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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.

Key facts

Who runs it
Paul Szabolcs
Where
Pittsburgh, United StatesSee the site
Trial code
Registry title
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Status
Recruiting
People taking part
50 people, planned
Started
20 Aug 2020
Expected to end
30 Jun 2028
Type of study
Observational
Treatments tested
  • data collection
Record updated
13 January 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children and adults with a non-cancer disease that stem cell transplantation can treat. They get a transplant with a reduced-intensity conditioning regimen at UPMC Children's Hospital of Pittsburgh.

Age
2 months to 60 years
Sex
All
Disease stage
A non-malignant (non-cancer) disorder that stem cell transplantation can treat, such as primary immune deficiency, inherited metabolic disorder, hereditary anemia, bone marrow failure syndrome or inflammatory condition.
Earlier treatment
Must receive a 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.

The registry criteria, word for word

Show all 4 criteriaShow less

Who can take part

  • 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

Where in United States

  • UPMC Children's Hospital of PittsburghPittsburgh, United StatesContact: Shawna McIntyre, RNTaking people

About the trial

What the trial wants to find out

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.

Treatments tested

  • data collection

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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)
Plain-language title
Collects data on stem cell transplants for non-cancer blood and immune disordersGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
13 January 2026