Spasms, InfantilePhase 3NCT05128344
A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms
A Phase 3 study testing AMZ002 injectable solution, 0.5mg/mL and Vigabatrin, oral in people with Spasms, Infantile, run by Amzell at 41 sites in 8 countries.
Trial summary
The main purpose of this study is to evaluate the efficacy of AMZ002 compared to Vigabatrin in participants with newly diagnosed infantile spasms (IS). Read more about this study
- Who is it for?
- Ages 2 to 24 months
- Conditions the study is for
- Spasms, Infantile
- What is being tested?
- AMZ002 injectable solution, 0.5mg/mL and Vigabatrin, oral
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 41 sitesin 8 countries
- Study dates
- Apr 2023to Dec 2025
- Phase
- Phase 3
- 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 2 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant has been diagnosed with IS within 6 weeks prior to Screening. Diagnostic criteria include both clinical spasms and an EEG pattern consistent with hypsarrhythmia or significant abnormality compatible with IS
- Participant has normal renal function as defined by an estimated glomerular filtration rate (eGFR) greater than [>] 60 milliliter/minute (mL/min)/1.73 m^2, calculated as eGFR = 0.413 × (height [Centimeter (cm)]/serum creatinine [milligrams per deciliter {mg/dL}])
- Participant's legally authorized representative (that is [i.e.], parent or guardian) must provide written informed consent obtained per Institutional Review Board (IRB) policy and requirements, consistent with the International Council for Harmonisation (ICH)
- Participant's parent/guardian is able to understand and willing to comply with study procedures and restrictions
Who cannot join
- Participant has been diagnosed with tuberous sclerosis
- Participant has acute illness considered clinically significant by the Investigator within 30 days prior to Screening
- Participant has a diagnosis of recent systemic fungal infection; history of ocular herpes simplex; history of or current peptic ulcer; uncontrolled hypertension or congestive heart failure; or any other condition that would be significantly impacted by the study drug
- Participant has a preplanned surgery or procedure(s) that would interfere with the conduct of the study
- Participant has received any prior treatment for IS
- Participant has been previously treated with adrenocorticotropic hormone (ACTH), corticosteroids, or Vigabatrin for seizures;
Show all 11Show fewer
- Participant has been previously treated with a course of corticosteroids for an indication other than seizures within 30 days prior to Screening
- Participant has a known or suspected allergy to ACTH or Vigabatrin or any component of AMZ002 or Vigabatrin
- Participant has used any other investigational drug within 30 days or 5 half-lives prior to the first dose of AMZ002 or Vigabatrin (whichever is longer)
- Participant' s parent/guardian is unable to provide written informed consent and/or to complete the daily diary
- Participant has any other disease, condition, or therapy that, in the opinion of the Investigator, might compromise safety or compliance, preclude the participant from successfully completing the study, or interfere with the interpretation of the results
Trial locations and contacts
41 sites in 8 countries.
41 sites in 8 countries
United States12 sites
Childrens Healthcare of Atlanta
Atlanta, Georgia, 30329
The Childrens Hospital Colorado
Aurora, Colorado, 80045-7106
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
UT Southwestern Medical Center
Dallas, Texas, 75207
Children's Hospital of Michigan
Detroit, Michigan, 48201-2119
University of Kentucky Hospital
Lexington, Kentucky, 40536
Children's Brain Institute
Lexington, Massachusetts, 02420
Miami Children's Hospital
Miami, Florida, 33155
Advent Health Orlando
Orlando, Florida, 32803
Arnold Palmer Hospital For Children
Orlando, Florida, 32806
Staten Island University Hospital, North
Staten Island, New York, 10305
Wake Forest Baptist Medical Center - PPDS
Winston-Salem, North Carolina, 27157
Italy9 sites
Azienda Ospedaliero Universitaria
Ancona
Pediatric Hospital G. Salesi
Ancona
AOU Anna Meyer - Clinica di Neurologia Pediatrica
Florence
Istituto Pediatrico Giannina Gaslini
Genova
Ospedale dei Bambini Vittore Buzzi
Milan
Universita degli Studi di Napoli Federico II
Naples
Ospedale Pediatrico Bambino Gesu
Roma
Policlinico Universitario A.Gemelli
Roma
Azienda Ospedaliera Universitaria Integrata
Verona
France5 sites
CHU Bordeaux - Hopital des Enfants
Bordeaux, 33076
HFME-Hospices Civils de Lyon
Bron, 69500
Hopital Roger Salengro - CHU de Lille
Lille
Hopital Necker - Enfants Malades
Paris
Hopital de Hautepierre
Strasbourg
India4 sites
Indira Gandhi Institute of Child Health
Bangalore, Karnataka, 560029
Amrita Advanced Centre for Epilepsy
Kochi, Kerala, 682041
All India Institute of Medical Sciences
New Delhi, National Capital Territory of Delhi, 110029
Bharati Vidyapeeth Deemed University - Bharati Hospital
Pune, Maharashtra, 411043
Poland4 sites
The University Clinical Center
Gdansk, Gdansk
Provincial Specialist Children's Hospital st. Ludwika
Krakow, Krakow
Medical University of Warsaw
Warsaw, 02-091
Institute of Mother and Child
Warsaw
Argentina3 sites
Fundacion para la Lucha contra las Enfermedades Neurologicas de la Infancia
Buenos Aires
Hospital Italiano de Buenos Aires
Buenos Aires
Paediatric Hospital Dr. Juan P. Garrahan
Buenos Aires
Mexico3 sites
Neurociencias Estudios Clinicos S.C.
