States (Completed)
- St. Louis University — St Louis, Missouri, United States (Recruiting)
- Washington University School of Medicine (in St. Louis) — St Louis, Missouri, United States (Recruiting)
- Hackensack University Medical Center — Hackensack, New Jersey, United States (Completed)
- Columbia University Medical Center — New York, New York, United States (Recruiting)
- University of North Carolina — Chapel Hill, North Carolina, United States (Recruiting)
- Carolinas Medical Center — Charlotte, North Carolina, United States (Completed)
- Duke Children's Hospital — Durham, North Carolina, United States (Completed)
- The University of Oklahoma — Oklahoma City, Oklahoma, United States (Completed)
- University of Pittsburg — Pittsburgh, Pennsylvania, United States (Completed)
- Cook Children's Hospital — Forth Worth, Texas, United States (Completed)
- Texas Transplant Institute — San Antonio, Texas, United States (Completed)
- University of Utah — Salt Lake City, Utah, United States (Recruiting)
- BC Children's Hospital — Vancouver, British Columbia, Canada (Completed)
- University of Manitoba — Winnipeg, Manitoba, Canada (Completed)
- University of Calgary — Calgary, Canada (Completed)
Study contacts
- Principal investigator: Shalini Shenoy, MD — Washington University School of Medicine (in St. Louis)
- Study coordinator: Lissy Keller, CCRP
- Email: kellerl@wustl.edu
- Phone: 3142861168
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.