Human Placental-Derived Stem Cell Transplantation

Phase 1

Completed

• Conditions: Metachromatic Leukodystrophy; Gaucher's Disease; Acute Lymphocytic Leukemia; Wolman Disease; Batten Disease; Severe Aplastic Anemia; Diamond-Blackfan Anemia; Amegakaryocytic Thrombocytopenia; Acute Myelogenous Leukemia; Mucopolysaccharidosis VI
• Interventions: Drug: Human Placental Derived Stem Cell

• Registration Number: NCT01586455
• Lead Sponsor: New York Medical College

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-04-25
• Study First Submitted QC: 2012-04-25
• Study First Posted: 2012-04-26
• Study Start: 2013-04
• Primary Completion: 2020-06
• Study Completion: 2022-09
• Status Verified: 2022-10
• Last Update Submitted: 2022-10-24
• Last Update Posted: 2022-10-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 43

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Group A	Human Placental Derived Stem Cell	related cord blood with ≥3/6 HLA match to the patient and related HPDSC
Group B	Human Placental Derived Stem Cell	unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC
Group C	Human Placental Derived Stem Cell	unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC
Group D	Human Placental Derived Stem Cell	double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC

Primary Outcome Measures

Name	Time	Method
Safety	100 days	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

Name	Time	Method
donor chimerism	1 year	donor chimerism will be assessed at set timepoints
engraftment	1 year
Survival	100 days and 180 days
Relapse	100 days and 180 days
Mortality	1 year

Trial Locations

Locations (3)

Children's Hospital Colorado; 🇺🇸 Denver, Colorado, United States

New York Medical College; 🇺🇸 Valhalla, New York, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States