Culiacán
HSRT
Mexico City
Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia
Nuevo León, 66278
About this study
Official title: A Phase 3, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of AMZ002, Compared to Vigabatrin, in the Treatment of Infantile Spasms
The main purpose of this study is to evaluate the efficacy of AMZ002 compared to Vigabatrin in participants with newly diagnosed infantile spasms (IS).
- Conditions
- Spasms, Infantile
- Being tested
- AMZ002 injectable solution, 0.5mg/mL, Vigabatrin, oral
- Keywords
- AMZ002, Vigabatrin, Seizures, Electroencephalograms, Infantile spasms, West syndrome, Central Nervous System Diseases, Epileptic Syndrome
- Medical subject headings
- Spasms, Infantile, Seizures, Central Nervous System Diseases, Epileptic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMZ002 injectable solution, 0.5mg/mLDrug
Injectable solution
Vigabatrin, oralDrug
Oral administration
| Study group | Role | What they receive |
|---|---|---|
| AMZ002 | Experimental | Not described Receives: Drug: AMZ002 injectable solution, 0.5mg/mL |
| Vigabatrin | Active comparator | Not described Receives: Drug: Vigabatrin, oral |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14
Measured: At Day 14
Proportion of Participants with Resolution of Hypsarrhythmia or Significant Abnormality Compatible with Infantile Spasms (IS) as Assessed by Video EEG at Day 14
Measured: At Day 14
Secondary outcomes (4)
Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
Measured: Baseline up to Day 58
Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
Measured: At Day 14
Proportion of Participants with Recurrence of Clinical Spasms After Initial Response as Assessed by Video EEG/ by Parent/Guardian Diary
Measured: From Day 14 up to Day 58
Time from Initial Response to Recurrence of Clinical Spasms
Measured: From Day 14 up to Day 58
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2021
- Study startApr 2023 (estimated)
- Primary completionJun 2025 (estimated)
- Study completionDec 2025 (estimated)
Why it stopped: Strategic decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spasms, InfantileStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amzell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05128344
- Sponsor's protocol ID
- AMZ002-002
- Other IDs
- 2021-003015-26
- Status last verified
- Apr 2023
- First submitted
- 9 Nov 2021
- Last updated
- 20 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of AMZ002, Compared to Vigabatrin, in the Treatment of Infantile Spasms
- ClinicalTrials.gov ID
- NCT05128344
- Acronym / sponsor ID
- AMZ002-002
- Conditions
- Spasms, Infantile
- Keywords
- AMZ002, Vigabatrin, Seizures, Electroencephalograms, Infantile spasms, West syndrome, Central Nervous System Diseases, Epileptic Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Strategic decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- AmzellIndustry (pharma, biotech, devices)
- Study start
- Apr 2023 (estimated)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 22 Nov 2021 (actual)
- 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 main purpose of this study is to evaluate the efficacy of AMZ002 compared to Vigabatrin in participants with newly diagnosed infantile spasms (IS).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AMZ002 | Experimental | Drug: AMZ002 injectable solution, 0.5mg/mL |
| Vigabatrin | Active comparator | Drug: Vigabatrin, oral |
Who may qualify
- Ages
- 2 to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Participant has been diagnosed with IS within 6 weeks prior to Screening. Diagnostic criteria include both clinical spasms and an EEG pattern consistent with hypsarrhythmia or significant abnormality compatible with IS
- Participant has normal renal function as defined by an estimated glomerular filtration rate (eGFR) greater than [>] 60 milliliter/minute (mL/min)/1.73 m^2, calculated as eGFR = 0.413 × (height [Centimeter (cm)]/serum creatinine [milligrams per deciliter {mg/dL}])
- Participant's legally authorized representative (that is [i.e.], parent or guardian) must provide written informed consent obtained per Institutional Review Board (IRB) policy and requirements, consistent with the International Council for Harmonisation (ICH)
- Participant's parent/guardian is able to understand and willing to comply with study procedures and restrictions
Key exclusion criteria (11 listed)
- Participant has been diagnosed with tuberous sclerosis
- Participant has acute illness considered clinically significant by the Investigator within 30 days prior to Screening
- Participant has a diagnosis of recent systemic fungal infection; history of ocular herpes simplex; history of or current peptic ulcer; uncontrolled hypertension or congestive heart failure; or any other condition that would be significantly impacted by the study drug
- Participant has a preplanned surgery or procedure(s) that would interfere with the conduct of the study
- Participant has received any prior treatment for IS
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 |
|---|---|---|
| AMZ002 injectable solution, 0.5mg/mLInjectable solution | Drug | AMZ002 |
| Vigabatrin, oralOral administration | Drug | Vigabatrin |
Primary outcome
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14
Time frame: At Day 14
Proportion of Participants with Resolution of Hypsarrhythmia or Significant Abnormality Compatible with Infantile Spasms (IS) as Assessed by Video EEG at Day 14
Time frame: At Day 14
4 secondary outcomes
Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
Time frame: Baseline up to Day 58
Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
Time frame: At Day 14
Proportion of Participants with Recurrence of Clinical Spasms After Initial Response as Assessed by Video EEG/ by Parent/Guardian Diary
Time frame: From Day 14 up to Day 58
Time from Initial Response to Recurrence of Clinical Spasms
Time frame: From Day 14 up to Day 58
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
41 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AMZ002 injectable solution, 0.5mg/mL and Vigabatrin, oral in people with Spasms, Infantile, run by Amzell at 41 sites in 8 countries.
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 4 inclusion and 11 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: withdrawn, as of the sponsor's last update on 20 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, Italy, France, India, Poland and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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?
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14 (time frame: At Day 14). The study lists 2 primary outcomes in all. 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 20 Apr 2023.