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CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by New York Medical College
Sponsor:
Information provided by (Responsible Party):
Mitchell Cairo, New York Medical College
ClinicalTrials.gov Identifier:
NCT01049854
First received: May 5, 2008
Last updated: September 23, 2014
Last verified: September 2014
  Purpose

CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well tolerated and be associated with a low incidence of serious (Grade III/IV) acute and chronic graft versus host disease (GVHD).


Condition Intervention Phase
Leukemia
Lymphoma
Bone Marrow Failure
Immunodeficiencies
Histiocytosis
Drug: Full Intensity with TBI
Drug: Full Intensity
Drug: Reduced Intensity
Drug: Reduced Intensity (Fanconi)
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: CD34+Stem Cell Selection for Patients Receiving Partially Matched Family or Matched Unrelated Adult Donor Allogeneic Stem Cell Transplantations for Malignant and Non-Malignant Disease

Resource links provided by NLM:


Further study details as provided by New York Medical College:

Primary Outcome Measures:
  • To determine the safety, toxicity and feasibility of CD34+ stem cell selection in children, adolescents and young adults receiving a partially matched family or matched unrelated adult donor allogeneic stem cell transplant [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To determine the time to immune reconstitution (T, B, DC) following CD34+ selection in children, adolescents and young adults receiving a partially matched family or matched unrelated adult donor allogeneic stem cell transplant [ Time Frame: 10 years ] [ Designated as safety issue: No ]
  • To establish the incidence of post transplant lymphoproliferative syndrome, (PTLD) following CD34+ selection in children, adolescents and young adults receiving a partially matched family or matched unrelated adult donor allogeneic stem cell transplant. [ Time Frame: 10 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 35
Study Start Date: September 2011
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Thiotepa/Cyclophosphamide/ATG
Full intensity with TBI
Drug: Full Intensity with TBI
Patients will start their pre-conditioning regimen on Day -8. Fractionated TBI will be administered twice daily for 3 days on Days -8, -7, and -6. Patients will receive Thiotepa on Days -5, -4, Cyclophosphamide on Days -3, -2 and rabbit antithymocyte globulin on Days -4, -3, -2 and -1. The stem cell infusion will be performed on Day 0. GM-CSF hematopoietic growth factor will start on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Other Names:
  • ThioTepa
  • Cytoxan
  • Atgam
Experimental: Busulfan/Melphalan/ATG
Full intensity without TBI
Drug: Full Intensity
Patients will start their pre-conditioning regimen on Day -9. Patients will receive busulfan twice daily on Days - 8, -7, -6, and -5 and Melphalan on Days -4, -3 and -2 and rabbit antithymocyte globulin on Days -4, -3, -2 and -1 with stem cell infusion on Day 0. GM-CSF hematopoietic growth factor will start on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Other Names:
  • Myleran
  • Alkeran
  • Atgam
Experimental: Busulfan/Fludarabine/Alemtuzumab
Reduced Intensity Chemotherapy
Drug: Reduced Intensity
Patients will start their GVHD prophylaxis with Tacrolimus on Day -9. Patients will receive busulfan twice daily on Days -8, -7, -6, and -5; fludarabine on Days -7, -6, -5, -4, -3 and -2 and alemtuzumab on Days -5, -4, -3, -2, and -1. The stem cell infusion will be performed on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Other Names:
  • Fludara
  • Myleran
  • Campath
Experimental: Fludarabine/Cyclophosphamide/ATG
Reduced Intensity Chemotherapy for Fanconi Anemia
Drug: Reduced Intensity (Fanconi)
Patients will start their pre-conditioning regimen on Day -6. Patients will receive TBI as a single fraction on Day -6. Patients will receive fludarabine and cyclophosphamide on Days - 5, -4, -3, and -2 and antithymocyte globulin (horse) on Days -5, -4, -3, -2 and -1. The stem cell infusion will be performed on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Other Names:
  • Fludara
  • Cytoxan
  • Atgam

Detailed Description:

The selection of CD34+ cells is associated with the simultaneous depletion of T cells that are responsible for severe acute and chronic graft versus host disease (GVHD). Successful engraftment is reported in adult patients with malignant and non-malignant disease who received CD34+ selected stem cells from HLA-matched or mismatched mobilized peripheral blood (PBSC) or bone marrow.

Study Design:

Selected patients defined in the eligibility criteria will enrolled on this study. Patients will receive one of either full intensity or reduced intensity regimen based on the patient's disease status, organ function and performance and determined by the PI and will have peripheral blood undergo CD34 selection.

  Eligibility

Ages Eligible for Study:   up to 26 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Adequate renal function defined as:Serum creatinine <1.5 x normal, or Creatinine clearance or radioisotope GFR >60 ml/min/m2 or >60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
  • Adequate liver function defined as:Total bilirubin <1.5 x normal, or SGOT (AST) or SGPT (ALT) <3.0 x normal
  • Adequate cardiac function defined as:Shortening fraction >27% by echocardiogram, or Ejection fraction of >47% by radionucleotide angiogram or echocardiogram.
  • Adequate pulmonary function defined as:Uncorrected DLCO >50% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% on room air.

Eligibility for Reduced Intensity Regimen:

  • Adequate renal function defined as:Serum creatinine 2.0 x normal, or creatinine clearance or radioisotope GFR > 40 ml/min/m2 or >40 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
  • Adequate liver function defined as:Total bilirubin < 2.5 x normal; or SGOT (AST) or SGPT (ALT) < 5.0 x normal.
  • Adequate cardiac function defined as:Shortening fraction of >25% by echocardiogram, or Ejection fraction of >40% by radionuclide angiogram or echocardiogram.
  • Adequate pulmonary function defined as:DLCO >35% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% in room air.

Exclusion Criteria:

  • Pregnancy/Breast Feeding: Females who are pregnant or breast-feeding are not eligible.
  • Infection: Patients with documented uncontrolled infection at the time of study entry are not eligible.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01049854

Contacts
Contact: Mitchell S Cairo, MD 914-594-3650 Mitchell_Cairo@nymc.edu
Contact: Lauren Harrison, RN 978-993-4372 lauren_harrison@nymc.edu

Locations
United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Lauren Harrison, RN    978-993-4372    lauren_harrison@nymc.edu   
Sponsors and Collaborators
New York Medical College
Investigators
Principal Investigator: Mitchell S Cairo, MD New York Medical College
  More Information

No publications provided

Responsible Party: Mitchell Cairo, Principal Investigator, New York Medical College
ClinicalTrials.gov Identifier: NCT01049854     History of Changes
Other Study ID Numbers: L 10,321, NYMC 525
Study First Received: May 5, 2008
Last Updated: September 23, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by New York Medical College:
unrelated donor transplant
CD34 selection

Additional relevant MeSH terms:
Histiocytosis, Langerhans-Cell
Histiocytosis
Immunologic Deficiency Syndromes
Pancytopenia
Hematologic Diseases
Immune System Diseases
Lung Diseases
Lung Diseases, Interstitial
Lymphatic Diseases
Respiratory Tract Diseases
Cyclophosphamide
Fludarabine
Fludarabine phosphate
Thiotepa
Alkylating Agents
Antimetabolites
Antimetabolites, Antineoplastic
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 24, 2014