NCT01962415View on ClinicalTrials.gov

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

PHASE2RecruitingINTERVENTIONAL
Enrollment

100

Participants

Timeline

Start Date

February 4, 2014

Primary Completion Date

November 30, 2025

Study Completion Date

November 30, 2026

Conditions
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory ConditionsSystemic Juvenile Idiopathic Arthritis (sJIA)Juvenile Rheumatoid Arthritis (JRA)
Interventions
DRUG

Hydroxyurea

Oral administration

DRUG

Alemtuzumab

Intravenous (IV) administration.

DRUG

Fludarabine

IV administration

DRUG

Melphalan

IV administration

DRUG

Thiotepa

IV administration

Trial Locations (1)

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

All Listed Sponsors
lead

Paul Szabolcs

OTHER