RecruitingPhase 1Phase 2NCT03128996

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

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


Sponsor

Washington University School of Medicine

Enrollment

29 participants

Start Date

Mar 20, 2017

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 1 DayMax Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This study offers a bone marrow transplant from a partially matched family donor for children and young adults who have serious non-cancerous conditions that require transplantation — such as sickle cell disease, bone marrow failure syndromes, metabolic disorders, or immune system disorders — and who do not have a fully matched sibling donor available. You may be eligible if: - You are 21 years old or younger - You have a serious non-malignant disorder that requires a bone marrow transplant (e.g., sickle cell disease, aplastic anemia, a metabolic or immune disorder) - You do not have a fully matched sibling donor but do have a partially matched family member willing to donate - Your heart, lung, kidney, and liver function meet minimum requirements - For sickle cell disease: you must have experienced certain severe complications (stroke, recurrent chest syndrome, kidney disease, severe pain episodes, etc.) You may NOT be eligible if: - You have a fully matched sibling donor available - You have liver cirrhosis, active hepatitis, or have recently had an uncontrolled serious infection - You are HIV-positive - You have had a previous stem cell transplant - You are pregnant or breastfeeding - You have active autoimmune disease (e.g., lupus, scleroderma) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGRIC 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

DRUGGVHD 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

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT03128996


Related Trials