Human Placental-Derived Stem Cell Transplantation (HPDSC)
Primary Purpose
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Human Placental Derived Stem Cell
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring umbilical cord blood, stem cell transplantation, placental stem cell, inborn errors of metabolism, marrow failure, Severe Combined Immunodeficiency Disease, AML, ALL, MDS
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
Sites / Locations
- Children's Hospital Colorado
- New York Medical College
- University of Utah
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Group A
Group B
Group C
Group D
Arm Description
related cord blood with ≥3/6 HLA match to the patient and related HPDSC
unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC
unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC
double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC
Outcomes
Primary Outcome Measures
Safety
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
donor chimerism
donor chimerism will be assessed at set timepoints
engraftment
Survival
Relapse
Mortality
Full Information
NCT ID
NCT01586455
First Posted
April 25, 2012
Last Updated
October 24, 2022
Sponsor
New York Medical College
1. Study Identification
Unique Protocol Identification Number
NCT01586455
Brief Title
Human Placental-Derived Stem Cell Transplantation
Acronym
HPDSC
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
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
April 2013 (Actual)
Primary Completion Date
June 2020 (Actual)
Study Completion Date
September 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
New York Medical College
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
43 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group A
Arm Type
Experimental
Arm Description
related cord blood with ≥3/6 HLA match to the patient and related HPDSC
Arm Title
Group B
Arm Type
Experimental
Arm Description
unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC
Arm Title
Group C
Arm Type
Experimental
Arm Description
unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC
Arm Title
Group D
Arm Type
Experimental
Arm Description
double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC
Intervention Type
Drug
Intervention Name(s)
Human Placental Derived Stem Cell
Other Intervention Name(s)
HPDSC
Intervention Description
Infusions of thawed HPDSC to be given following UCB infusion.
Primary Outcome Measure Information:
Title
Safety
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.
Time Frame
100 days
Secondary Outcome Measure Information:
Title
donor chimerism
Description
donor chimerism will be assessed at set timepoints
Time Frame
1 year
Title
engraftment
Time Frame
1 year
Title
Survival
Time Frame
100 days and 180 days
Title
Relapse
Time Frame
100 days and 180 days
Title
Mortality
Time Frame
1 year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitchell S Cairo, MD
Organizational Affiliation
New York Medical College
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Colorado
City
Denver
State/Province
Colorado
Country
United States
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Human Placental-Derived Stem Cell Transplantation
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