We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 14 for:    krabbe disease cord blood
Previous Study | Return to List | Next Study

Human Placental-Derived Stem Cell Transplantation (HPDSC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01586455
Recruitment Status : Completed
First Posted : April 26, 2012
Last Update Posted : October 25, 2022
Sponsor:
Information provided by (Responsible Party):
New York Medical College

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
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 or disease Intervention/treatment 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 Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 43 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : April 2013
Actual Primary Completion Date : June 2020
Actual Study Completion Date : September 2022

Resource links provided by the National Library of Medicine

Genetic and Rare Diseases Information Center resources: Acute Lymphoblastic Leukemia Lymphoblastic Lymphoma Myelodysplastic Syndromes Myeloid Leukemia Acute Myeloid Leukemia Acute Non Lymphoblastic Leukemia Frontotemporal Dementia Frontotemporal Dementia, Ubiquitin-positive Primary Progressive Aphasia Semantic Dementia Acute Graft Versus Host Disease Niemann-Pick Disease Niemann-Pick Disease Type A Aplastic Anemia Leukodystrophy Metachromatic Leukodystrophy Behavioral Variant of Frontotemporal Dementia Krabbe Disease Gaucher Disease Mucopolysaccharidosis Neuronal Ceroid Lipofuscinosis Diamond-Blackfan Anemia Mucopolysaccharidosis Type I Wolman Disease Cholesteryl Ester Storage Disease Lysosomal Acid Lipase Deficiency Mucopolysaccharidosis Type VI Severe Combined Immunodeficiency Adrenomyeloneuropathy X-linked Adrenoleukodystrophy Fucosidosis Lymphosarcoma Niemann-Pick Disease Type C1 Adult Neuronal Ceroid Lipofuscinosis Sphingolipidosis Peroxisomal Biogenesis Disorders Non-Langerhans-Cell Histiocytosis Pure Red Cell Aplasia

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
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


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Safety [ Time Frame: 100 days ]
    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 :
  1. donor chimerism [ Time Frame: 1 year ]
    donor chimerism will be assessed at set timepoints

  2. engraftment [ Time Frame: 1 year ]
  3. Survival [ Time Frame: 100 days and 180 days ]
  4. Relapse [ Time Frame: 100 days and 180 days ]
  5. Mortality [ Time Frame: 1 year ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   0 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01586455


Locations
Layout table for location information
United States, Colorado
Children's Hospital Colorado
Denver, Colorado, United States
United States, New York
New York Medical College
Valhalla, New York, United States, 10595
United States, Utah
University of Utah
Salt Lake City, Utah, United States
Sponsors and Collaborators
New York Medical College
Investigators
Layout table for investigator information
Principal Investigator: Mitchell S Cairo, MD New York Medical College
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: New York Medical College
ClinicalTrials.gov Identifier: NCT01586455    
Other Study ID Numbers: NYMC 550
NYMC IRB L-10,733 ( Other Identifier: New York Medical College )
First Posted: April 26, 2012    Key Record Dates
Last Update Posted: October 25, 2022
Last Verified: October 2022
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:
Layout table for MeSH terms
Gaucher Disease
Pick Disease of the Brain
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Leukodystrophy, Globoid Cell
Wolman Disease
Hematologic Diseases
Bone Marrow Diseases
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Blood Platelet Disorders
Bone Marrow Failure Disorders
 Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lipid Metabolism Disorders
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System