Phase 2 a Study to Assess Safety and Pharmacokinetics of VAS203 in Patients With Traumatic Brain Injury (NOSTRA)
Primary Purpose
Traumatic Brain Injury
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VAS203
Saline
Sponsored by
About this trial
This is an interventional treatment trial for Traumatic Brain Injury
Eligibility Criteria
Inclusion Criteria:
- Written informed consent from patient's legal guardian or legal representative
- 18 - 65 years of age, inclusive
- Head trauma within the last 12 hours
- Traumatic brain injury with Glasgow Coma Score (GCS) ≥ 5 and that requires ICP monitoring
- Catheter placement for monitoring and management of increased ICP
- Microdialysis probe placement in penumbra zone or ipsilateral to contusion if focal
- Systolic blood pressure ≥ 100 mmHg
- Females of child-bearing potential must have a negative pregnancy test
Exclusion Criteria:
- Penetrating head injury (e.g. missile, stab wound)
- Not expected to survive more than 24 hours after admission
- Concurrent, but not pre-existing, spinal cord injury
- Unilateral and bilateral fixed and dilated pupil (> 4 mm)
- Cardiopulmonary resuscitation performed post injury
- continuing bleeding likely to require multiple transfusions (> 4 units red blood cells)
- Coma due to a "pure" epidural hematoma (lucid interval and absence of structural brain damage on CT scan)
- Coma suspected to be primarily due to other causes than head injury (e.g. drug overdose)
- Known or CT scan evidence of pre-existing major cerebral damage
- Decompressive craniectomy, planned prior to randomization
- Polytraumatic patients with Injury Severity Score > 25, or rhabdomyolysis
- Injuries to ascending aorta and/or carotid arteries
- serum creatinine values > 1.5 mg/dL
- estimated Glomerular Filtration Rate < 60 ml/min (MDRD-formula)
- body mass index (BMI) > 35, Body weight > 120 kg
- Any severe concomitant condition (cancer; hematologic, renal, hepatic, coronary disease; major psychiatric disorder; alcohol or drug abuse), that can be ascertained at admission
- Known to have received an experimental drug within 4 weeks prior to current injury
- Administration of > 100 ml of contrast media containing iodine
Sites / Locations
- Medical University Innsbruck Department of Neurology
- HIA Sainte-Anne Boulevard Sainte-Anne
- Vall d'Hebron University Hospital Department of Neurosurgery
- Hospital Clinic University of Barcelona Surgical Intensive Care Unit
- University Hospital Zuerich Surgical Intensive Care
- Southampton University Hospital Division of Clinical Neurosciences
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Placebo Comparator
Arm Label
VAS203 15 mg/kg
VAS203 20 mg/kg
VAS203 30 mg/kg
Saline
Arm Description
Three 5 mg/kg/12-hours infusion with 12 hours break after each infusion. Total dose: 15 mg/kg
10 mg/kg/24 hours, 48 hour continuous infusion. Total dose: 20 mg/kg
10 mg/kg/24 hours, 72 hour continuous infusion. Total dose: 30 mg/kg
0.9 % Sodium chloride infusion
Outcomes
Primary Outcome Measures
Safety and Tolerability of VAS203 in Patients With Moderate and Severe TBI
Tolerability (good, satisfactory, sufficient, poor) of VAS203 in patients with moderate and severe TBI, as judged by the investigators at day 14.
