Back to Trials
NCT00920972PHASE1, PHASE2Recruiting

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

Washington University School of Medicine

Start Date

12/1/2001

Completion Date

12/1/2031

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.

Detailed Description

The study uses reduced intensity conditioning that is immune suppressive to achieve donor cell engraftment without exposure to radiation or high dose chemotherapy in children with non-malignant disorders. The intent is to minimize early and late regimen related toxicities in the context of a reduced intensity regimen. In addition to maximizing opportunity for donor cell engraftment, the trial seeks to minimize toxicities associated with transplant such as graft versus host disease and employs GVHD prophylaxis that seeks to decrease rates of acute and chronic GVHD in the setting of matched and mismatched donor stem cell transplants from marrow and cord blood sources.

Eligibility Criteria

Age Range: No minimum to 20 years

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

Interventions

DRUG

Treatment Plan 1: Stratum 1

DRUG

Treatment Plan 2: Strata 2, 3, or 4

DRUG

GVHD Regimen A: UCB Recipients

DRUG

GVHD Regimen B: BM Recipients

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

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

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Children's Hospital of Orange County

Orange, California 92868

United States

University of California

San Diego, California 92123

United States

Yale School of Medicine

New Haven, Connecticut 06510

United States

George Washington University School of Medicine

Washington D.C., District of Columbia 20010

United States

Nemours Children's Health

Jacksonville, Florida 32207

United States

University of Miami

Miami, Florida 33136

United States

Miami Children's Hospital

Miami, Florida 33155

United States

All Children's Hospital

St. Petersburg, Florida 33701

United States

Children's Memorial Hospital

Chicago, Illinois 60614

United States

Indiana University School of Medicine

Indianapolis, Indiana 46202

United States

Children's Hospital of New Orleans

New Orleans, Louisiana 70118

United States

Children's Mercy

Kansas City, Missouri 64108

United States

St. Louis University

St Louis, Missouri 63104

United States

Washington University School of Medicine (in St. Louis)

St Louis, Missouri 63110

United States

Hackensack University Medical Center

Hackensack, New Jersey 07601

United States

Columbia University Medical Center

New York, New York 10032

United States

University of North Carolina

Chapel Hill, North Carolina 27514

United States

Carolinas Medical Center

Charlotte, North Carolina 28232

United States

Duke Children's Hospital

Durham, North Carolina 27705

United States

The University of Oklahoma

Oklahoma City, Oklahoma 73104

United States

University of Pittsburg

Pittsburgh, Pennsylvania 15224

United States

Cook Children's Hospital

Forth Worth, Texas 76104

United States

Texas Transplant Institute

San Antonio, Texas 78229

United States

University of Utah

Salt Lake City, Utah 84132

United States

BC Children's Hospital

Vancouver, British Columbia V6H 3N1

Canada

University of Manitoba

Winnipeg, Manitoba MB R3E 0T4

Canada

University of Calgary

Calgary,

Canada