Data collection for patients with non-malignant disorders undergoing stem cell transplantation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study collects data on patients with non-malignant disorders who are undergoing hematopoietic stem cell transplantation (HSCT) using a reduced-intensity chemotherapy regimen. The focus is on understanding the effectiveness of a novel conditioning regimen that includes alemtuzumab and other agents to prevent graft failure and support immune reconstitution. By gathering clinical data, the study aims to assess outcomes such as engraftment, graft-versus-host disease (GVHD), immunosuppressant use, and overall survival in this patient population. The findings may also provide insights for future gene therapy strategies.

Why it matters

Who can join: the full rules

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.

Where it's running

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

Study contacts

• Principal investigator: Paul Szabolcs, MD — UPMC Children's Hospital of Pittsburgh
• Study coordinator: Paul Szabolcs, MD
• Email: paul.szabolcs@chp.edu
• Phone: 412-692-5427

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.