Safety outcome measure description see safety section
Secondary Outcome Measures
Duration (Number of Time-points) of Intracranial Pressure (ICP) > 20 mmHg
Duration (Number of Hours) of Cerebral Perfusion Pressure (CPP) < 60 mmHg
Duration (number of hours) of cerebral perfusion pressure (CPP) < 60 mmHg calculated from ICP and mean arterial blood pressure (MAP): CPP = MAP - ICP)
Therapy Intensity Level Score
Therapy Intensity Level Score: Total Score calculated daily as the sum of all individual measures, range from 3 (good outcome) to 50 (worst outcome):
Scores:
0-2 Head elevation 0-8 Sedation 0-1 Paralysis 1-3 Hyperventilation 0-2 Increased Oxygenation 1-3 Cooling 0-2 Osmotherapy 0-3 CSF Drainage 0-1 Red Blood Cell Transfusion 1-3 Cerebral perfusion pressure 0-1 Surgery for mass lesion 0/5/10 none/unilateral/bilateral Decompressive Craniectomy 0/10 Laparatomy to treat intracranial hypertension due to abdominal hypertension
Full Information
NCT ID
NCT02012582
First Posted
December 10, 2013
Last Updated
February 8, 2016
Sponsor
veriNOS operations GmbH
1. Study Identification
Unique Protocol Identification Number
NCT02012582
Brief Title
Phase 2 a Study to Assess Safety and Pharmacokinetics of VAS203 in Patients With Traumatic Brain Injury
Acronym
NOSTRA
Official Title
An Exploratory Phase 2 a Study to Assess Safety, Tolerability, Pharmacodynamic and Pharmacokinetics of VAS203 in Patients With Moderate and Severe Traumatic Brain Injury
Study Type
Interventional
2. Study Status
Record Verification Date
February 2016
Overall Recruitment Status
Completed
Study Start Date
November 2009 (undefined)
Primary Completion Date
June 2012 (Actual)
Study Completion Date
September 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
veriNOS operations GmbH
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
In the exploratory multi-center Phase 2 a study safety, tolerability, pharmacodynamics and pharmacokinetics of the Nitric Oxide Synthase inhibitor VAS203 is assessed in patients with moderate and severe traumatic brain injury. Traumatic brain injury patients (32 males) receive 15, 20 and 30 mg/kg VAS203, respectively, by continuous infusion in three cohorts (Cohort 1 open; Cohorts 2 and 3 double blind, randomised placebo-controlled). End of Study for all patients will be Day 14; adverse events and concomitant medications will be documented throughout the study.
Objectives are to assess safety and tolerability of VAS203, to evaluate concentrations of metabolites of VAS203 in plasma and microdialysate and to assess pharmacodynamic effects of VAS203 on surrogate parameters. Safety parameter will include vital signs (blood pressure heart rate, respiration rate, oxygen saturation and blood gases), fluid balance, ECG, laboratory examinations (clinical chemistry, liver function, haematology/coagulation, urinalysis, renal parameters) and adverse events. Concentration of VAS203 will be determined in plasma and microdialysate. Pharmacodynamic parameters will include intracranial pressure (ICP), biochemical parameters in microdialysate (nitrite/nitrate, arginine, citrulline, pyruvate, lactate, glucose), Partial Oxygen Pressure in brain parenchyma and Therapy Intensity Level (TIL).
Detailed Description
Intravenous administration of study medication for each patient will start as soon as possible but not later than 12 hours after trauma. Patients in Group 1 will receive open label study drug (VAS203). Patients in Group 2 and 3 will be randomised to treatment with VAS203 or placebo.
In addition to study treatment, each patient will receive the best "standard of care" for the study centre; no treatment will be withheld.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Traumatic Brain Injury
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
32 (Actual)
8. Arms, Groups, and Interventions
Arm Title
VAS203 15 mg/kg
Arm Type
Experimental
Arm Description
Three 5 mg/kg/12-hours infusion with 12 hours break after each infusion. Total dose: 15 mg/kg
Arm Title
VAS203 20 mg/kg
Arm Type
Experimental
Arm Description
10 mg/kg/24 hours, 48 hour continuous infusion. Total dose: 20 mg/kg
Arm Title
VAS203 30 mg/kg
Arm Type
Experimental
Arm Description
10 mg/kg/24 hours, 72 hour continuous infusion. Total dose: 30 mg/kg
Arm Title
Saline
Arm Type
Placebo Comparator
Arm Description
0.9 % Sodium chloride infusion
Intervention Type
Drug
Intervention Name(s)
VAS203
Other Intervention Name(s)
Ronopterin
Intervention Description
i.v. infusion
Intervention Type
Drug
Intervention Name(s)
Saline
Other Intervention Name(s)
0.9 % NaCl
Intervention Description
i.v. infusion
Primary Outcome Measure Information:
Title
Safety and Tolerability of VAS203 in Patients With Moderate and Severe TBI
Description
Tolerability (good, satisfactory, sufficient, poor) of VAS203 in patients with moderate and severe TBI, as judged by the investigators at day 14.
