Human Placental-Derived Stem Cell Transplantation

NCT ID: NCT01586455

Last Updated: 2022-10-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-04-30

Study Completion Date

2022-09-30

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.

Detailed Description

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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

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group A

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

Group Type EXPERIMENTAL

Human Placental Derived Stem Cell

Intervention Type DRUG

Infusions of thawed HPDSC to be given following UCB infusion.

Group B

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

Group Type EXPERIMENTAL

Human Placental Derived Stem Cell

Intervention Type DRUG

Infusions of thawed HPDSC to be given following UCB infusion.

Group C

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

Group Type EXPERIMENTAL

Human Placental Derived Stem Cell

Intervention Type DRUG

Infusions of thawed HPDSC to be given following UCB infusion.

Group D

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

Group Type EXPERIMENTAL

Human Placental Derived Stem Cell

Intervention Type DRUG

Infusions of thawed HPDSC to be given following UCB infusion.

Interventions

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Human Placental Derived Stem Cell

Infusions of thawed HPDSC to be given following UCB infusion.

Intervention Type DRUG

Other Intervention Names

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HPDSC

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
Minimum Eligible Age

0 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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New York Medical College

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mitchell S Cairo, MD

Role: PRINCIPAL_INVESTIGATOR

New York Medical College

Locations

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Children's Hospital Colorado

Denver, Colorado, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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NYMC IRB L-10,733

Identifier Type: OTHER

Identifier Source: secondary_id

NYMC 550

Identifier Type: -

Identifier Source: org_study_id

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