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

NCT04528355 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-01-13

No results posted yet for this study

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.

Conditions

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

Interventions

DRUG

data 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.

Sponsors & Collaborators

  • Paul Szabolcs

    lead OTHER

Principal Investigators

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

Eligibility

Min Age
2 Months
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-20
Primary Completion
2027-12-31
Completion
2028-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04528355 on ClinicalTrials.gov