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RECRUITING

NCT04528355

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

Sponsor: Paul Szabolcs

View on ClinicalTrials.gov

Summary

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.

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)

Key Details

Gender

All

Age Range

2 Months - 60 Years

Study Type

OBSERVATIONAL

Enrollment

Start Date

2020-08-20

Completion Date

2028-06-30

Last Updated

2026-01-13

Healthy Volunteers

Conditions

Primary Immunodeficiency (PID)Congenital Bone Marrow Failure Syndromes Inherited Metabolic Disorders (IMD)Hereditary Anemias Inflammatory Conditions

Interventions

DRUG

data collection

Study subjects will receive alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen in accordance with clinical practice at UPMC Children's Hospital of Pittsburgh at the discretion of the treating physician. Medical data will be abstracted from subject's medical charts once the patient signs the informed consent.

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.

Locations (1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States