Human Placental-Derived Stem Cell Transplantation (HPDSC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 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: February 18, 2014
Last verified: February 2014
  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:

Genetics Home Reference related topics: adenosine deaminase deficiency alpha-methylacyl-CoA racemase deficiency CASK-related intellectual disability Chanarin-Dorfman syndrome CHMP2B-related frontotemporal dementia cholesteryl ester storage disease congenital neuronal ceroid lipofuscinosis core binding factor acute myeloid leukemia cytogenetically normal acute myeloid leukemia D-bifunctional protein deficiency Diamond-Blackfan anemia familial acute myeloid leukemia with mutated CEBPA frontotemporal dementia with parkinsonism-17 fucosidosis Gaucher disease GRN-related frontotemporal dementia inclusion body myopathy with early-onset Paget disease and frontotemporal dementia infantile neuronal ceroid lipofuscinosis juvenile Batten disease Krabbe disease Kufs disease Langerhans cell histiocytosis late-infantile neuronal ceroid lipofuscinosis leukoencephalopathy with vanishing white matter MECP2 duplication syndrome megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy mucopolysaccharidosis type I mucopolysaccharidosis type VI Niemann-Pick disease peroxisomal acyl-CoA oxidase deficiency PPM-X syndrome purine nucleoside phosphorylase deficiency Renpenning syndrome Schindler disease succinic semialdehyde dehydrogenase deficiency Wolman disease X-linked adrenoleukodystrophy X-linked severe combined immunodeficiency ZAP70-related severe combined immunodeficiency Zellweger spectrum
Genetic and Rare Diseases Information Center resources: Leukemia, Myeloid Myelodysplastic Syndromes Acute Lymphoblastic Leukemia Fucosidosis Gaucher Disease Leukodystrophy Krabbe Leukodystrophy Metachromatic Leukodystrophy Mucopolysaccharidosis Type I Mucopolysaccharidosis Mucopolysaccharidosis Type VI Niemann-Pick Disease Wolman Disease Lysosomal Acid Lipase Deficiency Cholesteryl Ester Storage Disease Adrenomyeloneuropathy Adrenoleukodystrophy X-linked Pick's Disease Frontotemporal Dementia Frontotemporal Dementia, Ubiquitin-positive Primary Progressive Aphasia Semantic Dementia Aplastic Anemia Acute Myelocytic Leukemia Acute Non Lymphoblastic Leukemia Acute Myeloid Leukemia, Adult Severe Combined Immunodeficiency Batten Disease Spielmeyer-Vogt Disease Neuronal Ceroid Lipofuscinoses Diamond-Blackfan Anemia Niemann-Pick Disease Type C1 Adult Neuronal Ceroid Lipofuscinosis Ceroid Storage Disease Sphingolipidosis Hand-Schuller-Christian Disease Langerhans Cell Histiocytosis Hypoadrenalism
U.S. FDA Resources

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
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 914-594-3650 mitchell_cairo@nymc.edu
Contact: Lauren Harrison, MSN 978-993-4372 lauren_harrison@nymc.edu

Locations
United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Mitchell Cairo, MD    914-594-3650    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

No publications provided

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: February 18, 2014
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:
Gaucher Disease
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Wolman Disease
Pick Disease of the Brain
Neuronal Ceroid-Lipofuscinoses
Hematologic Diseases
Bone Marrow Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Lipid Metabolism Disorders
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Connective Tissue Diseases
Precancerous Conditions
Blood Platelet Disorders
Cholesterol Ester Storage Disease
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on July 29, 2014