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Human Placental-Derived Stem Cell Transplantation (HPDSC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2016 by New York Medical College
Sponsor:
Information provided by (Responsible Party):
New York Medical College
ClinicalTrials.gov Identifier:
NCT01586455
First received: April 25, 2012
Last updated: October 4, 2016
Last verified: July 2016
  Purpose
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.

Condition Intervention Phase
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Adrenoleukodystrophy
Niemann-Pick Disease
Metachromatic Leukodystrophy
Wolman Disease
Krabbe's Disease
Gaucher's Disease
Fucosidosis
Batten Disease
Severe Aplastic Anemia
Diamond-Blackfan Anemia
Amegakaryocytic Thrombocytopenia
Myelodysplastic Syndrome
Acute Myelogenous Leukemia
Acute Lymphocytic Leukemia
Drug: Human Placental Derived Stem Cell
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders

Resource links provided by NLM:


Further study details as provided by New York Medical College:

Primary Outcome Measures:
  • Safety [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]
    to evaluate the safety of human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells in patients with malignant and non-malignant diseases.


Secondary Outcome Measures:
  • donor chimerism [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    donor chimerism will be assessed at set timepoints

  • engraftment [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Survival [ Time Frame: 100 days and 180 days ] [ Designated as safety issue: No ]
  • Relapse [ Time Frame: 100 days and 180 days ] [ Designated as safety issue: No ]
  • Mortality [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: April 2013
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group A
related cord blood with ≥3/6 HLA match to the patient and related HPDSC
Drug: Human Placental Derived Stem Cell
Infusions of thawed HPDSC to be given following UCB infusion.
Other Name: HPDSC
Experimental: Group B
unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC
Drug: Human Placental Derived Stem Cell
Infusions of thawed HPDSC to be given following UCB infusion.
Other Name: HPDSC
Experimental: Group C
unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC
Drug: Human Placental Derived Stem Cell
Infusions of thawed HPDSC to be given following UCB infusion.
Other Name: HPDSC
Experimental: Group D
double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC
Drug: Human Placental Derived Stem Cell
Infusions of thawed HPDSC to be given following UCB infusion.
Other Name: HPDSC

  Eligibility

Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • < 55 years of age
  • Life expectancy greater than 3 months
  • Lansky performance status ≥ 50% (children) or Karnofsky performance status ≥ 70% (adults) or ECOG performance status 0-2 (adults)
  • DLCO > 50 percent predicted
  • Left ventricular ejection fraction > 40% estimated
  • Creatinine clearance or estimated GFR . 60 mL/min/1.73m2
  • Serum bilirubin < 1.5x upper limit of normal
  • Transaminases < 3x upper limit of normal
  • Absence of uncontrolled infection
  • HIV negative

Exclusion Criteria:

  • Fanconi Anemia
  • Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities
  • Uncontrolled infection
  • Pregnant or breast-feeding females
  • Received other investigational agents within 30 days prior to the start of the conditioning regimen
  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: NCT01586455

Contacts
Contact: Mitchell S Cairo, MD 914-594-2150 mitchell_cairo@nymc.edu
Contact: Lauren Harrison, MSN 617-285-7844 lauren_harrison@nymc.edu

Locations
United States, Colorado
Children's Hospital Colorado Recruiting
Denver, Colorado, United States
Contact: Roger Giller, MD       Roger.Giller@childrenscolorado.org   
United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Mitchell Cairo, MD    914-594-2150    mitchell_cairo@nymc.edu   
Contact: Lauren Harrison, MSN    6172857844    lauren_harrison@nymc.edu   
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States
Contact: Michael Pulsipher, MD       michael.pulsipher@hsc.utah.edu   
Sponsors and Collaborators
New York Medical College
Investigators
Principal Investigator: Mitchell S Cairo, MD New York Medical College
  More Information

Responsible Party: New York Medical College
ClinicalTrials.gov Identifier: NCT01586455     History of Changes
Other Study ID Numbers: NYMC 550  NYMC IRB L-10,733 
Study First Received: April 25, 2012
Last Updated: October 4, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by New York Medical College:
umbilical cord blood
stem cell transplantation
placental stem cell
inborn errors of metabolism
marrow failure
Severe Combined Immunodeficiency Disease
AML
ALL
MDS

Additional relevant MeSH terms:
Leukemia
Anemia
Myelodysplastic Syndromes
Preleukemia
Leukemia, Lymphoid
Leukemia, Myeloid
Thrombocytopenia
Leukemia, Myeloid, Acute
Mucopolysaccharidoses
Gaucher Disease
Anemia, Aplastic
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukodystrophy, Globoid Cell
Neuronal Ceroid-Lipofuscinoses
Mucopolysaccharidosis VI
Wolman Disease
Adrenoleukodystrophy
Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Anemia, Diamond-Blackfan
Leukodystrophy, Metachromatic
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Mucopolysaccharidosis I
Fucosidosis
Neoplasms by Histologic Type
Neoplasms
Hematologic Diseases

ClinicalTrials.gov processed this record on December 02, 2016