Trial record 12 of 24 for:    Krabbe Disease

Human Placental-Derived Stem Cell Transplantation (HPDSC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by New York Medical College
Information provided by (Responsible Party):
New York Medical College Identifier:
First received: April 25, 2012
Last updated: September 23, 2014
Last verified: September 2014

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
Niemann-Pick Disease
Metachromatic Leukodystrophy
Wolman Disease
Krabbe's Disease
Gaucher's Disease
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: 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 familial glucocorticoid deficiency 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 ZAP70-related severe combined immunodeficiency Zellweger spectrum
Genetic and Rare Diseases Information Center resources: Acute Lymphoblastic Leukemia Acute Monoblastic Leukemia Acute Myeloblastic Leukemia Type 5 Acute Myelocytic Leukemia Acute Myeloid Leukemia, Adult Acute Non Lymphoblastic Leukemia Adrenoleukodystrophy X-linked Adrenomyeloneuropathy Aplastic Anemia Batten Disease Cholesteryl Ester Storage Disease Diamond-Blackfan Anemia Frontotemporal Dementia Frontotemporal Dementia, Ubiquitin-positive Gaucher Disease Hand-Schuller-Christian Disease Hypoadrenalism Krabbe Leukodystrophy Langerhans Cell Histiocytosis Leukemia, Myeloid Leukodystrophy Lymphosarcoma Lysosomal Acid Lipase Deficiency Metachromatic Leukodystrophy Mucopolysaccharidosis Mucopolysaccharidosis Type I Mucopolysaccharidosis Type VI Myelodysplastic Syndromes Neuronal Ceroid Lipofuscinoses Niemann-Pick Disease Niemann-Pick Disease Type C1 Pick's Disease Primary Progressive Aphasia Semantic Dementia Severe Combined Immunodeficiency Sphingolipidosis Spielmeyer-Vogt Disease Wolman Disease
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


Ages Eligible for Study:   up to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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 identifier: NCT01586455

Contact: Mitchell S Cairo, MD 914-594-3650
Contact: Lauren Harrison, MSN 978-993-4372

United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Mitchell Cairo, MD    914-594-3650   
Contact: Lauren Harrison, MSN    6172857844   
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States
Contact: Michael Pulsipher, MD   
Sponsors and Collaborators
New York Medical College
Principal Investigator: Mitchell S Cairo, MD New York Medical College
  More Information

No publications provided

Responsible Party: New York Medical College Identifier: NCT01586455     History of Changes
Other Study ID Numbers: NYMC 550, NYMC IRB L-10,733
Study First Received: April 25, 2012
Last Updated: September 23, 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

Additional relevant MeSH terms:
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Adrenal Gland Diseases
Bone Marrow Diseases
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Connective Tissue Diseases
Demyelinating Diseases
Endocrine System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hereditary Central Nervous System Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Immune System Diseases
Immunoproliferative Disorders
Lipid Metabolism Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Nervous System Diseases
Peroxisomal Disorders
Anemia, Diamond-Blackfan
Leukemia, Myeloid, Acute processed this record on March 26, 2015