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

We're studying treatments for non-cancer conditions using different stem cell transplants.

Recruiting
2-60 years
All
Phase N/A

This study is about collecting information from patients with non-cancer disorders who are undergoing a type of treatment called hematopoietic stem cell transplantation (HSCT). This treatment uses healthy donor cells to help cure or improve certain diseases. The study focuses on a specific way of preparing patients for this treatment, called reduced-intensity conditioning (RIC), which uses less intense chemotherapy to make the body ready for the new cells. The aim is to find out how well the treatment works and how safe it is.

To join, you must be between 2 months and 60 years old and have a disorder that can be treated with stem cell transplantation. These disorders include certain immune system problems, bone marrow failures, metabolic disorders, anemias, and inflammatory conditions. During the study, patients receive a transplant from umbilical cord blood, bone marrow, or peripheral blood stem cells, along with medicines to help the process.

  • The study takes place at UPMC Children's Hospital of Pittsburgh.
  • There are no specific reasons to be excluded from the study.
  • Participants will help researchers learn more about this treatment's success and safety.
Study details
    Primary Immunodeficiency (PID)
    Congenital Bone Marrow Failure Syndromes
    Inherited Metabolic Disorders (IMD)
    Hereditary Anemias
    Inflammatory Conditions

NCT04528355

Paul Szabolcs

17 April 2026

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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