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

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.

Conditions

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

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Study Overview

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Study Details

Study overview

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.

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)

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

Condition
Primary Immunodeficiency (PID)
Intervention / Treatment

-

Contacts and Locations

Pittsburgh

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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:
  • * 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 Amegakaryocytic Thrombocytopenia (CAMT)
  • * Osteopetrosis
  • * 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
  • * Thalassemia major
  • * Sickle cell disease (SCD)
  • * Diamond Blackfan Anemia (DBA)
  • * 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.

Ages Eligible for Study

2 Months to 60 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Paul Szabolcs,

Paul Szabolcs, MD, PRINCIPAL_INVESTIGATOR, UPMC Children's Hospital of Pittsburgh

Study Record Dates

2026-06-30