Safety outcome measure description see safety section
Time Frame
14 days
Secondary Outcome Measure Information:
Title
Duration (Number of Time-points) of Intracranial Pressure (ICP) > 20 mmHg
Time Frame
Hourly from start of infusion to 144 hours
Title
Duration (Number of Hours) of Cerebral Perfusion Pressure (CPP) < 60 mmHg
Description
Duration (number of hours) of cerebral perfusion pressure (CPP) < 60 mmHg calculated from ICP and mean arterial blood pressure (MAP): CPP = MAP - ICP)
Time Frame
Hourly from start of infusion to 144 hours
Title
Therapy Intensity Level Score
Description
Therapy Intensity Level Score: Total Score calculated daily as the sum of all individual measures, range from 3 (good outcome) to 50 (worst outcome):
Scores:
0-2 Head elevation 0-8 Sedation 0-1 Paralysis 1-3 Hyperventilation 0-2 Increased Oxygenation 1-3 Cooling 0-2 Osmotherapy 0-3 CSF Drainage 0-1 Red Blood Cell Transfusion 1-3 Cerebral perfusion pressure 0-1 Surgery for mass lesion 0/5/10 none/unilateral/bilateral Decompressive Craniectomy 0/10 Laparatomy to treat intracranial hypertension due to abdominal hypertension
Time Frame
Daily from day 1 to day 6
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:
Written informed consent from patient's legal guardian or legal representative
18 - 65 years of age, inclusive
Head trauma within the last 12 hours
Traumatic brain injury with Glasgow Coma Score (GCS) ≥ 5 and that requires ICP monitoring
Catheter placement for monitoring and management of increased ICP
Microdialysis probe placement in penumbra zone or ipsilateral to contusion if focal
Systolic blood pressure ≥ 100 mmHg
Females of child-bearing potential must have a negative pregnancy test
Exclusion Criteria:
Penetrating head injury (e.g. missile, stab wound)
Not expected to survive more than 24 hours after admission
Concurrent, but not pre-existing, spinal cord injury
Unilateral and bilateral fixed and dilated pupil (> 4 mm)
Cardiopulmonary resuscitation performed post injury
continuing bleeding likely to require multiple transfusions (> 4 units red blood cells)
Coma due to a "pure" epidural hematoma (lucid interval and absence of structural brain damage on CT scan)
Coma suspected to be primarily due to other causes than head injury (e.g. drug overdose)
Known or CT scan evidence of pre-existing major cerebral damage
Decompressive craniectomy, planned prior to randomization
Polytraumatic patients with Injury Severity Score > 25, or rhabdomyolysis
Injuries to ascending aorta and/or carotid arteries
serum creatinine values > 1.5 mg/dL
estimated Glomerular Filtration Rate < 60 ml/min (MDRD-formula)
body mass index (BMI) > 35, Body weight > 120 kg
Any severe concomitant condition (cancer; hematologic, renal, hepatic, coronary disease; major psychiatric disorder; alcohol or drug abuse), that can be ascertained at admission
Known to have received an experimental drug within 4 weeks prior to current injury
Administration of > 100 ml of contrast media containing iodine
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Frank Tegtmeier, Dr.
Organizational Affiliation
veriNOS operations GmbH
Official's Role
Study Director
Facility Information:
Facility Name
Medical University Innsbruck Department of Neurology
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
HIA Sainte-Anne Boulevard Sainte-Anne
City
Toulon
ZIP/Postal Code
83800
Country
France
Facility Name
Vall d'Hebron University Hospital Department of Neurosurgery
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Clinic University of Barcelona Surgical Intensive Care Unit
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
University Hospital Zuerich Surgical Intensive Care
City
Zuerich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
Southampton University Hospital Division of Clinical Neurosciences
City
Southampton
ZIP/Postal Code
SO16 6YD
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
24831445
Citation
Stover JF, Belli A, Boret H, Bulters D, Sahuquillo J, Schmutzhard E, Zavala E, Ungerstedt U, Schinzel R, Tegtmeier F; NOSTRA Investigators. Nitric oxide synthase inhibition with the antipterin VAS203 improves outcome in moderate and severe traumatic brain injury: a placebo-controlled randomized Phase IIa trial (NOSTRA). J Neurotrauma. 2014 Oct 1;31(19):1599-606. doi: 10.1089/neu.2014.3344. Epub 2014 Jul 28.
Results Reference
result
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Phase 2 a Study to Assess Safety and Pharmacokinetics of VAS203 in Patients With Traumatic Brain Injury
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