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Data Collection for Non-Malignant Disorders Treated with Stem Cell Transplant

This study is collecting information about patients with non-cancerous conditions like primary immunodeficiency (problems with the immune system), inherited metabolic disorders, or hereditary anemias, who are receiving a stem cell transplant. Researchers are looking at how well a specific reduced-intensity chemotherapy regimen works. This regimen includes alemtuzumab, melphalan, thiotepa, fludarabine, and hydroxyurea. The study aims to understand how often acute graft versus host disease (when donor cells attack the patient's body) occurs and how long patients survive after their transplant, for up to 5 years. You may be able to join if you are between 2 months and 60 years old and have one of these non-cancerous conditions.

Study design
This is an observational study collecting data from about 50 participants. It is not a randomized trial, meaning treatments are not assigned by chance.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 5 years to track outcomes like graft versus host disease and overall survival.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04528355

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

Recruiting
Not specifiedAges 2–60Observational
Paul Szabolcs
~50 participants
Updated 2026-01-13 on ClinicalTrials.gov
What's tested:data collection

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
incidence of acute graft versus host disease (GVHD)
Measured over up to 5 years
+1 more outcome measured

Conditions

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

Explore related trials

AdrenoleukodystrophyAlpha-MannosidosisAutoimmune Lymphoproliferative SyndromeBare Lymphocyte SyndromeBeta Thalassemia MajorChediak-Higashi SyndromeChronic Granulomatous DiseaseCommon Variable ImmunodeficiencyCongenital Amegakaryocytic ThrombocytopeniaCrohn DiseaseDiamond-Blackfan AnemiaGaucher DiseaseHemophagocytic LymphohistiocytosisHunter SyndromeHurler SyndromeHyperimmunoglobulin M SyndromeImmune Dysregulation, Polyendocrinopathy, and Enteropathy X-Linked SyndromeInflammatory Bowel DiseaseKrabbe DiseaseLeukocyte Adhesion DeficiencyLymphocyte Signaling defectsMetachromatic LeukodystrophyOmenn SyndromeOsteopetrosisRheumatoid ArthritisSevere Combined ImmunodeficiencySickle Cell DiseaseWiskott-Aldrich Syndrome

Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

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

Who to contact

Paul Szabolcs, MD
[email protected]
412-692-5427
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Eligibility criteria

Inclusion

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.

What this trial measures

  • incidence of acute graft versus host disease (GVHD)up to 5 years

    grades 3-4, chronic extensive GVHD

  • overall survival after HSCTup to 5 years

    review of the existing medical records to check on the participant's survival status