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

Part of paid clinical trials in New Haven, Connecticut.

Sponsor
Washington University School of Medicine
Study ID
NCT03128996
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Bone Marrow Failure Syndromes
  • Hemoglobinopathies
  • Immunologic Disorders
  • Metabolic Disorders
  • Non-malignant Disorders
  • Severe Sickle Cell Disease

Eligibility Criteria

Sex
ALL
Age
1 Day - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • RIC regimen — DRUG
    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
  • GVHD prophylaxis regimen — DRUG
    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)

Study Details

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.

Key Dates

Start date
Mar 20, 2017
Status verified
May 2026
Primary completion
Apr 30, 2028
Completion
Apr 30, 2033

Study Design

Enrollment
29 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: RIC Prep Regimen & GVHD Prophylaxis
    Single arm study. All patients receive the same Reduced Intensity Conditioning (RIC) regimen and GVHD prophylaxis regimen

Primary Outcome Measure

Donor engraftment [ Time Frame: 100 days and 1 year post-transplant ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Yale School of MedicineNew HavenConnecticut06510
Lakshmanan Krishnamurti, MD
Lakshmanan Krishnamurti, MD (PRINCIPAL_INVESTIGATOR)
Nemours Children's HealthWilmingtonDelaware19803
Emi Caywood, MD
800-416-4441
Emi Caywood, MD (PRINCIPAL_INVESTIGATOR)
Helen DeVos Children's HospitalGrand RapidsMichigan49503
Troy Quigg, DO
Troy Quigg, DO (PRINCIPAL_INVESTIGATOR)
Washington University School of MedicineSt LouisMissouri63110
Shalini Shenoy, MD
[email protected]
314-454-6018
Ian Snyder, BS, CCRP
[email protected]
314-273-5953
Shalini Shenoy, MD (PRINCIPAL_INVESTIGATOR)

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Yale School of Medicine· New Haven, CTNemours Children's Health· Wilmington, DEHelen DeVos Children's Hospital· Grand Rapids, MIWashington University School of Medicine· St Louis, MO

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