A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms

Spasms, Infantile Clinical Trial

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT05128344
• Other study ID #: AMZ002-002
• Secondary ID: 2021-003015-26
• Status: Withdrawn
• Phase: Phase 3
• Start date: April 2023
• Est. completion date: December 2025

Study information

• Verified date: April 2023
• Source: Amzell
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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).

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Months to 24 Months

Eligibility

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

Related Conditions & MeSH terms

• Muscle Cramp
• Spasm
• Spasms, Infantile

Intervention

Drug:
• AMZ002 injectable solution, 0.5mg/mL
Injectable solution
• Vigabatrin, oral
Oral administration

Locations

Country	Name	City	State
Argentina	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
France	CHU Bordeaux - Hopital des Enfants	Bordeaux
France	HFME-Hospices Civils de Lyon	Bron
France	Hopital Roger Salengro - CHU de Lille	Lille
France	Hopital Necker - Enfants Malades	Paris
France	Hopital de Hautepierre	Strasbourg
India	Indira Gandhi Institute of Child Health	Bangalore	Karnataka
India	Amrita Advanced Centre for Epilepsy	Kochi	Kerala
India	All India Institute of Medical Sciences	New Delhi	Dehli
India	Bharati Vidyapeeth Deemed University - Bharati Hospital	Pune	Maharashtra
Italy	Azienda Ospedaliero Universitaria	Ancona
Italy	Pediatric Hospital G. Salesi	Ancona
Italy	AOU Anna Meyer - Clinica di Neurologia Pediatrica	Firenze
Italy	Istituto Pediatrico Giannina Gaslini	Genova
Italy	Ospedale dei Bambini Vittore Buzzi	Milano
Italy	Universita degli Studi di Napoli Federico II	Neapel
Italy	Ospedale Pediatrico Bambino Gesu	Roma
Italy	Policlinico Universitario A.Gemelli	Roma
Italy	Azienda Ospedaliera Universitaria Integrata	Verona
Mexico	Neurociencias Estudios Clinicos S.C.	Culiacán
Mexico	HSRT	Mexico City
Mexico	Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y Neurocirugia	Nuevo León
Poland	The University Clinical Center	Gdansk	Gdansk
Poland	Provincial Specialist Children's Hospital st. Ludwika	Kraków	Krakow
Poland	Institute of Mother and Child	Warsaw
Poland	Medical University of Warsaw	Warsaw
Spain	Hospital de la Santa Creu i Sant Pau	Barcelona
United States	Childrens Healthcare of Atlanta	Atlanta	Georgia
United States	The Childrens Hospital Colorado	Aurora	Colorado
United States	Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Children's Brain Institute	Lexington	Massachusetts
United States	University of Kentucky Hospital	Lexington	Kentucky
United States	Miami Children's Hospital	Miami	Florida
United States	Advent Health Orlando	Orlando	Florida
United States	Arnold Palmer Hospital For Children	Orlando	Florida
United States	Staten Island University Hospital, North	Staten Island	New York
United States	Wake Forest Baptist Medical Center - PPDS	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Amzell

Countries where clinical trial is conducted

United States,  Argentina,  France,  India,  Italy,  Mexico,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Participants with Absence of Clinical Spasms as Assessed by Video Electroencephalogram (EEG) at Day 14		At Day 14
Primary	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	Time from Treatment Initiation to Absence of Clinical Spasms as Assessed by Parent/Guardian Diary		Baseline up to Day 58
Secondary	Proportion of Participants with Absence of Clinical Spasms as Assessed by Parent/Guardian Diary at Day 14		At Day 14
Secondary	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
Secondary	Time from Initial Response to Recurrence of Clinical Spasms		From Day 14 up to Day 58