Status:

RECRUITING

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

Lead Sponsor:

Paul Szabolcs

Conditions:

Primary Immunodeficiency (PID)

Congenital Bone Marrow Failure Syndromes

Eligibility:

All Genders

2-60 years

Brief 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 strat...

Detailed Description

Hematopoietic stem cell transplantation (HSCT) from a healthy donor can cure or alleviate a broad spectrum of non-malignant disorders (NMD). Although reduced-intensity conditioning (RIC) regimens prom...

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.

Key Trial Info

Start Date :

August 20 2020

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

June 30 2028

Estimated Enrollment :

50 Patients enrolled

Trial Details

Trial ID

NCT04528355

Start Date

August 20 2020

End Date

June 30 2028

Last Update

January 13 2026

Active Locations (1)

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UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224