RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

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

Sponsor: Washington University School of Medicine • ClinicalTrials.gov ID: NCT00920972

Registry updated: 2026-09-16

Recruiting for Similar Conditions?

Sponsors are looking for patients like you. See if you qualify for compensated research studies.

Description

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.

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

Eligibility criteria

Inclusion Criteria: 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 Criteria: * HIV positive * Invasive infection * Pregnancy/lactating

Conditions

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

Locations

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

Partner with Nurenyx

Showcase your study to thousands of motivated patients matching your criteria.

Learn More

Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record

Accelerate Your Clinical Trial Recruitment