RecruitingNCT04528355

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

A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)


Sponsor

Paul Szabolcs

Enrollment

50 participants

Start Date

Aug 20, 2020

Study Type

OBSERVATIONAL

Conditions

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 strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.


Eligibility

Min Age: 2 MonthsMax Age: 60 Years

Plain Language Summary

Simplified for easier understanding

This study is collecting data on outcomes for patients with non-cancerous (non-malignant) blood and immune disorders who undergo stem cell transplantation, to help improve transplant techniques and care for these rare conditions. **You may be eligible if...** - You are between 2 months and 60 years old - You have a non-malignant disorder that can be treated with stem cell transplantation — such as severe combined immunodeficiency (SCID), sickle cell disease, thalassemia, bone marrow failure syndromes, or metabolic storage diseases - You or your parent/guardian has given written consent **You may NOT be eligible if...** - You have a malignant (cancerous) condition - You do not meet the transplant criteria for your specific diagnosis - You are outside the age range of 2 months to 60 years Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGdata collection

Study subjects will receive alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen in accordance with clinical practice at UPMC Children's Hospital of Pittsburgh at the discretion of the treating physician. Medical data will be abstracted from subject's medical charts once the patient signs the informed consent.


Locations(1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

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NCT04528355


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