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
N/A
55 Years
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
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 College | Valhalla, New York 10595 |
