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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


Phase 1
N/A
55 Years
Open (Enrolling)
Both
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

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Trial Information

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


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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety

Outcome Description:

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.

Outcome Time Frame:

100 days

Safety Issue:

Yes

Principal Investigator

Mitchell S Cairo, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

New York Medical College

Authority:

United States: Food and Drug Administration

Study ID:

NYMC 550

NCT ID:

NCT01586455

Start Date:

March 2013

Completion Date:

December 2019

Related Keywords:

  • 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
  • umbilical cord blood
  • stem cell transplantation
  • placental stem cell
  • inborn errors of metabolism
  • marrow failure
  • Severe Combined Immunodeficiency Disease
  • AML
  • ALL
  • MDS
  • Anemia
  • Anemia, Aplastic
  • Fucosidosis
  • Leukodystrophy, Globoid Cell
  • Gaucher Disease
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukodystrophy, Metachromatic
  • Mucopolysaccharidosis I
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis VI
  • Myelodysplastic Syndromes
  • Preleukemia
  • Niemann-Pick Diseases
  • Niemann-Pick Disease, Type A
  • Niemann-Pick Disease, Type C
  • Thrombocytopenia
  • Wolman Disease
  • Cholesterol Ester Storage Disease
  • Adrenoleukodystrophy
  • Aphasia, Primary Progressive
  • Pick Disease of the Brain
  • Frontotemporal Dementia
  • Anemia, Diamond-Blackfan
  • Neuronal Ceroid-Lipofuscinoses

Name

Location

New York Medical CollegeValhalla, New York  10595