Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT ID
NCT04528355
Registry status
Recruiting
Study type
Observational
Sponsor
Paul Szabolcs
Conditions
Congenital Bone Marrow Failure Syndromes, Hereditary Anemias, Inflammatory Conditions, Inherited Metabolic Disorders (IMD), Primary Immunodeficiency (PID)

Overview

ClinicalTrials.gov lists this study as Recruiting.

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)

Enrollment: 50 participants (Estimated).

What is being studied

  • Drug: data collection
  • Intervention: Data Collection
  • Intervention: Environment and Public Health
  • Intervention: Epidemiologic Methods
  • Intervention: Health Care Evaluation Mechanisms
  • Intervention: Health Care Quality, Access, and Evaluation
  • Intervention: Investigative Techniques
  • Intervention: Public Health
  • Intervention: Quality of Health Care

Who may be eligible

Ages
2 Months to 60 Years
Sex
All
Healthy volunteers accepted
No

ClinicalTrials.gov lists the following eligibility 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.

Study locations

1 study location is listed.

Recruiting site status is explicitly provided for 1 location; 1 is listed as Recruiting.

Countries: United States

  • Pennsylvania, United States: 1

Sample of listed locations

  • UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, 15224, United States — Recruiting

Study details

Start date
August 20, 2020
Primary completion date
December 31, 2027
Completion date
June 30, 2028
First posted
August 27, 2020

Source and update information

Source
ClinicalTrials.gov
NCT ID
NCT04528355
Registry last updated
January 13, 2026
QualifiedStudies registry data refreshed
October 2, 2026
QualifiedStudies record fetched
October 2, 2026

View NCT04528355 on ClinicalTrials.gov

Trial information can change. Confirm current study details with the study team or the registry.

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