Clinical trial NCT05128344: 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
Overall status: WITHDRAWN
Phase: PHASE3
Conditions: Spasms, Infantile
Keywords: AMZ002
Vigabatrin
Seizures
Electroencephalograms
Infantile spasms
West syndrome
Central Nervous System Diseases
Epileptic Syndrome
Lead sponsor: Amzell
Sponsor class: INDUSTRY
First posted: 2021-11-22
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14
timeFrame: At Day 14
measure: Proportion of Participants with Resolution of Hypsarrhythmia or Significant Abnormality Compatible with Infantile Spasms (IS) as Assessed by Video EEG at Day 14
timeFrame: At Day 14
Secondary outcomes:
measure: Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary
timeFrame: Baseline up to Day 58
measure: Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14
timeFrame: At Day 14
measure: Proportion of Participants with Recurrence of Clinical Spasms After Initial Response as Assessed by Video EEG/ by Parent/Guardian Diary
timeFrame: From Day 14 up to Day 58
measure: Time from Initial Response to Recurrence of Clinical Spasms
timeFrame: From Day 14 up to Day 58
Other outcomes:
Locations:
city: Aurora
country: United States
facility: The Childrens Hospital Colorado
state: Colorado
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Advent Health Orlando
state: Florida
city: Orlando
country: United States
facility: Arnold Palmer Hospital For Children
state: Florida
city: Atlanta
country: United States
facility: Childrens Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Lexington
country: United States
facility: University of Kentucky Hospital
state: Kentucky
city: Lexington
country: United States
facility: Children's Brain Institute
state: Massachusetts
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Staten Island
country: United States
facility: Staten Island University Hospital, North
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Medical Center - PPDS
state: North Carolina
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Buenos Aires
country: Argentina
facility: Fundacion para la Lucha contra las Enfermedades Neurologicas de la Infancia
city: Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: Buenos Aires
country: Argentina
facility: Paediatric Hospital Dr. Juan P. Garrahan
city: Bordeaux
country: France
facility: CHU Bordeaux - Hopital des Enfants
city: Bron
country: France
facility: HFME-Hospices Civils de Lyon
city: Lille
country: France
facility: Hopital Roger Salengro - CHU de Lille
city: Paris
country: France
facility: Hopital Necker - Enfants Malades
city: Strasbourg
country: France
facility: Hopital de Hautepierre
city: New Delhi
country: India
facility: All India Institute of Medical Sciences
state: Dehli
city: Bangalore
country: India
facility: Indira Gandhi Institute of Child Health
state: Karnataka
city: Kochi
country: India
facility: Amrita Advanced Centre for Epilepsy
state: Kerala
city: Pune
country: India
facility: Bharati Vidyapeeth Deemed University - Bharati Hospital
state: Maharashtra
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria
city: Ancona
country: Italy
facility: Pediatric Hospital G. Salesi
city: Firenze
country: Italy
facility: AOU Anna Meyer - Clinica di Neurologia Pediatrica
city: Genova
country: Italy
facility: Istituto Pediatrico Giannina Gaslini
city: Milano
country: Italy
facility: Ospedale dei Bambini Vittore Buzzi
city: Neapel
country: Italy
facility: Universita degli Studi di Napoli Federico II
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Roma
country: Italy
facility: Policlinico Universitario A.Gemelli
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Integrata
city: Culiacán
country: Mexico
facility: Neurociencias Estudios Clinicos S.C.
city: Mexico City
country: Mexico
facility: HSRT
city: Nuevo León
country: Mexico
facility: Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia
city: Gdańsk
country: Poland
facility: The University Clinical Center
state: Gdansk
city: Kraków
country: Poland
facility: Provincial Specialist Children's Hospital st. Ludwika
state: Krakow
city: Warsaw
country: Poland
facility: Medical University of Warsaw
city: Warsaw
country: Poland
facility: Institute of Mother and Child
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
Study officials:
References: