Human Placental-Derived Stem Cell Transplantation

NCT01586455 | Completed Phase 1 DMC

• 2 other identifiers: NYMC 550NYMC IRB L-10,733
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 3

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.

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• donor chimerism

  1 year

• engraftment

  1 year

• Survival

  100 days and 180 days

• Relapse

  100 days and 180 days

• Mortality

  1 year

Study Arms (4)

Group A

EXPERIMENTAL

related cord blood with ≥3/6 HLA match to the patient and related HPDSC

Drug: Human Placental Derived Stem Cell

Group B

EXPERIMENTAL

unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC

Drug: Human Placental Derived Stem Cell

Group C

EXPERIMENTAL

unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC

Drug: Human Placental Derived Stem Cell

Group D

EXPERIMENTAL

double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC

Drug: Human Placental Derived Stem Cell

Interventions

Human Placental Derived Stem Cell DRUG

Infusions of thawed HPDSC to be given following UCB infusion.

Also known as: HPDSC

Group A; Group B; Group C; Group D

Eligibility Criteria

• Age: 0 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \< 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

You may not qualify if:

• 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

Sponsors & Collaborators

• New York Medical College: lead

Study Sites (3)

Children's Hospital Colorado

Denver, Colorado, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

University of Utah

Salt Lake City, Utah, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I; Mucopolysaccharidosis VI; Adrenoleukodystrophy; Niemann-Pick Diseases; Leukodystrophy, Metachromatic; Wolman Disease; Leukodystrophy, Globoid Cell; Gaucher Disease; Fucosidosis; Neuronal Ceroid-Lipofuscinoses; Anemia, Aplastic; Anemia, Diamond-Blackfan; Myelodysplastic Syndromes; Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Metabolism, Inborn Errors; X-Linked Combined Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Peroxisomal Disorders; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Sulfatidosis; Cholesterol Ester Storage Disease; Infant, Newborn, Diseases; Neurodegenerative Diseases; Anemia; Hematologic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Anemia, Hypoplastic, Congenital; Red-Cell Aplasia, Pure; Congenital Bone Marrow Failure Syndromes; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Leukemia, Lymphoid; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Severe Combined Immunodeficiency; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes

Study Officials

• Mitchell S Cairo, MD

  New York Medical College

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2012
• First Posted: April 26, 2012
• Study Start: April 1, 2013
• Primary Completion: June 1, 2020
• Study Completion: September 1, 2022
• Last Updated: October 25, 2022

Record last verified: 2022-10

Locations

US (3)