Collects data on stem cell transplants for non-cancer blood and immune disorders
Collects data on a reduced-intensity transplant in people with inherited immune, blood, or metabolic disorders.
Plain-language title generated from the registry record
Registry title: Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Key facts
- Where
- Pittsburgh, United StatesSee the site
Where in United States
- UPMC Children's Hospital of PittsburghPittsburgh, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Paul Szabolcs
- Trial code
- NCT04528355
- Record updated
- 13 January 2026
- Original description
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.Read full original descriptionShow less
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 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.
Inclusion: 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 the following:; 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; Congenital Bone Marrow Failure Syndromes; Congenital Amegakaryocytic Thrombocytopenia (CAMT); Osteopetrosis; 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; Hereditary Anemias; Thalassemia major; Sickle cell disease (SCD); Diamond Blackfan Anemia (DBA); Inflammatory Conditions; Crohn's Disease or Inflammatory Bowel Disease; IPEX or IPEX-like Syndromes; Rheumatoid Arthritis; Other inflammatory conditions where HSCT may be beneficial; 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.