RecruitingPhase 1Phase 2NCT00920972

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

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime


Sponsor

Washington University School of Medicine

Enrollment

220 participants

Start Date

Dec 1, 2001

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Max Age: 20 Years

Inclusion Criteria10

  • Stratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8/8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.
  • Stratum 3: Patient must have a hemoglobinopathy receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.
  • Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.
  • All strata:
  • Recipient age \< 21 years
  • Lansky/Karnofsky \>/= 40
  • Adequate pulmonary, renal, liver, and other organ function as defined in protocol
  • Negative pregnancy test
  • Adequate total nucleated cell or CD34+ dose of product as defined in protocol
  • If sickle cell, Hemoglobin S \<30%

Exclusion Criteria3

  • HIV positive
  • Invasive infection
  • Pregnancy/lactating

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Interventions

DRUGTreatment Plan 1: Stratum 1

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...

DRUGTreatment Plan 2: Strata 2, 3, or 4

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...

DRUGGVHD Regimen A: UCB Recipients

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

DRUGGVHD Regimen B: BM Recipients

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


Locations(28)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Children's Hospital of Orange County

Orange, California, United States

University of California

San Diego, California, United States

Yale School of Medicine

New Haven, Connecticut, United States

George Washington University School of Medicine

Washington D.C., District of Columbia, United States

Nemours Children's Health

Jacksonville, Florida, United States

University of Miami

Miami, Florida, United States

Miami Children's Hospital

Miami, Florida, United States

All Children's Hospital

St. Petersburg, Florida, United States

Children's Memorial Hospital

Chicago, Illinois, United States

Indiana University School of Medicine

Indianapolis, Indiana, United States

Children's Hospital of New Orleans

New Orleans, Louisiana, United States

Children's Mercy

Kansas City, Missouri, United States

St. Louis University

St Louis, Missouri, United States

Washington University School of Medicine (in St. Louis)

St Louis, Missouri, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Columbia University Medical Center

New York, New York, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Carolinas Medical Center

Charlotte, North Carolina, United States

Duke Children's Hospital

Durham, North Carolina, United States

The University of Oklahoma

Oklahoma City, Oklahoma, United States

University of Pittsburg

Pittsburgh, Pennsylvania, United States

Cook Children's Hospital

Forth Worth, Texas, United States

Texas Transplant Institute

San Antonio, Texas, United States

University of Utah

Salt Lake City, Utah, United States

BC Children's Hospital

Vancouver, British Columbia, Canada

University of Manitoba

Winnipeg, Manitoba, Canada

University of Calgary

Calgary, Canada

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