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

NCT00920972 · tracked via the Priya Life Science Canada tracker

Sponsor

Washington University School of Medicine

Phase

Phase 1/2

Started

2001-12

Last updated

2026-09-16

Condition(s) studied

Metabolic DisordersHematologic, Immune, or Bone Marrow DisordersHemoglobinopathiesNon-malignant Disorders

Investigational drug(s) / intervention(s)

Treatment Plan 1: Stratum 1Treatment Plan 2: Strata 2, 3, or 4GVHD Regimen A: UCB RecipientsGVHD Regimen B: BM Recipients

Treatment 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...

Treatment 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...

GVHD 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

GVHD 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

Study 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

Sex

ALL

Min age

—

Max age

20 Years

Healthy volunteers

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

Primary outcome measure(s)

Donor engraftment as measured by chimerism — 100 days post-transplant
Engraftment is measured in myeloid and lymphoid lineage cells
Major toxicities as graded by the CTC v4 — 100 days post-transplant
Toxicity monitoring includes unanticipated side effects (new) and all severe irreversible toxicities Grade 3 and above unexpected Grade 4 and above - all toxicities that are possibly, probably or definitely related to protocol therapy All deaths irrespective of attribution

Trial sites (28)

Facility	City	Region	Status
Phoenix Children's Hospital	Phoenix	Arizona	Active Not Recruiting
Children's Hospital of Orange County	Orange	California	Active Not Recruiting
University of California	San Diego	California	Active Not Recruiting
Yale School of Medicine	New Haven	Connecticut	Active Not Recruiting
George Washington University School of Medicine	Washington D.C.	District of Columbia	Active Not Recruiting
Nemours Children's Health	Jacksonville	Florida	Completed
University of Miami	Miami	Florida	Active Not Recruiting
Miami Children's Hospital	Miami	Florida	Completed
All Children's Hospital	St. Petersburg	Florida	Completed
Children's Memorial Hospital	Chicago	Illinois	Completed
Indiana University School of Medicine	Indianapolis	Indiana	Active Not Recruiting
Children's Hospital of New Orleans	New Orleans	Louisiana	Completed
Children's Mercy	Kansas City	Missouri	Completed
St. Louis University	St Louis	Missouri	Active Not Recruiting
Washington University School of Medicine (in St. Louis)	St Louis	Missouri	Recruiting
Hackensack University Medical Center	Hackensack	New Jersey	Completed
Columbia University Medical Center	New York	New York	Active Not Recruiting
University of North Carolina	Chapel Hill	North Carolina	Active Not Recruiting
Carolinas Medical Center	Charlotte	North Carolina	Completed
Duke Children's Hospital	Durham	North Carolina	Completed
The University of Oklahoma	Oklahoma City	Oklahoma	Completed
University of Pittsburg	Pittsburgh	Pennsylvania	Completed
Cook Children's Hospital	Forth Worth	Texas	Completed
Texas Transplant Institute	San Antonio	Texas	Completed
University of Utah	Salt Lake City	Utah	Active Not Recruiting
BC Children's Hospital	Vancouver	British Columbia	Completed
University of Manitoba	Winnipeg	Manitoba	Completed
University of Calgary	Calgary	Canada	Completed

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Other trials for the same condition

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) Active, not recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 Enrolling by invitation

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT00920972 on ClinicalTrials.gov ↗ ← All trials in Canada