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Study to determine the safety, tolerability, pharmacokinetics and immunogenicity of single intravenous administrations of GGF2 in patients with heart failure.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GGF2 | Placebo Comparator | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Glial growth factor 2/ Neuregulin 1β3 | Drug |
|
| |
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Anthony Caggiano, MD, PhD | Acorda Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Medical Group of Saint Joseph's | Atlanta | Georgia | 30342 | United States | ||
| Vanderbilt University |
There were six patients in each cohort/dose level, four received active treatment (GGF2) and two received placebo. In each dose level, the first two patients were randomized 1:1 to GGF2 or placebo and monitored for safety prior to randomizing the remaining patients in the cohort 3:1. GGF2 or placebo was administered as an IV infusion.
After informed consent, 40 patients with symptomatic Heart Failure (HF) were randomized (4:2) to GGF2 or placebo in 7 ascending dose cohorts. Patients were observed in hospital for 30 hours, then evaluated for adverse effects at 1, 2, 4, 12, and 24 weeks after infusion.
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| ID | Title | Description |
|---|---|---|
| FG000 | GGF2 First Dose | First dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| FG001 | GGF2 Second Escalated Dose | Second dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| FG002 | GGF2 Third Escalated Dose | Third dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| FG003 | GGF2 Fourth Escalated Dose | Fourth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| FG004 | GGF2 Fifth Escalated Dose | Fifth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| FG005 | GGF2 Sixth Escalated Dose | Sixth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed . |
| FG006 | GGF2 Seventh Escalataed Dose | Seventh dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort randomized (3:1) and dosed |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety Population. Consists of all patients who took one of the double-blind investigational drugs.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Patients in each of 7 cohorts randomized to Placebo |
| BG001 | GGF2 First Dose | First dosing: patients in cohort randomized to GGF2 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Safety population | Posted | Number | participants | 6 months |
|
6 months
Serious Treatment Emergent Adverse Events (TEAE) are defined as serious Adverse Events (AE) with date of onset (or worsening) on or after the start date of double-blind treatment.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Angina pectoris | Cardiac disorders | MedDRA |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anthony Caggiano, MD, PhD | Acorda Therapeutics, Inc. | 914-347-4300 | 5150 | tcaggiano@acorda.com |
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| ID | Term |
|---|---|
| D006333 | Heart Failure |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
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Not provided
| ID | Term |
|---|---|
| D020880 | Neuregulins |
| ID | Term |
|---|---|
| D009414 | Nerve Growth Factors |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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| Placebo |
| Other |
|
| 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 |
| Nashville |
| Tennessee |
| 37232-8802 |
| United States |
| BG002 | GGF2 Second Escalated Dose | Second dosing: patients in cohort randomized to GGF2 |
| BG003 | GGF2 Third Escalated Dose | Third dosing: patients in cohort randomized to GGF2 |
| BG004 | GGF2 Fourth Escalated Dose | Fourth dosing: patients in cohort randomized to GGF2 |
| BG005 | GGF2 Fifth Escalated Dose | Fifth dosing: patients in cohort randomized to GGF2 |
| BG006 | GGF2 Sixth Escalated Dose | Sixth dosing: patients in cohort randomized to GGF2. |
| BG007 | GGF2 Seventh Escalataed Dose | Seventh dosing: patients in cohort randomized to GGF2 |
| BG008 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| OG003 | GGF2 Third Escalated Dose |
| OG004 | GGF2 Fourth Escalated Dose |
| OG005 | GGF2 Fifth Escalated Dose |
| OG006 | GGF2 Sixth Escalated Dose |
| OG007 | GGF2 Seventh Escalataed Dose |
|
|
| Secondary | 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 | Safety Population | Posted | Mean | Standard Deviation | percent change | Screening, day 8, day 14, day 28, and 3 months post-dose |
|
|
|
| Secondary | 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 | Safety Population | Posted | Mean | Standard Deviation | Milliliter(s) | Screening, day 8, day 14, day 28, and 3 months post-dose |
|
|
|
| Secondary | 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 | Safety Population | Posted | Mean | Standard Deviation | Milliliter(s) | Screening, day 8, day 14, day 28, and 3 months post-dose |
