Mobilization of Stem Cells With G-CSF and Mozobil in Patients With End Stage Liver Disease

A phase I trial to study the safety of mobilization of stem cells with G-CSF and Mozobil in patients with chronic liver disease.

Study Overview

• Status: Unknown
• Conditions: End Stage Liver DIsease
• Intervention / Treatment: Drug: Mobilization with G-CSF and Mozobil

Detailed Description

Liver cirrhosis in humans represents the end stage of chronic liver injury. Supply of "new" stem cells to the liver could regenerate hepatocytes and restore the lost function. Delivery of Mesenchymal Stem Cells (MSCs) has been shown in animal models and limited clinical trials to result in improved liver disease (MELD) score.

In preclinical studies we have demonstrated that the combination of G-CSF plus Mozobil can effectively mobilize both hematopoietic stem cells (HSCs) and MSCs into the peripheral circulation. While G-CSF only mobilizes HSCs.

The clinical trial will test the safety of treating patients with end stage liver disease with G-CSF and Mozobil to mobilize MSCs into the peripheral circulation.

• Study Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Newark, New Jersey, United States, 07101
      • Recruiting
      • University of Medicine and Dentistry of New Jersey
      • Contact: Baburao Koneru, MD; Email: koneruba@umdnj.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients with a clinical diagnosis of cirrhosis Age greater than or equal to 18 years MELD score less than or equal to 12 able to provide informed consent HIV and HBsAg seronegative Platelet count >50,000, WBC count > 2,000 No history of malignancy within the last 5 years, except for non-melanoma skin cancer or cervical carcinoma in situ No lesions suspicious for liver cancer on CT and/or MRI within prior 4 months

Exclusion Criteria:

Patients with acute or subacute onset of liver disease Patients who have received a liver transplant Age < 18 MELD score >12 Patients whose MELD scores are currently less than or equal to 12 but with history of prior deterioration with MELD score >12 Unable to provide informed consent Patients with HIV or HBsAg seropositivity Pregnant or lactating females Enrolled in another research protocol Any condition that precludes serial follow up Patients with history of malignancy within the last 5 years, except for non-melanoma skin cancer or cervical carcinoma in situ Any lesions suspicious for liver cancer on CT and/or MRI within prior 4 months Patients with palpable splenomegaly on physical examination ANy condition that in the investigators opinion would likely increase the risk of particpation or would likely confound interpretation of the data

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Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mobilization with G-CSF plus Mozobil; Patients will receive G-CSF (Filgrastim) plus Mozobil (Plerixafor)	Drug: Mobilization with G-CSF and Mozobil; Treatment with drugs for mobilization of MSCs; Other Names: Filgrastim; Neupogen; AMD3100; Plerixafor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Toxicity as measured by bone pain, hematologic parameters, GI measures and renal parameters	The primary end point for this study is the safety of mobilization of stem cells in patients with end stage liver disease. Adverse events will be documented to assess safety.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effects of Mobilization	The secondary objective is to study the mobilization of stem cells, including MSCs, to the peripheral circulation and the effect on liver function. Functional assays will define the levels of heamtopoietic stem cells (CD34+ cells) and MSCs (CFU-F) in the circulation of patients.	12 months

Collaborators and Investigators

• Sponsor: Proteonomix, Inc.
• Collaborators: University of Medicine and Dentistry of New Jersey; Numoda
• Investigators: Principal Investigator: Baburao Koneru, MD, University of Medicine and Dentistry of New Jersey

Study record dates

Study Major Dates

• Study Start: October 1, 2012
• Primary Completion (Anticipated): September 1, 2013
• Study Completion (Anticipated): September 1, 2013

Study Registration Dates

• First Submitted: October 11, 2012
• First Submitted That Met QC Criteria: October 19, 2012
• First Posted (Estimate): October 22, 2012

Study Record Updates

• Last Update Posted (Estimate): January 24, 2013
• Last Update Submitted That Met QC Criteria: January 23, 2013
• Last Verified: January 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Liver Failure; Hepatic Insufficiency; Liver Diseases; End Stage Liver Disease; Anti-Infective Agents; Antiviral Agents; Anti-HIV Agents; Anti-Retroviral Agents; Plerixafor
• Other Study ID Numbers: Prot001