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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04391569




Registration number
NCT04391569
Ethics application status
Date submitted
30/04/2020
Date registered
18/05/2020

Titles & IDs
Public title
Randomized Therapy In Status Epilepticus
Scientific title
A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus
Secondary ID [1] 0 0
1042-SE-3003
Universal Trial Number (UTN)
Trial acronym
RAISE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Status Epilepticus 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ganaxolone
Treatment: Drugs - Placebo

Placebo comparator: IV Placebo - Placebo bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper

Experimental: IV ganaxolone active - Ganaxolone bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper


Treatment: Drugs: Ganaxolone
Ganaxolone will be administered.

Treatment: Drugs: Placebo
Placebo will be administered.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of participants reporting SE cessation within 30 minutes of IP initiation without medications for the acute treatment of SE
Timepoint [1] 0 0
Up to 30 minutes
Primary outcome [2] 0 0
Percentage of participants with no progression to IV anesthesia for 36 hours following IP initiation
Timepoint [2] 0 0
Up to 36 hours
Secondary outcome [1] 0 0
Percentage of participants with no progression to IV anesthesia for 72 hours following IP initiation
Timepoint [1] 0 0
Up to 72 hours
Secondary outcome [2] 0 0
Time to SE Cessation following IP initiation
Timepoint [2] 0 0
Up to 48 hours

Eligibility
Key inclusion criteria
1. Participant, participant's parent, guardian, or legal authorized representative (LAR) must provide signed of informed consent/assent, and once capable (per institution guidelines), there must be documentation of consent/assent by the participant demonstrating they are willing and aware of the investigational nature of the study and related procedures. Where allowed by law, where the participant lacks the capacity to make informed decisions regarding his/her medical treatment options, the treating clinician may follow their deferred consenting practices. The clinician will make the final decision based on the best interests of the particiapant.
2. Male or females 12 years of age and older at the time of the first dose of IP
3. SE meeting the following criteria:

a. A diagnosis of SE with or without prominent motor features based on clinical and EEG findings:

i. Diagnosis is established by:
* For SE with prominent motor features: Clinical and EEG seizure activity indicative of convulsive, myoclonic or focal motor SE.
* For SE without prominent motor features (nonconvulsive SE): Appropriate clinical features and an EEG indicative of non-convulsive status epilepticus (NCSE)

ii. For any type of SE:
* At least 6 minutes of cumulative seizure activity over a 30-minute period within the hour before IP initiation, AND
* Seizure activity during the 30 minutes immediately prior to IP initiation

b. The treating clinician(s) anticipate that IV anesthesia is likely to be the next treatment for SE that persists following initiation of IP
4. Participants must have received any two or more of the following agents for treatment of the current episode of SE administered at an adequate dose and for a sufficient duration, in the judgment of the investigator, to demonstrate efficacy

* Benzodiazepines,
* IV Fosphenytoin/phenytoin,
* IV Valproic acid,
* IV Levetiracetam,
* IV Lacosamide,
* IV Brivaracetam, or
* IV Phenobarbital
5. Body mass index (BMI) < 40 or, if BMI is not able to be calculated at screening, participant is assessed by investigator as not morbidly obese
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Life expectancy of less than 24 hours
2. Anoxic brain injury or an uncorrected rapidly reversable metabolic condition as the primary cause of SE (e.g., hypoglycemia < 50 milligram per deciliter [mg/dL] or hyperglycemia > 400 mg/dL)
3. Participants who have received high-dose IV anesthetics (e.g., midazolam, propofol, thiopental, or pentobarbital) during the current episode of SE for more than 18 hours, or who continue to have clinical or electrographic evidence of persistent seizures while receiving high-dose IV anesthetics.
4. Clinical condition or advance directive that would NOT permit use of IV anesthesia
5. Participants known or suspected to be pregnant
6. Participants with known allergy or sensitivity to progesterone or allopregnanolone medications/supplements
7. Receiving a concomitant IV product containing Captisol®
8. Known or suspected hepatic insufficiency or hepatic failure leading to impaired synthetic liver function.
9. Known or suspected stage 3B (moderate to severe; estimated glomerular filtration rate [eGFR] 44-30 milliliter/minutes/1.73-meter square [mL/min/1.73m^2]), stage 4 (severe; eGFR 29-15 mL/min/1.73m^2), or stage 5 (kidney failure; eGFR < 15 mL/min/1.73m^2 or dialysis) kidney disease
10. Use of an investigational product for which less than 30 days or 5 half-lives have elapsed from the final product administration. Participation in a non-interventional clinical study does not exclude eligibility.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/10/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2024
Actual
Sample size
Target
Accrual to date
Final
124
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Marinus Research Site - Randwick
Recruitment hospital [2] 0 0
Marinus Research Site - South Brisbane
Recruitment hospital [3] 0 0
Marinus Research Site - Box Hill
Recruitment hospital [4] 0 0
Marinus Research Site - Melbourne
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3128 - Box Hill
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment postcode(s) [5] 0 0
3050 - Melbourne
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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Arkansas
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California
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Colorado
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Connecticut
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Florida
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Illinois
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Kentucky
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Louisiana
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Maryland
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Massachusetts
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Michigan
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Missouri
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New Jersey
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New Mexico
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Tennessee
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Texas
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Utah
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Virginia
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Wisconsin
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Canada
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Alberta
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Canada
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Ontario
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Canada
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Quebec
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Canada
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Saskatchewan

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Marinus Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04391569