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

NCT05128344 — Phase 3, Withdrawn, Amzell

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A Phase 3, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of AMZ002, Compared to Vigabatrin, in the Treatment of Infantile Spasms

RegistryNCT05128344
StatusWithdrawn - Strategic decision
PhasePhase 3
Study typeInterventional; randomized, parallel, masking: none, treatment
SponsorAmzell (Industry)
ConditionsSpasms, Infantile
InterventionsAMZ002 injectable solution, 0.5mg/mL, Vigabatrin, oral (Drug)
Datesstart 2023-04-01 (estimated), primary completion 2025-06-01 (estimated), completion 2025-12-01 (estimated), first posted 2021-11-22, last updated 2023-04-20
Locations41 sites in United States, Argentina, France, India, Italy, Mexico, Poland, Spain

Related Papers Related Patents Related Grants Related Experts
All trials for Spasms, Infantile All trials of AMZ002 injectable solution, 0.5mg/mL All trials of Vigabatrin, oral

-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).

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+Arms and interventions

ArmTypeInterventionsDescription
AMZ002ExperimentalDrug: AMZ002 injectable solution, 0.5mg/mL
VigabatrinActive comparatorDrug: Vigabatrin, oral
  • AMZ002 injectable solution, 0.5mg/mL (Drug): Injectable solution
  • Vigabatrin, oral (Drug): Oral administration

+Outcome measures

Primary outcomes

  • 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

  • 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]

+Eligibility

Sex: All, ages: 2 Months to 24 Months

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

+Locations (41)

FacilityCityCountryStatus
The Childrens Hospital ColoradoAurora, ColoradoUnited States
Miami Children's HospitalMiami, FloridaUnited States
Advent Health OrlandoOrlando, FloridaUnited States
Arnold Palmer Hospital For ChildrenOrlando, FloridaUnited States
Childrens Healthcare of AtlantaAtlanta, GeorgiaUnited States
Ann and Robert H. Lurie Children's Hospital of ChicagoChicago, IllinoisUnited States
University of Kentucky HospitalLexington, KentuckyUnited States
Children's Brain InstituteLexington, MassachusettsUnited States
Children's Hospital of MichiganDetroit, MichiganUnited States
Staten Island University Hospital, NorthStaten Island, New YorkUnited States
Wake Forest Baptist Medical Center - PPDSWinston-Salem, North CarolinaUnited States
UT Southwestern Medical CenterDallas, TexasUnited States
Fundacion para la Lucha contra las Enfermedades Neurologicas de la InfanciaBuenos AiresArgentina
Hospital Italiano de Buenos AiresBuenos AiresArgentina
Paediatric Hospital Dr. Juan P. GarrahanBuenos AiresArgentina
CHU Bordeaux - Hopital des EnfantsBordeauxFrance
HFME-Hospices Civils de LyonBronFrance
Hopital Roger Salengro - CHU de LilleLilleFrance
Hopital Necker - Enfants MaladesParisFrance
Hopital de HautepierreStrasbourgFrance
Indira Gandhi Institute of Child HealthBangalore, KarnatakaIndia
Amrita Advanced Centre for EpilepsyKochi, KeralaIndia
Bharati Vidyapeeth Deemed University - Bharati HospitalPune, MaharashtraIndia
All India Institute of Medical SciencesNew Delhi, National Capital Territory of DelhiIndia
Azienda Ospedaliero UniversitariaAnconaItaly
Pediatric Hospital G. SalesiAnconaItaly
AOU Anna Meyer - Clinica di Neurologia PediatricaFlorenceItaly
Istituto Pediatrico Giannina GasliniGenovaItaly
Ospedale dei Bambini Vittore BuzziMilanItaly
Universita degli Studi di Napoli Federico IINaplesItaly
Ospedale Pediatrico Bambino GesuRomaItaly
Policlinico Universitario A.GemelliRomaItaly
Azienda Ospedaliera Universitaria IntegrataVeronaItaly
Neurociencias Estudios Clinicos S.C.CuliacánMexico
HSRTMexico CityMexico
Tecnologico De Monterrey - Hospital Zambrano Hellion - Instituto de Neurologia y NeurocirugiaNuevo LeónMexico
The University Clinical CenterGdansk, GdanskPoland
Provincial Specialist Children's Hospital st. LudwikaKrakow, KrakowPoland
Medical University of WarsawWarsawPoland
Institute of Mother and ChildWarsawPoland
Hospital de la Santa Creu i Sant PauBarcelonaSpain