Single Ascending Doses of GGF2 in Patients With Left Ventricular Dysfunction and Symptomatic Heart Failure (GGF2-1101-1)

A Phase 1, Double-Blind, Placebo-Controlled Study of Single Ascending Doses of GGF2 in Patients With Left Ventricular Dysfunction and Symptomatic Heart Failure

Study to determine the safety, tolerability, pharmacokinetics and immunogenicity of single intravenous administrations of GGF2 in patients with heart failure.

Study Overview

• Status: Completed
• Conditions: Heart Failure
• Intervention / Treatment: Other: Placebo; Drug: Glial growth factor 2/ Neuregulin 1β3

Detailed Description

Phase 1, double-blind, placebo-controlled, dose escalation study to determine the safety, tolerability, pharmacokinetics and immunogenicity of single intravenous administrations of GGF2 in cohorts of patients with left ventricular dysfunction and symptomatic heart failure.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • The Medical Group of Saint Joseph's
  • Tennessee
    • Nashville, Tennessee, United States, 37232-8802
      • Vanderbilt University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Left ventricular ejection fraction (LVEF) between 10% and 40%.
2. Male or female, aged 18 to 75 years, inclusive.

Exclusion Criteria:

1. Received any investigational agent or participation in any clinical study of an investigational agent or investigational therapy up to 30 days prior to the screening visit.
2. Use of any new prescription medication up to 14 days prior to receiving investigational drug.
3. Patients with known specific hepatic disease; bilirubin >2 mg/dL, SGOT > 100 IU.
4. Patients with a history of hepatic impairment (hepatitis B and C).
5. Serum creatinine >2.5 mg/dL.
6. Documented stroke or transient ischemic attack (TIA) within 60 days of study enrollment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Double

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: GGF2; Seven dosing cohorts: 2 patients randomized to receive 1 GGF2, 1 placebo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed	Other: Placebo; Drug: Glial growth factor 2/ Neuregulin 1β3; Other Names: Recombinant Human Glial Growth Factor 2

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of Single Ascending Doses of GGF2 Via an Assessment of the Toxicology Profile as Measured by Treatment Emergent Adverse Events (TEAEs)	Safety/ tolerability of single dose; cumulative safety over 6 months TEAEs are defined as adverse events with date of onset (or worsening) on or after the start date of double-blind treatment and no more than 28 days from the start date of double-blind treatment.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline of Two-Dimensional Echocardiogram (2D-ECHO) Measured by Ejection Fraction (EF)	An echocardiogram is a type of ultrasound test that uses high-pitched sound waves that are sent through a device called a transducer. The device picks up echoes of the sound waves as they bounce off the different parts of your heart. These echoes are turned into moving pictures of your heart that can be seen on a video screen.¹ Ejection fraction is a measurement of the percentage of blood leaving your heart each time it contracts.² ¹http://wakeinternalmedicine.com/services-and-procedures/services/radiology/2d-echo/ ²http://www.mayoclinic.org/ejection-fraction/expert-answers/faq-20058286	Screening, day 8, day 14, day 28, and 3 months post-dose
Change From Baseline of 2D-ECHO Measured by End-Diastolic Volume (EDV)	EDV is the amount of blood in the ventricle immediately before a cardiac contraction begins; used as a measurement of diastolic function.¹ ¹http://medical-dictionary.thefreedictionary.com/end-diastolic+volume	Screening, day 8, day 14, day 28, and 3 months post-dose
Change From Baseline of 2D-ECHO Measured by End-Systolic Volume (ESV)	ESV is the volume of blood remaining in each ventricle at the end of systole.¹ ¹http://medical-dictionary.thefreedictionary.com/end-diastolic+volume	Screening, day 8, day 14, day 28, and 3 months post-dose

Collaborators and Investigators

• Sponsor: Acorda Therapeutics
• Investigators: Study Director: Anthony Caggiano, MD, PhD, Acorda Therapeutics

Study record dates

Study Major Dates

• Study Start: December 1, 2010
• Primary Completion (Actual): December 1, 2012
• Study Completion (Actual): March 1, 2013

Study Registration Dates

• First Submitted: December 8, 2010
• First Submitted That Met QC Criteria: December 9, 2010
• First Posted (Estimate): December 13, 2010

Study Record Updates

• Last Update Posted (Estimate): July 1, 2014
• Last Update Submitted That Met QC Criteria: June 30, 2014
• Last Verified: June 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Heart Failure; Ventricular Dysfunction; Ventricular Dysfunction, Left; Molecular Mechanisms of Pharmacological Action; Mitosis Modulators; Mitogens
• Other Study ID Numbers: 1101.1