Actively Recruiting

Age: 2Months - 60Years
All Genders
NCT04528355

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

Led by Paul Szabolcs · Updated on 2026-01-13

50

Participants Needed

1

Research Sites

410 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 2Months - 60Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient, parent, or legal guardian must have given written informed consent.
  • Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
  • Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to primary immunodeficiency syndromes, congenital bone marrow failure syndromes, inherited metabolic disorders, hereditary anemias, and inflammatory conditions.
  • 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.
Not Eligible

You will not qualify if you...

History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety

AI-Screening

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Trial Site Locations

Total: 1 location

1

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

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Research Team

P

Paul Szabolcs, MD

CONTACT

S

Shawna McIntyre, RN

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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