Pilot Study of Simtuzumab in the Treatment of Liver Fibrosis
Primary Purpose
Liver Fibrosis
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Simtuzumab
Sponsored by
About this trial
This is an interventional treatment trial for Liver Fibrosis focused on measuring Gilead, Gilead Sciences, GSI, Liver Fibrosis, Liver, Fibrosis
Eligibility Criteria
Inclusion Criteria:
- Males and females 18 - 65 years of age
- Chronic liver disease of any etiology
- Stage 1-3 fibrosis by Metavir score on a liver biopsy.
- Body mass index <36 kg/m2
Exclusion Criteria:
- Any evidence of hepatic decompensation past or present
- Subjects currently abusing amphetamines, cocaine, opiates, or alcohol
- Clinically significant cardiac disease
- History of cancer, other than non-melanomatous skin cancer, within 5 years prior to Screening
- Systemic fungal, bacterial, viral, or other infection that is not controlled
- Use of systemic immunosuppressants within 28 days of the Pre-treatment Phase
- Use of approved therapy for hepatitis C or hepatitis B virus within 28 days of the Pre-treatment Phase
- Pregnant or lactating
- History of bleeding diathesis within the last 6 months of study Day 1
Sites / Locations
- Weill Cornell Medical College: NewYork-Presbyterian Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Cohort 1
Cohort 2
Arm Description
Participants will receive simtuzumab at a dose of 10 mg/kg by intravenous (IV) infusion every other week for a total of 3 infusions.
Participants will receive simtuzumab IV every other week for a total of 3 infusions. The dose will depend on the safety and tolerability of simtuzumab seen in Cohort 1 but will not exceed 20 mg/kg.
Outcomes
Primary Outcome Measures
Incidence of adverse events on multiple, escalating IV doses of simtuzumab
The endpoints to be evaluated will include graded Adverse Events, laboratory abnormalities, and vital sign measurements
Secondary Outcome Measures
Assessment of serum concentration of simtuzumab
Trough concentrations will be summarized by day, treatment and dose.
Antibody formation to simtuzumab (anti-simtuzumab Abs)
Immunogenicity endpoints will be geometric mean titer (GMT) and geometric mean fold rate (GMFR) for a select set of antibodies.
Measurement of pharmacodynamic (PD) markers after administration of simtuzumab
Pharmacodynamic markers include: Tissue PD markers through mRNA expression, LOXL2, LOX, Other LOXL proteins, αSMA, Collagen 1A1, NFKB1, Caspase 1, SMAD, and NOD; Serum and plasma PD markers include: APRI, LOXL2, Osteopontin, Hyaluronic Acid, CXCL 9, 10 and 11, MMP1, MMP3, MMP9, TIMP1, CD40L, TGF-β1, ET-1, VEGF, GAL3, IL-6 / IL-8 / TNFα / IFNγ, α2-macroglobulin, Apolipoprotein A1.
Assessing the effects of chronic dosing of simtuzumab on liver structure and fibrotic markers
Measuring the effect of an additional 24 weeks of simtuzumab dosing on liver histology, LOXL2 and mRNA expression in the liver and serum markers of liver fibrosis
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01452308
Brief Title
Pilot Study of Simtuzumab in the Treatment of Liver Fibrosis
Official Title
A Phase 2a, Pilot, Open-Label Trial Evaluating the Safety, Tolerability and Pharmacodynamic Effects of GS-6624 in Subjects With Fibrosis of the Liver
Study Type
Interventional
2. Study Status
Record Verification Date
January 2014
Overall Recruitment Status
Completed
Study Start Date
November 2011 (undefined)
Primary Completion Date
January 2013 (Actual)
Study Completion Date
August 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gilead Sciences
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will evaluate the safety and tolerability of simtuzumab (GS-6624) in patients with fibrosis of the liver.
Up to 20 participants will be enrolled into two sequential cohorts. Cohort 1 will consist of 10 participants who will receive simtuzumab every other week for a total of 3 infusions. Participants in Cohort 2 (10 subjects) will also receive simtuzumab every other week for a total of 3 infusions; the dose will depend on the safety and tolerability of simtuzumab seen in Cohort 1.
Participants from both cohorts who have completed the main study will be allowed to continue on simtuzumab treatment for an additional extension period, and will receive up to 13 additional infusions of simtuzumab at a fixed dose of 700 mg for an additional 24 weeks.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Liver Fibrosis
Keywords
Gilead, Gilead Sciences, GSI, Liver Fibrosis, Liver, Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Participants will receive simtuzumab at a dose of 10 mg/kg by intravenous (IV) infusion every other week for a total of 3 infusions.
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
Participants will receive simtuzumab IV every other week for a total of 3 infusions. The dose will depend on the safety and tolerability of simtuzumab seen in Cohort 1 but will not exceed 20 mg/kg.
Intervention Type
Biological
Intervention Name(s)
Simtuzumab
Other Intervention Name(s)
GS-6624
Primary Outcome Measure Information:
Title
Incidence of adverse events on multiple, escalating IV doses of simtuzumab
Description
The endpoints to be evaluated will include graded Adverse Events, laboratory abnormalities, and vital sign measurements
Time Frame
Through Week 14
Secondary Outcome Measure Information:
Title
Assessment of serum concentration of simtuzumab
Description
Trough concentrations will be summarized by day, treatment and dose.
Time Frame
Through Week 14
Title
Antibody formation to simtuzumab (anti-simtuzumab Abs)
Description
Immunogenicity endpoints will be geometric mean titer (GMT) and geometric mean fold rate (GMFR) for a select set of antibodies.
Time Frame
Through Week 14
Title
Measurement of pharmacodynamic (PD) markers after administration of simtuzumab
Description
Pharmacodynamic markers include: Tissue PD markers through mRNA expression, LOXL2, LOX, Other LOXL proteins, αSMA, Collagen 1A1, NFKB1, Caspase 1, SMAD, and NOD; Serum and plasma PD markers include: APRI, LOXL2, Osteopontin, Hyaluronic Acid, CXCL 9, 10 and 11, MMP1, MMP3, MMP9, TIMP1, CD40L, TGF-β1, ET-1, VEGF, GAL3, IL-6 / IL-8 / TNFα / IFNγ, α2-macroglobulin, Apolipoprotein A1.
Time Frame
Through Week 14
Title
Assessing the effects of chronic dosing of simtuzumab on liver structure and fibrotic markers
Description
Measuring the effect of an additional 24 weeks of simtuzumab dosing on liver histology, LOXL2 and mRNA expression in the liver and serum markers of liver fibrosis
Time Frame
Up to 24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Males and females 18 - 65 years of age
Chronic liver disease of any etiology
Stage 1-3 fibrosis by Metavir score on a liver biopsy.
Body mass index <36 kg/m2
Exclusion Criteria:
Any evidence of hepatic decompensation past or present
Subjects currently abusing amphetamines, cocaine, opiates, or alcohol
Clinically significant cardiac disease
History of cancer, other than non-melanomatous skin cancer, within 5 years prior to Screening
Systemic fungal, bacterial, viral, or other infection that is not controlled
Use of systemic immunosuppressants within 28 days of the Pre-treatment Phase
Use of approved therapy for hepatitis C or hepatitis B virus within 28 days of the Pre-treatment Phase
Pregnant or lactating
History of bleeding diathesis within the last 6 months of study Day 1
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeffrey Bornstein, MD
Organizational Affiliation
Gilead Sciences
Official's Role
Study Director
Facility Information:
Facility Name
Weill Cornell Medical College: NewYork-Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Pilot Study of Simtuzumab in the Treatment of Liver Fibrosis
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