StrokePhase 2GMAISNCT01221246
Efficacy and Safety Study of GM602 in Patients With Acute Middle Cerebral Artery Ischemic Stroke Within 18 Hours
A Phase 2 study testing GM602 in people with Stroke, run by Genervon Biopharmaceuticals, LLC at 8 sites in United States.
Trial summary
The purpose of this research study is to determine whether the investigational drug GM602, is effective and safe in the treatment of ischemic stroke (strokes caused by a blood clot blocking the flow of blood through one, or more of the blood vessels supplying the brain) when administered up to 18 hours after symptoms… Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Stroke
- What is being tested?
- GM602
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 8 sitesUnited States
- Study dates
- 8 Mar 2011to 7 Jul 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- > 18 years old
- Be eligible for MRI or CT scan
- Have suffered acute ischemic stroke in the middle cerebral artery (MCA) distribution, as verified by the Screening diffusion-weighted imaging (DWI) abnormality and Screening perfusion-weighted imaging pressure-work index (PWI ) abnormality
- Have NIH Stroke Scale (NIHSS) score total score of 9-20 inclusive at screening
- Have suffered acute ischemic stroke within 18 hours
- Have been functionally independent with a Modified Rankin Score (mRS) of 0 or 1 prior to suffering stroke
- Patients who received tPA or FDA approved mechanical device can also enroll
- completed informed consent form
Who cannot join
- Have history of stroke in the past 3 months
- Cannot be evaluated using MRI/CT
- Have stroke of the brainstem or cerebellum
- Have clinical presentation consistent with acute MI by EKG criteria (STEMI) at screening
- Have hemorrhage revealed by CT or MRI scan
- Have > 1/3 MCA territory HYPER intensity as seen on MRI OR >1/3 MCA territory HYPO intensity as seen on CT
Show all 17Show fewer
- Have blood sugar level >400 mg/DL or<50 mg/dL
- Have kidney disease, creatinine > 2.0
- Have had recent (within 90 days) serious head trauma or head trauma with loss of consciousness
- Have any prior history of seizure
- Have clinically relevant pre-existing neurological deficit (Historical Rankin score ≥ 2)
- Have any other known clinically significant medical disorder (cardiovascular, hepatic, renal, endocrine, respiratory, immunological, cancer, AIDS)
- Life expectancy of less than 6 months due to comorbid conditions
- Women of child bearing potential who are pregnant or breast-feeding or unable to practice birth control during the study period
- Have participated in any other trial of an investigational agent within 90 days prior to screening
- Informed consent cannot be obtained
- Unable to participate in study visits
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
University Erlanger Hospital
Chattanooga, Tennessee, 37403
UCLA Stroke Center (Departments of Emergency Medicine and Neurology at the University of California, Los Angeles Medical Center)
Los Angeles, California, 90095
University of Louisville
Louisville, Kentucky, 40202
Columbia University Medical Center
New York, New York, 10032
Hoag Memorial Hospital Presbyterian
Newport Beach, California, 92658
Huntington Memorial Hospital Stroke Center
Pasadena, California, 91105
California Pacific Medical Center Research Institute
San Francisco, California, 94107
Sarasota Memorial Hospital
Sarasota, Florida, 34239
About this study
Official title: A Phase 2 Double Blinded, Randomized, Placebo Controlled Dose Escalation Study to Evaluate the Efficacy and the Safety of GM602 in Patients With Acute Middle Cerebral Artery Ischemic Stroke Within an 18-hour Treatment Window
The purpose of this research study is to determine whether the investigational drug GM602, is effective and safe in the treatment of ischemic stroke (strokes caused by a blood clot blocking the flow of blood through one, or more of the blood vessels supplying the brain) when administered up to 18 hours after symptoms begin.
Read the detailed description
Stroke is a serious and life threatening disease. About 85% of all strokes are ischemic, caused by a blood clot or plaque that blocks a blood vessel in the brain. The thrombolytic drug tissue plasminogen activator (tPA) is the only early treatment for acute ischemic stroke approved by the FDA. Treatment with tPA must be administered within three hours of the stroke onset. Furthermore, tPA treatment carries a recognized risk of bleeding in the brain. GM602 is an investigational drug that may act as a neuroprotectant in patients who have had a stroke. It is thought to stop cell death and reduce inflammation in the injured area of the brain. This study is designed to evaluate the safety and efficacy of GM602 administered intravenously to patients in three consecutive daily doses of 320 mg/dose or 480 mg/dose, the initial dose administered within 18 hours after onset of acute ischemic stroke in the Middle Cerebral artery region.
