Infantile SpasmsPhase 2NCT00442104
Open-label Extension to Protocol 1042-0500
A Phase 2 study testing Ganaxolone in people with Infantile Spasms, run by Marinus Pharmaceuticals at 14 sites in United States.
Trial summary
To allow open-label extension to patients who have completed Protocol 1042-0500 Read more about this study
- Who is it for?
- Ages 4 to 24 months
- Conditions the study is for
- Infantile Spasms
- What is being tested?
- Ganaxolone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 14 sitesUnited States
- Study dates
- Jan 2007to Mar 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 4 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Have completed all scheduled clinical study visits in the previous Protocol 1042 0500 and have been deemed eligible (no SAEs thought to be drug related and had a response to treatment) by the Investigator.
- Be diagnosed with IS regardless of etiology. Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
- Have a 24 hour vEEG recording confirming the diagnosis of IS.
- Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
- Have been previously treated with 3 AEDs or fewer.
- Have a parent/guardian who is properly informed of the nature and potential risks and benefits of the clinical study, is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study.
Who cannot join
- Current treatment with more than 2 concomitant AEDs.
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain MRI.
- Have any disease or condition (medical or surgical) at Screening that might compromise the hematologic, cardiovascular, pulmonary, renal, GI, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.
- Aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin greater than 4 times the upper limit of laboratory normal or any clinical laboratory value deemed clinically significant by the Investigator.
- History of recurrent status epilepticus.
- Have been exposed to any other investigational drug within 30 days prior to enrollment.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
University of Chicago Comer Children's Hospital
Chicago, Illinois, 60637
Dallas Pediatric Neurology Associates
Dallas, Texas, 75230
Texas Children's Hospital
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Mattel Children's Hospital at UCLA
Los Angeles, California, 90095
Le Bonheur Children's Medical Center
Memphis, Tennessee, 38105
Miami Children's Hospital, The Brain Institute
Miami, Florida, 33155
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53201
Child Neurology Center of Nrothwest Florida, P.A.
Pensacola, Florida, 32504
Virginia Commonwealth University Health Systems
Richmond, Virginia, 23298
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, 55102
Children's Hospital and Regional Medical Center
Seattle, Washington, 98105
Montefiore Medical Center- Albert Einstein College of Medicine
The Bronx, New York, 10467
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.
To allow open-label extension to patients who have completed Protocol 1042-0500
Read the detailed description
Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.
There will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.
- Conditions
- Infantile Spasms
- Being tested
- Ganaxolone
- Keywords
- infantile spasms, anticonvulsant, pediatric epilepsy, West Syndrome, epileptic spasms
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GanaxoloneDrug
| Study group | Role | What they receive |
|---|---|---|
| ganaxolone | Experimental | Not described Receives: Drug: Ganaxolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Were Free of Spasms
Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.
Measured: Weeks 4 through Week 96
Secondary outcomes (8)
Change From Baseline in Frequency of Spasm Clusters
Infantile spasms that come one after another in a cluster and lasts several minutes are called Spasm Clusters. Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.
Measured: Baseline and Week 4 through Week 32
Change From Baseline in Frequency of Individual Spasm
Individual Spasm are seizures that may last only a second or two and were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.
Measured: Baseline and Week 4 through Week 32
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Caregiver global assessment of seizure severity and response to treatment rated the participants' based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale ([1] marked improvement, [2] moderate improvement, [3] slight improvement, [4] no change from baseline, [5] slight worsening, [6] moderate worsening, or [7] marked worsening). Higher score indicated worse symptoms. The assessment compared the participants' current status to their condition prior to initiating study medication.
Measured: Week 4 through Week 32
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
The investigators rated the participants' overall clinical status based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale ([1] marked improvement, [2] moderate improvement, [3] slight improvement, [4] no change from baseline, [5] slight worsening, [6] moderate worsening, or [7] marked worsening). Higher score indicated worse symptoms. The investigators assessed the participants' status compared to their condition prior to initiating study medication.
Measured: Week 4 through Week 32
Number of Participants With Spasm-free Durations
Spasm-free duration is defined as total number of spasm-free days recorded in the spasm/seizure diary. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The number of participants with spasm-free duration of at least 24 hours have been presented.
Measured: Week 4 through Week 32
Number of Participants With Absence of Spasms
Absence of spasms is defined as percentage of total spasm-free days during a visit period=100%. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The participants achieving absence of spasms have been presented.
