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Completed

NCT Number: NCT01586455

Human Placental-Derived Stem Cell Transplantation

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.

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Key information

Conditions

Mucopolysaccharidosis I Acute Lymphocytic Leukemia Acute Myelogenous Leukemia Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Amegakaryocytic Thrombocytopenia Anemia Anemia, Aplastic Anemia, Diamond-Blackfan Anemia, Hypoplastic, Congenital Batten Disease Bone Marrow Diseases Bone Marrow Failure Disorders Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Cholesterol Ester Storage Disease Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Demyelinating Diseases Diamond-Blackfan Anemia Endocrine System Diseases Fucosidosis Gaucher Disease Gaucher's Disease Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Histiocytosis Histiocytosis, Non-Langerhans-Cell Immune System Diseases Immunologic Deficiency Syndromes Immunoproliferative Disorders Infant, Newborn, Diseases Intellectual Disability Krabbe's Disease Leukemia Leukemia, Lymphoid Leukemia, Myeloid Leukemia, Myeloid, Acute Leukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Leukoencephalopathies Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lymphatic Diseases Lymphoproliferative Disorders Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Metachromatic Leukodystrophy Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis VI Myelodysplastic Syndrome Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type Nervous System Diseases Neurobehavioral Manifestations Neurodegenerative Diseases Neurologic Manifestations Neuronal Ceroid-Lipofuscinoses Niemann-Pick Disease Niemann-Pick Diseases Nutritional and Metabolic Diseases Peroxisomal Disorders Precursor Cell Lymphoblastic Leukemia-Lymphoma Primary Immunodeficiency Diseases Red-Cell Aplasia, Pure Severe Aplastic Anemia Severe Combined Immunodeficiency Skin and Connective Tissue Diseases Sphingolipidoses Sulfatidosis Wolman Disease X-Linked Combined Immunodeficiency Diseases X-Linked Intellectual Disability

Age range

0 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Children's Hospital Colorado, Denver, Colorado, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Human Placental Derived Stem Cell

Drug

Infusions of thawed HPDSC to be given following UCB infusion.

Other names: HPDSC

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

New York Medical College

Other

Registry information

Official study 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

Acronym: HPDSC

Important dates

Study start
2013
Primary completion
2020
Study completion
2022
First posted
Apr 26, 2012
Registry last updated
Oct 25, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.