- Conditions
- Stroke
- Being tested
- GM602, Placebo Comparator
- Keywords
- Acute Middle Cerebral Artery Ischemic Stroke within 18 hours
- Medical subject headings
- Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GM602Drug
Also called GM6, GM604, GM608, MNTF
First 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 12 moderate and 12 severe patients will receive GM602.
Placebo ComparatorDrug
Also called Bacteriostatic saline
First 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or Matching Placebo (Bacteriostatic Saline) for GM602 in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 6 moderate and 6 severe patients will receive placebo.
| Study group | Role | What they receive |
|---|---|---|
| GM602 | Experimental | First 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 12 moderate and 12 severe patients will receive GM602. Receives: Drug: GM602 |
| Placebo Comparator | Placebo comparator | First 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio; then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 6 moderate and 6 severe patients receive Placebo. Receives: Drug: Placebo Comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional Outcome as measured by the difference in percent change in NIHSS from baseline to 90 days in patients treated with GM602 within 18 hours compared to treated with placebo as primary efficacy endpoint
NIH Stroke Scale is a standardized neurological examination intended to describe the neurological deficits found in large groups of stroke patients participating in treatment trials. Percent change from baseline in NIHSS is calculated and compared.
Measured: Day 90
Secondary outcomes (6)
Functional Outcome as measured by the difference in percent change in NIHSS from baseline to 30 days in patients treated with GM602 compared to treated with placebo
Percent change in NIHSS from baseline to 30 days in patients treated with any active dose of GM602 compared with placebo
Measured: Day 30
Percent change in Barthel Index (BI) from baseline to 90 days in patients treated with GM602 compared to treated with placebo
The Barthel Index is measured using both historical and direct observational information. It measures self-care and mobility and will help define the degree of residual disability. Percent change from baseline in BI is calculated.
Measured: Day 90
Percent change in Barthel Index (BI) from baseline to 30 days in patients treated with GM602 compared to treated with placebo
Percent change in BI from baseline to 30 days in patients treated with any active dose of GM602 compared with placebo
Measured: Day 30
Proportion of patients treated with any active dose of GM602 compared with placebo at each mRS level at 90 days
compared with placebo at each modified Rankin Scale (mRS) level at 90 days.
Measured: Day 90
Proportion of patients treated with any active dose of GM602 compared with placebo at each mRS level at 30 days
compared with placebo at each modified Rankin Scale (mRS) level at 30 days.
Measured: Day 30
Secondary safety endpoint as measured by all cause mortality data through 3 months for patients treated with GM602 compared with placebo
all cause mortality data through 3 months
Measured: Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2010 (estimated)
- Study start8 Mar 2011
- Primary completion7 Jul 2016
- Study completion7 Jul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for StrokeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genervon Biopharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of California, Los Angeles, Huntington Hospital, Hoag Memorial Hospital Presbyterian, Columbia University, California Pacific Medical Center Research Institute, University Hospital Erlangen, Sarasota Memorial Hospital, University of Louisville
- Responsible party
- Sponsor
- Investigators
- Arbi G Ohanian, MD, Principal investigator, Huntington Memorial HospitalSidney Starkman, MD, Principal investigator, UCLA Stroke CenterJeff Saver, MD, Principal investigator, UCLA Stroke CenterDavid Brown, MD, Principal investigator, Hoag Memorial Hospital PresbyterianStephan A Mayer, M.D., Principal investigator, Columbia UniversityNobl Barazangi, M.D., Principal investigator, California Pacific Medical Center Research InstituteThomas G Devlin, M.D., Principal investigator, University Erlanger HospitalMauricio Concha, M.D., Principal investigator, Sarasota Memorial Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01221246
- Sponsor's protocol ID
- GEN-002
- Acronym
- GMAIS
- Status last verified
- Jul 2019
- First submitted
- 11 Oct 2010
- Last updated
- 12 Aug 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Double Blinded, Randomized, Placebo Controlled Dose Escalation Study to Evaluate the Efficacy and the Safety of GM602 in Patients With Acute Middle Cerebral Artery Ischemic Stroke Within an 18-hour Treatment Window
- ClinicalTrials.gov ID
- NCT01221246
- Acronym / sponsor ID
- GMAIS / GEN-002
- Conditions
- Stroke
- Keywords
- Acute Middle Cerebral Artery Ischemic Stroke within 18 hours
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Genervon Biopharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Collaborators
- University of California, Los Angeles, Huntington Hospital, Hoag Memorial Hospital Presbyterian, Columbia University, California Pacific Medical Center Research Institute, University Hospital Erlangen, Sarasota Memorial Hospital, University of Louisville
- Study start
- 8 Mar 2011 (actual)
- Primary completion
- 7 Jul 2016 (actual)
- Study completion
- 7 Jul 2016 (actual)
- First posted
- 14 Oct 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is to determine whether the investigational drug GM602, is effective and safe in the treatment of ischemic stroke (strokes caused by a blood clot blocking the flow of blood through one, or more of the blood vessels supplying the brain) when administered up to 18 hours after symptoms begin.
