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

This study is currently recruiting participants.
Verified January 2013 by Washington University School of Medicine
Sponsor:
Collaborator:
St. Louis Children's Hospital
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT00920972
First received: June 15, 2009
Last updated: January 14, 2013
Last verified: January 2013
History of Changes
  Purpose

The purpose of this study is to determine if treatment with alemtuzumab, fludarabine and melphalan followed by related/unrelated bone marrow, peripheral blood stem cell, or umbilical cord blood cell transplant will result in donor engraftment on day +100. It is also to determine major toxicities from this conditioning regimen, within the first 100 days after transplantation.


Condition Intervention Phase
Metabolic Disorders
Hematologic, Immune, or Bone Marrow Disorders
Hemoglobinopathies
 Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Donor engraftment as measured by chimerism [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
  • Major toxicities as graded by the CTC v3 [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Time to neutrophil and platelet engraftment as measured by complete blood counts [ Time Frame: Notapplicable ] [ Designated as safety issue: Yes ]
  • Incidence of acute graft-versus-host disease as measured by protocol grading scale [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
  • Incidence of chronic graft-versus-host disease as measured by protocol grading scale [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
  • Long-term donor engraftment by donor chimerism [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
  • Immune reconstitution by laboratory evaluations [ Time Frame: 1 year post-transplant ] [ Designated as safety issue: Yes ]
  • Overall and disease free survival [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 183
Study Start Date: December 2001
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Matched related HSCT for hemoglobinopathies Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

Day -22 Campath-1H 3 mg IV... Day -21 Campath-1H 10 mg IV... Day -20 Campath-1H 15 mg IV... Day -19 Campath-1H 20 mg IV... 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

Bone marrow or peripheral blood stem cell infusion on Day 0

Other Names:
  • Alemtuzumab
  • Campath
Experimental: Unrelated HSCT for thalassemia Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IV... Day -21 Campath-1H 10 mg IV... Day -20 Campath-1H 15 mg IV... Day -19 Campath-1H 20 mg IV... 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

Bone marrow or umbilical cord blood stem cell infusion on Day 0

Other Names:
  • Alemtuzumab
  • Campath
Experimental: Minimally mismatched unrelated HSCT for hemoglobinopathies Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IV... Day -21 Campath-1H 10 mg IV... Day -20 Campath-1H 15 mg IV... Day -19 Campath-1H 20 mg IV... 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

Bone marrow or umbilical cord blood stem cell infusion on Day 0

Other Names:
  • Alemtuzumab
  • Campath
Experimental: Minor mismatched unrelated HSCT for non-malignant disorders Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IV... Day -21 Campath-1H 10 mg IV... Day -20 Campath-1H 15 mg IV... Day -19 Campath-1H 20 mg IV... 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

Bone marrow or umbilical cord blood stem cell infusion on Day 0

Other Names:
  • Alemtuzumab
  • Campath

  Eligibility

Ages Eligible for Study:   up to 20 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Stratum 1: Patient must have a hemoglobinopathy receiving a matched related transplant with bone marrow or peripheral blood stem cells.

Stratum 2: Patient must have thalassemia receiving an unrelated donor transplant with bone marrow or umbilical cord blood stem cells.

Stratum 3: Patient must have a hemoglobinopathy receiving a minimally mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving a minor mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00920972

Contacts
Contact: Lisa Murray, MA, CCRP 3144544240 Murray_L@kids.wustl.edu

Locations
United States, Arizona
Phoenix Children's Hospital Recruiting
Phoenix, Arizona, United States, 85016
Principal Investigator: Roberta Adams, MD            
United States, California
University of California Recruiting
San Diego, California, United States, 92123
Principal Investigator: Eric Anderson, MD            
United States, District of Columbia
George Washington University School of Medicine Recruiting
Washington DC, District of Columbia, United States, 20010
Principal Investigator: David Jacobsohn, MD            
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: John A Fort, MD            
Principal Investigator: Guillermo De Angulo, MD            
University of Miami Recruiting
Miami, Florida, United States, 33136
Principal Investigator: Martin Andreansky, MD            
All Children's Hospital Recruiting
St. Petersburg, Florida, United States, 33701
Principal Investigator: Gregory Hale, MD            
United States, Illinois
Children's Memorial Hospital Recruiting
Chicago, Illinois, United States, 60614
Principal Investigator: Sonali Chaudhury, MD            
United States, Indiana
Indiana University School of Medicine Recruiting
Indianapolis, Indiana, United States, 46202
Principal Investigator: Paul Haut, MD            
United States, Louisiana
Louisiana State University Recruiting
New Orleans, Louisiana, United States, 70112
Principal Investigator: Lolie Yu, MD            
United States, Missouri
Children's Mercy Hospitals and Clinics Recruiting
Kansas City, Missouri, United States, 64108
Principal Investigator: Jignesh Dalal, MD            
Washington University School of Medicine (in St. Louis) Recruiting
St. Louis, Missouri, United States, 63110
Contact: Shalini Shenoy, MD     314-454-6018     shenoy@wustl.edu    
Contact: Lisa Murray, MA, CCRP     3144544240     Murray_L@kids.wustl.edu    
United States, New Jersey
Hackensack University Medical Center Recruiting
Hackensack, New Jersey, United States, 07601
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Monica Bhatia, MD            
Principal Investigator: Monica Bhatia, MD            
United States, North Carolina
Carolinas Medical Center Recruiting
Charlotte, North Carolina, United States, 28232
Principal Investigator: Andrew Gilman, MD            
United States, Texas
Cook Children's Health Care System Recruiting
Fort Worth, Texas, United States, 76104
Principal Investigator: Richard P Howrey, MD            
Texas Transplant Institute Recruiting
San Antonio, Texas, United States, 78229
Principal Investigator: Ka-Wah Chan, MD            
United States, Utah
Primary Children's Medical Center Recruiting
Salt Lake City, Utah, United States, 84113
Principal Investigator: Michael Pulsipher, MD            
Canada, British Columbia
British Columbia Children's Hospital Recruiting
Vancouver, British Columbia, Canada, V6H3V4
Principal Investigator: Jeffrey Davis, MD            
Sponsors and Collaborators
Washington University School of Medicine
St. Louis Children's Hospital
Investigators
Principal Investigator: Shalini Shenoy, MD Washington University School of Medicine (in St. Louis)
  More Information

Publications:

Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT00920972     History of Changes
Other Study ID Numbers: 01-0923
Study First Received: June 15, 2009
Last Updated: January 14, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Washington University School of Medicine:
Bone marrow
Transplant
Transplantation
Hematopoietic
Umbilical cord
Related
 Unrelated
Reduced
Non-myeloablative
Nonmyeloablative
Non-malignant
Nonmalignant

Additional relevant MeSH terms:
Bone Marrow Diseases
Hemoglobinopathies
Metabolic Diseases
Hematologic Diseases
Genetic Diseases, Inborn
Fludarabine
Fludarabine monophosphate
Alemtuzumab
Hydroxyurea
Melphalan
Thiotepa
Campath 1G
Vidarabine
Antineoplastic Agents
Therapeutic Uses
 Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Antimetabolites, Antineoplastic
Antimetabolites
Antiviral Agents

ClinicalTrials.gov processed this record on May 22, 2013