Human Placental-Derived Stem Cell Transplantation

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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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

Intervention / Treatment
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* Drug: Human Placental Derived Stem Cell


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group A<br>related cord blood with ≥3/6 HLA match to the patient and related HPDSC | Drug: Human Placental Derived Stem Cell<br>* Infusions of thawed HPDSC to be given following UCB infusion.<br>* Other names: HPDSC;|
| Experimental: Group B<br>unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC | Drug: Human Placental Derived Stem Cell<br>* Infusions of thawed HPDSC to be given following UCB infusion.<br>* Other names: HPDSC;|
| Experimental: Group C<br>unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC | Drug: Human Placental Derived Stem Cell<br>* Infusions of thawed HPDSC to be given following UCB infusion.<br>* Other names: HPDSC;|
| Experimental: Group D<br>double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC | Drug: Human Placental Derived Stem Cell<br>* Infusions of thawed HPDSC to be given following UCB infusion.<br>* Other names: HPDSC;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 100 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| donor chimerism | donor chimerism will be assessed at set timepoints | 1 year | 
| engraftment |  | 1 year | 
| Survival |  | 100 days and 180 days | 
| Relapse |  | 100 days and 180 days | 
| Mortality |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALL, AML, umbilical cord blood, stem cell transplantation, placental stem cell, inborn errors of metabolism, marrow failure, Severe Combined Immunodeficiency Disease, MDS