|
|
|
| 0 |
| 13 |
| 6 |
| 13 |
| EG001 | GGF2 First Dose | First dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 1 | 4 | 4 | 4 |
| EG002 | GGF2 Second Escalated Dose | Second dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 1 | 4 | 4 | 4 |
| EG003 | GGF2 Third Escalated Dose | Third dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 0 | 4 | 2 | 4 |
| EG004 | GGF2 Fourth Escalated Dose | Fourth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 1 | 4 | 4 | 4 |
| EG005 | GGF2 Fifth Escalated Dose | Fifth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 0 | 4 | 4 | 4 |
| EG006 | GGF2 Sixth Escalated Dose | Sixth dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed . | 0 | 4 | 4 | 4 |
| EG007 | GGF2 Seventh Escalataed Dose | Seventh dosing: 1 GGF2, 1 pbo; if no drug-related dose-limiting toxicities in GGF2-treated patient, other 4 patients in cohort will be randomized (3:1) and dosed | 1 | 3 | 3 | 3 |
| Cardiac failure congestive | Cardiac disorders | MedDRA |
|
| Hy's law case | Hepatobiliary disorders | MedDRA |
|
| Viral infection | Infections and infestations | MedDRA |
|
| Dysgeusia | Nervous system disorders | MedDRA |
|
| Dizziness | Nervous system disorders | MedDRA |
|
| Lethargy | Nervous system disorders | MedDRA |
|
| Sinus headache | Nervous system disorders | MedDRA |
|
| Nausea | Gastrointestinal disorders | MedDRA |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA |
|
| Abdominal Pain | Gastrointestinal disorders | MedDRA |
|
| Abdominal tenderness | Gastrointestinal disorders | MedDRA |
|
| Gastritis | Gastrointestinal disorders | MedDRA |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA |
|
| Toothache | Gastrointestinal disorders | MedDRA |
|
| Vomiting | Gastrointestinal disorders | MedDRA |
|
| Fatigue | General disorders | MedDRA |
|
| Feeling hot | General disorders | MedDRA |
|
| Infusion site pain | General disorders | MedDRA |
|
| Injection site haematoma | General disorders | MedDRA |
|
| Injection site scab | General disorders | MedDRA |
|
| Pain | General disorders | MedDRA |
|
| Pyrexia | General disorders | MedDRA |
|
| Influenza like illness | General disorders | MedDRA |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA |
|
| Brain natriuretic peptide increased | Investigations | MedDRA |
|
| Blood bilirubin increased | Investigations | MedDRA |
|
| Blood chloride increased | Investigations | MedDRA |
|
| Blood creatinine increased | Investigations | MedDRA |
|
| Blood potassium increased | Investigations | MedDRA |
|
| Glomerular filtration rate decreased | Investigations | MedDRA |
|
| Heart rate increased | Investigations | MedDRA |
|
| Weight decreased | Investigations | MedDRA |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Nasal conjestion | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA |
|
| Hypotension | Vascular disorders | MedDRA |
|
| Flushing | Vascular disorders | MedDRA |
|
| Hot flush | Vascular disorders | MedDRA |
|
| Vein pain | Vascular disorders | MedDRA |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA |
|
| Bronchitis | Infections and infestations | MedDRA |
|
| Viral infection | Infections and infestations | MedDRA |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA |
|
| Fluid retention | Metabolism and nutrition disorders | MedDRA |
|
| Gout | Metabolism and nutrition disorders | MedDRA |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA |
|
| Angina pectoris | Cardiac disorders | MedDRA |
|
| Cardiac failure congestive | Cardiac disorders | MedDRA |
|
| Cardiac flutter | Cardiac disorders | MedDRA |
|
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA |
|
| Muscle strain | Injury, poisoning and procedural complications | MedDRA |
|
| Chromaturia | Renal and urinary disorders | MedDRA |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA |
|
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA |
|
| Ear pain | Ear and labyrinth disorders | MedDRA |
|
| Vitreous floaters | Eye disorders | MedDRA |
|
| Hy's law case | Hepatobiliary disorders | MedDRA |
|
Sponsor (Acorda) has the right to review and comment on proposed publications within a specified time frame, up to 60 days; multi-center trials require joint publication unless specifically permitted otherwise.
| D011506 | Proteins |
| D009419 | Nerve Tissue Proteins |
| D001685 | Biological Factors |
| Day 14 |
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| Day 28 |
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| 3 Months |
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| Day 14 |
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| Day 28 |
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| 3 Months |
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| Day 14 |
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| Day 28 |
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| 3 Months |
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