Back to resultsA Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms
Primary Purpose
Spasms, Infantile
Status
Withdrawn
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
AMZ002 injectable solution, 0.5mg/mL
Vigabatrin, oral
Sponsored by
About this trial
This is an interventional treatment trial for Spasms, Infantile focused on measuring AMZ002, Vigabatrin, Seizures, Electroencephalograms, Infantile spasms, West syndrome, Central Nervous System Diseases, Epileptic Syndrome
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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;
- 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
Sites / Locations
- The Childrens Hospital Colorado
- Miami Children's Hospital
- Advent Health Orlando
- Arnold Palmer Hospital For Children
- Childrens Healthcare of Atlanta
- Ann and Robert H. Lurie Children's Hospital of Chicago
- University of Kentucky Hospital
- Children's Brain Institute
- Children's Hospital of Michigan
- Staten Island University Hospital, North
- Wake Forest Baptist Medical Center - PPDS
- UT Southwestern Medical Center
- Fundacion para la Lucha contra las Enfermedades Neurologicas de la Infancia
- Hospital Italiano de Buenos Aires
- Paediatric Hospital Dr. Juan P. Garrahan
- CHU Bordeaux - Hopital des Enfants
- HFME-Hospices Civils de Lyon
- Hopital Roger Salengro - CHU de Lille
- Hopital Necker - Enfants Malades
- Hopital de Hautepierre
- All India Institute of Medical Sciences
- Indira Gandhi Institute of Child Health
- Amrita Advanced Centre for Epilepsy
- Bharati Vidyapeeth Deemed University - Bharati Hospital
- Azienda Ospedaliero Universitaria
- Pediatric Hospital G. Salesi
- AOU Anna Meyer - Clinica di Neurologia Pediatrica
- Istituto Pediatrico Giannina Gaslini
- Ospedale dei Bambini Vittore Buzzi
- Universita degli Studi di Napoli Federico II
- Ospedale Pediatrico Bambino Gesu
- Policlinico Universitario A.Gemelli
- Azienda Ospedaliera Universitaria Integrata
- Neurociencias Estudios Clinicos S.C.
- HSRT
- Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia
- The University Clinical Center
- Provincial Specialist Children's Hospital st. Ludwika
- Medical University of Warsaw
- Institute of Mother and Child
- Hospital de la Santa Creu i Sant Pau
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
AMZ002
Vigabatrin
Arm Description
Outcomes
Primary Outcome Measures
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) 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
Secondary Outcome Measures
Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
Proportion of Participants with Recurrence of Clinical Spasms After Initial Response as Assessed by Video EEG/ by Parent/Guardian Diary
Time from Initial Response to Recurrence of Clinical Spasms
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05128344
Brief Title
A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Withdrawn
Why Stopped
Strategic decision
Study Start Date
April 2023 (Anticipated)
Primary Completion Date
June 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amzell
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spasms, Infantile
Keywords
AMZ002, Vigabatrin, Seizures, Electroencephalograms, Infantile spasms, West syndrome, Central Nervous System Diseases, Epileptic Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AMZ002
Arm Type
Experimental
Arm Title
Vigabatrin
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
AMZ002 injectable solution, 0.5mg/mL
Intervention Description
Injectable solution
Intervention Type
Drug
Intervention Name(s)
Vigabatrin, oral
Intervention Description
Oral administration
Primary Outcome Measure Information:
Title
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14
Time Frame
At Day 14
Title
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
Secondary Outcome Measure Information:
Title
Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
Time Frame
Baseline up to Day 58
Title
Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
Time Frame
At Day 14
Title
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
Title
Time from Initial Response to Recurrence of Clinical Spasms
Time Frame
From Day 14 up to Day 58
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
24 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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;
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
Facility Information:
Facility Name
The Childrens Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045-7106
Country
United States
Facility Name
Miami Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Advent Health Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Arnold Palmer Hospital For Children
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Childrens Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Kentucky Hospital
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Children's Brain Institute
City
Lexington
State/Province
Massachusetts
ZIP/Postal Code
02420
Country
United States
Facility Name
Children's Hospital of Michigan
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201-2119
Country
United States
Facility Name
Staten Island University Hospital, North
City
Staten Island
State/Province
New York
ZIP/Postal Code
10305
Country
United States
Facility Name
Wake Forest Baptist Medical Center - PPDS
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Facility Name
Fundacion para la Lucha contra las Enfermedades Neurologicas de la Infancia
City
Buenos Aires
Country
Argentina
Facility Name
Hospital Italiano de Buenos Aires
City
Buenos Aires
Country
Argentina
Facility Name
Paediatric Hospital Dr. Juan P. Garrahan
City
Buenos Aires
Country
Argentina
Facility Name
CHU Bordeaux - Hopital des Enfants
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
HFME-Hospices Civils de Lyon
City
Bron
ZIP/Postal Code
69500
Country
France
Facility Name
Hopital Roger Salengro - CHU de Lille
City
Lille
Country
France
Facility Name
Hopital Necker - Enfants Malades
City
Paris
Country
France
Facility Name
Hopital de Hautepierre
City
Strasbourg
Country
France
Facility Name
All India Institute of Medical Sciences
City
New Delhi
State/Province
Dehli
ZIP/Postal Code
110029
Country
India
Facility Name
Indira Gandhi Institute of Child Health
City
Bangalore
State/Province
Karnataka
ZIP/Postal Code
560029
Country
India
Facility Name
Amrita Advanced Centre for Epilepsy
City
Kochi
State/Province
Kerala
ZIP/Postal Code
682041
Country
India
Facility Name
Bharati Vidyapeeth Deemed University - Bharati Hospital
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411043
Country
India
Facility Name
Azienda Ospedaliero Universitaria
City
Ancona
Country
Italy
Facility Name
Pediatric Hospital G. Salesi
City
Ancona
Country
Italy
Facility Name
AOU Anna Meyer - Clinica di Neurologia Pediatrica
City
Firenze
Country
Italy
Facility Name
Istituto Pediatrico Giannina Gaslini
City
Genova
Country
Italy
Facility Name
Ospedale dei Bambini Vittore Buzzi
City
Milano
Country
Italy
Facility Name
Universita degli Studi di Napoli Federico II
City
Neapel
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesu
City
Roma
Country
Italy
Facility Name
Policlinico Universitario A.Gemelli
City
Roma
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Integrata
City
Verona
Country
Italy
Facility Name
Neurociencias Estudios Clinicos S.C.
City
Culiacán
Country
Mexico
Facility Name
HSRT
City
Mexico City
Country
Mexico
Facility Name
Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia
City
Nuevo León
ZIP/Postal Code
66278
Country
Mexico
Facility Name
The University Clinical Center
City
Gdańsk
State/Province
Gdansk
Country
Poland
Facility Name
Provincial Specialist Children's Hospital st. Ludwika
City
Kraków
State/Province
Krakow
Country
Poland
Facility Name
Medical University of Warsaw
City
Warsaw
ZIP/Postal Code
02-091
Country
Poland
Facility Name
Institute of Mother and Child
City
Warsaw
Country
Poland
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
ZIP/Postal Code
8041
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms
We'll reach out to this number within 24 hrs