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RECRUITING
NCT03128996
PHASE1/PHASE2

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Sponsor: Washington University School of Medicine

View on ClinicalTrials.gov

Summary

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

Official title: A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders

Key Details

Gender

All

Age Range

1 Day - 21 Years

Study Type

INTERVENTIONAL

Enrollment

29

Start Date

2017-03-20

Completion Date

2033-04

Last Updated

2026-01-07

Healthy Volunteers

No

Interventions

DRUG

RIC regimen

Days -60 to -21: hydroxyurea (30mg/kg/day po) \>6hrs prior to 1st dose: alemtuzumab (3mg IV) Day -21: alemtuzumab (10mg IV or S/C) Day -20: alemtuzumab (15mg IV or S/C) (10mg if \< 10kg) Day -19: alemtuzumab (20mg IV or S/C) (10mg if \< 10kg) Days -8 to -4: fludarabine (30mg/m2/day IV) Day -4: thiotepa (8mg/kg IV) Day -3: melphalan (140mg/m2) Days -2 to -1: rest days/no therapy Day 0: bone marrow transplant

DRUG

GVHD prophylaxis regimen

Day +3 to +4: cyclophosphamide (50mg/kg/day IV) Day +5: Start of tacrolimus \& Start of mycophenolate mofetil (MMF) Days +5, +14, +30, +60, +90: abatacept (IND) (10mg/kg/day IV) Day +90: rituximab (375mg/m2 IV once) Patients \>/= 12 yrs - Days +120 to +180: abatacept (IND) monthly (10mg/kg/day IV) Patients \>/= 12 yrs - Days +210 to +390: abatacept (IND) monthly (5mg/kg/day) Patients \<12 yrs - Days +120 to +390: abatacept (IND) monthly (5mg/kg/day IV)

Locations (4)

Yale School of Medicine

New Haven, Connecticut, United States

Nemours Children's Health

Wilmington, Delaware, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Washington University School of Medicine

St Louis, Missouri, United States