Human Placental-Derived Stem Cell Transplantation (HPDSC)

New York Medical College

Status and phase: Completed; Phase 1

Conditions: Niemann-Pick Disease; Acute Myelogenous Leukemia; Adrenoleukodystrophy; Metachromatic Leukodystrophy; Krabbe's Disease; Amegakaryocytic Thrombocytopenia; Mucopolysaccharidosis VI; Batten Disease; Severe Aplastic Anemia; Acute Lymphocytic Leukemia; Mucopolysaccharidosis I; Diamond-Blackfan Anemia; Fucosidosis; Gaucher's Disease; Wolman Disease; Myelodysplastic Syndrome

Treatments: Drug: Human Placental Derived Stem Cell

Study type: Interventional

Funder types: Other

Identifiers

NCT01586455

NYMC 550

NYMC IRB L-10,733 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 43 patients

Sex: All

Ages: Under 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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43 participants in 4 patient groups

Group A

Experimental group

Description:

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

Treatment:

Drug: Human Placental Derived Stem Cell

Group B

Experimental group

Description:

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

Treatment:

Drug: Human Placental Derived Stem Cell

Group C

Experimental group

Description:

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

Treatment:

Drug: Human Placental Derived Stem Cell

Group D

Experimental group

Description:

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

Treatment:

Drug: Human Placental Derived Stem Cell