Detailed description
Stroke is a serious and life threatening disease. About 85% of all strokes are ischemic, caused by a blood clot or plaque that blocks a blood vessel in the brain. The thrombolytic drug tissue plasminogen activator (tPA) is the only early treatment for acute ischemic stroke approved by the FDA. Treatment with tPA must be administered within three hours of the stroke onset. Furthermore, tPA treatment carries a recognized risk of bleeding in the brain. GM602 is an investigational drug that may act as a neuroprotectant in patients who have had a stroke. It is thought to stop cell death and reduce inflammation in the injured area of the brain. This study is designed to evaluate the safety and efficacy of GM602 administered intravenously to patients in three consecutive daily doses of 320 mg/dose or 480 mg/dose, the initial dose administered within 18 hours after onset of acute ischemic stroke in the Middle Cerebral artery region.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GM602 | Experimental | Drug: GM602First 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 12 moderate and 12 severe patients will receive GM602. |
| Placebo Comparator | Placebo comparator | Drug: Placebo ComparatorFirst 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio; then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 6 moderate and 6 severe patients receive Placebo. |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- > 18 years old
- Be eligible for MRI or CT scan
- Have suffered acute ischemic stroke in the middle cerebral artery (MCA) distribution, as verified by the Screening diffusion-weighted imaging (DWI) abnormality and Screening perfusion-weighted imaging pressure-work index (PWI ) abnormality
- Have NIH Stroke Scale (NIHSS) score total score of 9-20 inclusive at screening
- Have suffered acute ischemic stroke within 18 hours
Key exclusion criteria (17 listed)
- Have history of stroke in the past 3 months
- Cannot be evaluated using MRI/CT
- Have stroke of the brainstem or cerebellum
- Have clinical presentation consistent with acute MI by EKG criteria (STEMI) at screening
- Have hemorrhage revealed by CT or MRI scan
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GM602Also: GM6, GM604, GM608, MNTFFirst 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or placebo in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 12 moderate and 12 severe patients will receive GM602. | Drug | GM602 |
| Placebo ComparatorAlso: Bacteriostatic salineFirst 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 320 mg/dose of GM602 or Matching Placebo (Bacteriostatic Saline) for GM602 in a 2:1 ratio, then the next 9 moderate patients (either co-treated or not co-treated) will be randomized to receive 480 mg/dose of GM602 or placebo in a 2:1 ratio. Concurrently, 18 severe patients will be randomized in the same manner. Total 6 moderate and 6 severe patients will receive placebo. | Drug | Placebo Comparator |
Primary outcome
Functional Outcome as measured by the difference in percent change in NIHSS from baseline to 90 days in patients treated with GM602 within 18 hours compared to treated with placebo as primary efficacy endpoint
Time frame: Day 90
NIH Stroke Scale is a standardized neurological examination intended to describe the neurological deficits found in large groups of stroke patients participating in treatment trials. Percent change from baseline in NIHSS is calculated and compared.
6 secondary outcomes
Functional Outcome as measured by the difference in percent change in NIHSS from baseline to 30 days in patients treated with GM602 compared to treated with placebo
Time frame: Day 30
Percent change in Barthel Index (BI) from baseline to 90 days in patients treated with GM602 compared to treated with placebo
Time frame: Day 90
Percent change in Barthel Index (BI) from baseline to 30 days in patients treated with GM602 compared to treated with placebo
Time frame: Day 30
Proportion of patients treated with any active dose of GM602 compared with placebo at each mRS level at 90 days
Time frame: Day 90
Proportion of patients treated with any active dose of GM602 compared with placebo at each mRS level at 30 days
Time frame: Day 30
Secondary safety endpoint as measured by all cause mortality data through 3 months for patients treated with GM602 compared with placebo
Time frame: Day 90
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Arbi G Ohanian, MD principal investigator
Huntington Memorial Hospital
Sidney Starkman, MD principal investigator
UCLA Stroke Center
Jeff Saver, MD principal investigator
UCLA Stroke Center
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GM602 in people with Stroke, run by Genervon Biopharmaceuticals, LLC at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional Outcome as measured by the difference in percent change in NIHSS from baseline to 90 days in patients treated with GM602 within 18 hours compared to treated with placebo as primary efficacy endpoint (time frame: Day 90). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2019.