Clinical trial NCT01586455: Human Placental-Derived Stem Cell Transplantation
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: 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
Keywords: umbilical cord blood
stem cell transplantation
placental stem cell
inborn errors of metabolism
marrow failure
Severe Combined Immunodeficiency Disease
AML
ALL
MDS
Lead sponsor: New York Medical College
Sponsor class: OTHER
First posted: 2012-04-26
Enrollment: 43
Sex: ALL
Minimum age (years): 0
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Safety
timeFrame: 100 days
Secondary outcomes:
measure: donor chimerism
timeFrame: 1 year
measure: engraftment
timeFrame: 1 year
measure: Survival
timeFrame: 100 days and 180 days
measure: Relapse
timeFrame: 100 days and 180 days
measure: Mortality
timeFrame: 1 year
Other outcomes:
Locations:
city: Denver
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Valhalla
country: United States
facility: New York Medical College
state: New York
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: New York Medical College
name: Mitchell S Cairo, MD
role: PRINCIPAL_INVESTIGATOR
References: