Study Stopped
Strategic decision
A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms
A Phase 3, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of AMZ002, Compared to Vigabatrin, in the Treatment of Infantile Spasms
2 other identifiers
interventional
N/A
8 countries
41
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Apr 2023
Typical duration for phase_3
41 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 9, 2021
CompletedFirst Posted
Study publicly available on registry
November 22, 2021
CompletedStudy Start
First participant enrolled
April 1, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2025
CompletedApril 20, 2023
April 1, 2023
2.2 years
November 9, 2021
April 18, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14
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
At Day 14
Secondary Outcomes (4)
Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
Baseline up to Day 58
Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
At Day 14
Proportion of Participants with Recurrence of Clinical Spasms After Initial Response as Assessed by Video EEG/ by Parent/Guardian Diary
From Day 14 up to Day 58
Time from Initial Response to Recurrence of Clinical Spasms
From Day 14 up to Day 58
Study Arms (2)
AMZ002
EXPERIMENTALVigabatrin
ACTIVE COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Amzelllead
Study Sites (41)
The Childrens Hospital Colorado
Aurora, Colorado, 80045-7106, United States
Miami Children's Hospital
Miami, Florida, 33155, United States
Advent Health Orlando
Orlando, Florida, 32803, United States
Arnold Palmer Hospital For Children
Orlando, Florida, 32806, United States
Childrens Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Kentucky Hospital
Lexington, Kentucky, 40536, United States
Children's Brain Institute
Lexington, Massachusetts, 02420, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201-2119, United States
Staten Island University Hospital, North
Staten Island, New York, 10305, United States
Wake Forest Baptist Medical Center - PPDS
Winston-Salem, North Carolina, 27157, United States
UT Southwestern Medical Center
Dallas, Texas, 75207, United States
Fundacion para la Lucha contra las Enfermedades Neurologicas de la Infancia
Buenos Aires, Argentina
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
Paediatric Hospital Dr. Juan P. Garrahan
Buenos Aires, Argentina
CHU Bordeaux - Hopital des Enfants
Bordeaux, 33076, France
HFME-Hospices Civils de Lyon
Bron, 69500, France
Hopital Roger Salengro - CHU de Lille
Lille, France
Hopital Necker - Enfants Malades
Paris, France
Hopital de Hautepierre
Strasbourg, France
All India Institute of Medical Sciences
New Delhi, Dehli, 110029, India
Indira Gandhi Institute of Child Health
Bangalore, Karnataka, 560029, India
Amrita Advanced Centre for Epilepsy
Kochi, Kerala, 682041, India
Bharati Vidyapeeth Deemed University - Bharati Hospital
Pune, Maharashtra, 411043, India
Azienda Ospedaliero Universitaria
Ancona, Italy
Pediatric Hospital G. Salesi
Ancona, Italy
AOU Anna Meyer - Clinica di Neurologia Pediatrica
Florence, Italy
Istituto Pediatrico Giannina Gaslini
Genova, Italy
Ospedale dei Bambini Vittore Buzzi
Milan, Italy
Universita degli Studi di Napoli Federico II
Naples, Italy
Ospedale Pediatrico Bambino Gesu
Roma, Italy
Policlinico Universitario A.Gemelli
Roma, Italy
Azienda Ospedaliera Universitaria Integrata
Verona, Italy
Neurociencias Estudios Clinicos S.C.
Culiacán, Mexico
HSRT
Mexico City, Mexico
Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia
Nuevo León, 66278, Mexico
The University Clinical Center
Gdansk, Gdansk, Poland
Provincial Specialist Children's Hospital st. Ludwika
Krakow, Krakow, Poland
Medical University of Warsaw
Warsaw, 02-091, Poland
Institute of Mother and Child
Warsaw, Poland
Hospital de la Santa Creu i Sant Pau
Barcelona, 8041, Spain
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 9, 2021
First Posted
November 22, 2021
Study Start
April 1, 2023
Primary Completion
June 1, 2025
Study Completion
December 1, 2025
Last Updated
April 20, 2023
Record last verified: 2023-04