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

RecruitingOBSERVATIONAL
Enrollment

50

Participants

Timeline

Start Date

August 20, 2020

Primary Completion Date

December 31, 2025

Study Completion Date

June 30, 2026

Conditions
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory 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.

Trial Locations (1)

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

All Listed Sponsors
lead

Paul Szabolcs

OTHER