Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Washington University School of Medicine
Study ID
NCT00920972
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Hematologic, Immune, or Bone Marrow Disorders
  • Hemoglobinopathies
  • Metabolic Disorders
  • Non-malignant Disorders

Eligibility Criteria

Sex
ALL
Age
N/A - 20 Years
Healthy Volunteers
Not accepted

Interventions

  • Treatment Plan 1: Stratum 1 — DRUG
    Day -50 to -21: Hydroxyurea 30mg/kg PO q day Day -22: Campath-1H 3 mg IV or SQ... Day -21: Campath-1H 10 mg IV or SQ... Day -20: Campath-1H 15 mg IV or SQ... Day -19: Campath-1H 20 mg IV or SQ... Day -8: Fludarabine 30mg/m2 IV... Day -7: Fludarabine 30mg/m2 IV... Day -6: Fludarabine 30mg/m2 IV... Day -5: Fludarabine 30mg/m2 IV... Day -4: Fludarabine 30mg/m2 IV... Day -3: Melphalan 140 mg/m2 IV... (dose modifications for patients \<10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on Day 0...
  • Treatment Plan 2: Strata 2, 3, or 4 — DRUG
    Day -50 to -21: Hydroxyurea 30mg/kg PO q day… Day -22: Campath-1H 3 mg IV or SQ... Day -21: Campath-1H 10 mg IV or SQ... Day -20: Campath-1H 15 mg IV or SQ... Day -19: Campath-1H 20 mg IV or SQ... Day -8: Fludarabine 30mg/m2 IV... Day -7: Fludarabine 30mg/m2 IV... Day -6: Fludarabine 30mg/m2 IV... Day -5: Fludarabine 30mg/m2 IV... Day -4: Fludarabine 30mg/m2 IV... Day -4: Thiotepa 8mg/kg IV… Day -3: Melphalan 140 mg/m2 IV... (dose modifications for patients \<10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on day 0...
  • GVHD Regimen A: UCB Recipients — DRUG
    Day -3: Begin Tacrolimus or cyclosporine Begin MMF Day -1: Abatacept 10mg/kg IV Day +5: Abatacept 10mg/kg IV Day +14: Abatacept 10mg/kg IV Day +28: Abatacept 10mg/kg IV Day +60: Abatacept 10mg/kg IV Day +100: Abatacept 10mg/kg IV
  • GVHD Regimen B: BM Recipients — DRUG
    Day -3: Begin Tacrolimus or cyclosporine Begin MMF Day -1: Abatacept 10mg/kg IV Day +1: Methotrexate 7.5mg/m2 IV Day +3: Methotrexate 7.5mg/m2 IV Day +5: Abatacept 10mg/kg IV Day +6: Methotrexate 7.5mg/m2 IV Day +14: Abatacept 10mg/kg IV Day +28: Abatacept 10mg/kg IV Day +60: Abatacept 10mg/kg IV Day +100: Abatacept 10mg/kg IV Day +180: Abatacept 10mg/kg IV Day +270: Abatacept 10mg/kg IV Day +365: Abatacept 10mg/kg IV

Study Details

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.

Key Dates

Start date
Dec 31, 2001
Status verified
Mar 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2031

Study Design

Enrollment
220 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Stratum 1
    Recipients with non-malignant disorders, excluding thalassemia. Related or unrelated 8/8 HLA-matched bone marrow
  • Experimental: Stratum 2
    Recipient with transfusion dependent thalassemia. Related or unrelated. 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
  • Experimental: Stratum 3
    Recipient with hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
  • Experimental: Stratum 4
    Recipient with non-malignant disorder, excluding hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB

Primary Outcome Measure

Donor engraftment as measured by chimerism [ Time Frame: 100 days post-transplant ]

Central Contacts

Locations (25)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016
Wilson A Vasconez Samaniego, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of Orange CountyOrangeCalifornia92868
David Buchbinder, MD (PRINCIPAL_INVESTIGATOR)
University of CaliforniaSan DiegoCalifornia92123
Eric Anderson, MD (PRINCIPAL_INVESTIGATOR)
Yale School of MedicineNew HavenConnecticut06510
Lakshmanan Krishnamurti, MD (PRINCIPAL_INVESTIGATOR)
George Washington University School of MedicineWashington D.C.District of Columbia20010
David Jacobsohn, MD (PRINCIPAL_INVESTIGATOR)
Nemours Children's HealthJacksonvilleFlorida32207-
Miami Children's HospitalMiamiFlorida33155-
University of MiamiMiamiFlorida33136
Edward Dela Ziga, MD (PRINCIPAL_INVESTIGATOR)
All Children's HospitalSt. PetersburgFlorida33701-
Children's Memorial HospitalChicagoIllinois60614-
Indiana University School of MedicineIndianapolisIndiana46202
Jodi Skiles, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of New OrleansNew OrleansLouisiana70118-
Children's MercyKansas CityMissouri64108-
St. Louis UniversitySt LouisMissouri63104
Deepika Bhatla, MD
Deepika Bhatla, MD (PRINCIPAL_INVESTIGATOR)
Washington University School of Medicine (in St. Louis)St LouisMissouri63110
Shalini Shenoy, MD
[email protected]
3144546018
Lissy L Keller, CCRP, BS
[email protected]
3142861168
Hackensack University Medical CenterHackensackNew Jersey07601-
Columbia University Medical CenterNew YorkNew York10032
Monica Bhatia, MD
Monica Bhatia, MD (PRINCIPAL_INVESTIGATOR)
University of North CarolinaChapel HillNorth Carolina27514
Andrew Gilman, MD (PRINCIPAL_INVESTIGATOR)
Carolinas Medical CenterCharlotteNorth Carolina28232-
Duke Children's HospitalDurhamNorth Carolina27705-
The University of OklahomaOklahoma CityOklahoma73104-
University of PittsburgPittsburghPennsylvania15224-
Cook Children's HospitalForth WorthTexas76104-
Texas Transplant InstituteSan AntonioTexas78229-
University of UtahSalt Lake CityUtah84132
Michael Pulsipher, MD (PRINCIPAL_INVESTIGATOR)

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Phoenix Children's Hospital· Phoenix, AZChildren's Hospital of Orange County· Orange, CAUniversity of California· San Diego, CAYale School of Medicine· New Haven, CTGeorge Washington University School of Medicine· Washington D.C., DCNemours Children's Health· Jacksonville, FL

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