Measured: Week 4 through Week 32
Developmental Assessment Using Denver-II Developmental Test
Denver-II Developmental Test measures a child's development in several areas:Personal-Social,Fine Motor-Adaptive,Language,and Gross Motor,from birth to 6 years old.It consists of 125 items that are organized into subscales and scored as pass,fail,or refused.To evaluate a child's progress,test compares their performance to a normative sample of children of same age.For each item,age at which 90% of children in normative sample pass it is determined.Derived score for each subscale is sum of item scores and represents difference between child's chronological age and age at which 90% of children in normative sample pass the items in that subscale.A higher derived score on a subscale indicates better performance on items in that subscale relative to other children of same age who have taken the test.Among subscales,Personal-Social subscale ranges from -16 months to 24 months;others range from - 12 months to 24 months.All subscales have a population mean of 0 and a standard deviation of 3.
Measured: Week 8 through Week 32
Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.
Percentage of total spasm-free days during a visit period is defined as total number of spasm free days as recorded in the Seizure Diary/ total number of days during that period, multiplied by 100.
Percentage of cumulative total spasm-free days during a visit period is defined as sum of total number of spasm-free days during this period as recorded in the Seizure Diary/ total number of days in the treatment period, multiplied by 100.
Measured: Weeks 4 through Week 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2007 (estimated)
- Study startJan 2007
- Primary completionMar 2009
- Study completionMar 2009
Why it stopped: Sponsor's decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Infantile SpasmsStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00442104
- Sponsor's protocol ID
- 1042-0501
- Other IDs
- Amend 5 (ROW), Amend 6 (US)
- Status last verified
- May 2024
- First submitted
- 27 Feb 2007
- Last updated
- 28 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.
- ClinicalTrials.gov ID
- NCT00442104
- Acronym / sponsor ID
- 1042-0501
- Conditions
- Infantile Spasms
- Keywords
- infantile spasms, anticonvulsant, pediatric epilepsy, West Syndrome, epileptic spasms
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor's decision
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2007 (actual)
- Primary completion
- Mar 2009 (actual)
- Study completion
- Mar 2009 (actual)
- First posted
- 1 Mar 2007 (estimated)
- Results
- Posted 2 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To allow open-label extension to patients who have completed Protocol 1042-0500
Detailed description
Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.
There will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ganaxolone | Experimental | Drug: Ganaxolone |
Who may qualify
- Ages
- 4 to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Have completed all scheduled clinical study visits in the previous Protocol 1042 0500 and have been deemed eligible (no SAEs thought to be drug related and had a response to treatment) by the Investigator.
- Be diagnosed with IS regardless of etiology. Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
- Have a 24 hour vEEG recording confirming the diagnosis of IS.
- Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
- Have been previously treated with 3 AEDs or fewer.
Key exclusion criteria (6 listed)
- Current treatment with more than 2 concomitant AEDs.
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain MRI.
- Have any disease or condition (medical or surgical) at Screening that might compromise the hematologic, cardiovascular, pulmonary, renal, GI, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.
- Aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin greater than 4 times the upper limit of laboratory normal or any clinical laboratory value deemed clinically significant by the Investigator.
- History of recurrent status epilepticus.
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 |
|---|---|---|
| Ganaxolone | Drug | ganaxolone |
Primary outcome
Percentage of Participants Who Were Free of Spasms
Time frame: Weeks 4 through Week 96
Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.
8 secondary outcomes
Change From Baseline in Frequency of Spasm Clusters
Time frame: Baseline and Week 4 through Week 32
Change From Baseline in Frequency of Individual Spasm
Time frame: Baseline and Week 4 through Week 32
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Time frame: Week 4 through Week 32
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Time frame: Week 4 through Week 32
Number of Participants With Spasm-free Durations
Time frame: Week 4 through Week 32
Number of Participants With Absence of Spasms
Time frame: Week 4 through Week 32
Developmental Assessment Using Denver-II Developmental Test
Time frame: Week 8 through Week 32
Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.
Time frame: Weeks 4 through Week 32
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Infantile Spasms
Not yet recruitingObservational study
IBIS - Investigating Reliability of BIS and SEDLINE Monitoring in Children With Developmental and Epileptic Encephalopathies (DEE).
Epileptic Encephalopathy, Early Infantile Epileptic Encephalopathy
Active, not recruitingPhase 3
A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.
CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure
RecruitingPhase 2
Treatment of Refractory Infantile Spasms With Fenfluramine
Infantile Spasm
RecruitingObservational study
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
Epilepsy, West Syndrome
RecruitingPhase 2
A Study to Prevent Infantile Spasms Relapse
Infantile Spasms, Infantile Epileptic Spasms Syndrome
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.
The record lists no contact. Use the official record to reach the sponsor.
14 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 Ganaxolone in people with Infantile Spasms, run by Marinus Pharmaceuticals at 14 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 6 inclusion and 6 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: stopped early, as of the sponsor's last update on 28 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 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. 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?
Yes. Results are posted in the registry (first posted 2 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants Who Were Free of Spasms (time frame: Weeks 4 through Week 96). 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 28 May 2024.