Data collection for patients with non-malignant disorders undergoing stem cell transplantation
A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)
This study is collecting information from patients with non-cancerous disorders who are getting stem cell transplants to see if a new treatment plan helps them recover better and avoid complications.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 2 Months to 60 Years |
| Sex | All |
| Sponsor | University of Pittsburgh Academic / other |
| Drugs / interventions | alemtuzumab, fludarabine, chemotherapy |
| Locations | 1 site (Pittsburgh, Pennsylvania) |
| Trial ID | NCT04528355 on ClinicalTrials.gov |
What this trial studies
This observational study collects data on patients with non-malignant disorders who are undergoing hematopoietic stem cell transplantation (HSCT) using a reduced-intensity chemotherapy regimen. The focus is on understanding the effectiveness of a novel conditioning regimen that includes alemtuzumab and other agents to prevent graft failure and support immune reconstitution. By gathering clinical data, the study aims to assess outcomes such as engraftment, graft-versus-host disease (GVHD), immunosuppressant use, and overall survival in this patient population. The findings may also provide insights for future gene therapy strategies.
Who should consider this trial
Good fit: Ideal candidates include patients aged 2 months to 60 years with non-malignant disorders suitable for stem cell transplantation, such as primary immunodeficiency syndromes and congenital bone marrow failure syndromes.
Not a fit: Patients with malignant disorders or those not eligible for stem cell transplantation will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve outcomes for patients undergoing stem cell transplantation for non-malignant disorders.
How similar studies have performed: Previous studies have shown promising results with similar reduced-intensity conditioning regimens, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
1. Patient, parent, or legal guardian must have given written informed consent.
2. Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
3. Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
A. Primary Immunodeficiency Syndromes
* Severe Combined Immune Deficiency (SCID) with NK cell activity
* Omenn Syndrome
* Bare Lymphocyte Syndrome (BLS)
* Combined Immune Deficiency (CID) syndromes
* Combined Variable Immune Deficiency (CVID) syndrome
* Wiskott-Aldrich Syndrome
* Leukocyte adhesion deficiency
* Chronic granulomatous disease (CGD)
* Hyper IgM (XHIM) syndrome
* IPEX syndrome
* Chediak-Higashi Syndrome
* Autoimmune Lymphoproliferative Syndrome (ALPS)
* Hemophagocytic Lymphohistiocytosis (HLH) syndromes
* Lymphocyte Signaling defects
B. Congenital Bone Marrow Failure Syndromes
* Congenital Amegakaryocytic Thrombocytopenia (CAMT)
* Osteopetrosis
C. Inherited Metabolic Disorders (IMD)
* Mucopolysaccharidoses
* Hurler syndrome (MPS I)
* Hunter syndrome (MPS II)
* Leukodystrophies
* Krabbe Disease, also known as globoid cell leukodystrophy
* Metachromatic leukodystrophy (MLD)
* X-linked adrenoleukodystrophy (ALD)
* Other inherited metabolic disorders
* Alpha Mannosidosis
* Gaucher Disease
* Other inheritable metabolic diseases where HSCT may be beneficial
D. Hereditary Anemias
* Thalassemia major
* Sickle cell disease (SCD)
* Diamond Blackfan Anemia (DBA)
E. Inflammatory Conditions
* Crohn's Disease or Inflammatory Bowel Disease
* IPEX or IPEX-like Syndromes
* Rheumatoid Arthritis
* Other inflammatory conditions where HSCT may be beneficial
4. 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.
There are no exclusion criteria.
Where this trial is running
Pittsburgh, Pennsylvania
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (Recruiting)
Study contacts
- Principal investigator: Paul Szabolcs, MD — UPMC Children's Hospital of Pittsburgh
- Study coordinator: Paul Szabolcs, MD
- Email: paul.szabolcs@chp.edu
- Phone: 412-692-5427
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.