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Milder chemo before stem cell transplant shows promise for rare blood disorders

NCT ID NCT04528355

Recruiting now Disease control Sponsor: Paul Szabolcs Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host disease. Researchers will collect medical data from participants' charts over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

stem cell transplant (umbilical cord blood, bone marrow, or peripheral blood stem cells) with reduced-intensity chemotherapy (alemtuzumab, melphalan, thiotepa, fludarabine, hydroxyurea)

What this could lead to

If successful, this could improve survival and reduce severe graft-versus-host disease for patients with non-malignant disorders needing a stem cell transplant.

What could go wrong

This is an early-stage, single-center study with only 50 participants, so results may not apply broadly. Risks include graft failure, infection, and side effects from chemotherapy.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

adrenoleukodystrophy alpha-mannosidosis autoimmune lymphoproliferative syndrome beta thalassemia Chediak-Higashi syndrome chronic granulomatous disease congenital amegakaryocytic thrombocytopenia 1 Congenital Bone Marrow Failure Syndromes Crohn disease Diamond-Blackfan anemia Gaucher disease hereditary anemia hyper-IgM syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome inborn error of immunity inborn errors of metabolism inflammatory bowel disease Krabbe disease leukocyte adhesion deficiency 1 Lymphohistiocytosis, Hemophagocytic metachromatic leukodystrophy Mucopolysaccharidosis I osteopetrosis Primary Immunodeficiency Diseases rheumatoid arthritis severe combined immunodeficiency sickle cell disease Sudden Infant Death syndromic disease Wiskott-Aldrich syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CONGENITAL BONE MARROW FAILURE SYNDROMES HEREDITARY ANEMIAS INFLAMMATORY CONDITIONS INHERITED METABOLIC DISORDERS (IMD) PRIMARY IMMUNODEFICIENCY (PID)

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

    Contact

    Contact

    Contact

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••