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A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS) (CoMetS)

30 juli 2026 bijgewerkt door: argenx

Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4-CMS

The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS). The study aims to determine whether adimanebart is safe and can help people with CMS feel better and perform daily activities more easily.

The study includes a double-blinded treatment period (DBTP) and an Open- label extension period (OLE). In the DBTP, all participants will be randomized in a 1:1 ratio to adimanebart or placebo. Participants who complete the DBTP will continue to the OLE.

Additionally, participants who complete part of the active-treatment period of ARGX-119-2302 study are eligible to enroll in the OLE of this study. In the OLE, all participants will receive open-label adimanebart. After final IMP dose, the participants will enter a follow-up period and their health will be monitored.

The total duration of the study is up to approximately 152 weeks (2 years and 11 months).

More information can be found here: clinicaltrials.argenx.com/Comets

Studie Overzicht

Toestand

Nog niet aan het werven

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Geschat)

105

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind
  • Volwassen
  • Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Inclusion Criteria:

DBTP:

  • At least 12 years of age.
  • Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations.
  • Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 months and agree to remain on a same stable dosing regimen of the same medication unless directed to change their CMS medication(s) by their treating physician.

OLE:

  • Completed part of the active-treatment period of ARGX-119-2302.

Exclusion Criteria:

DBTP:

  • Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.

OLE:

  • Investigational study drug discontinuation in ARGX-119-2302.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verviervoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Double-blinded treatment period (DBTP) - Adimanebart IV
Participants randomized to receive Adimanebart IV
Biologisch: Adimanebart IV
Intravenous infusion of Adimanebart
Placebo-vergelijker: Double-blinded treatment period (DBTP) - Placebo IV
Participants randomized to receive Placebo IV
Ander: Placebo IV
Intravenous infusion of Placebo
Experimenteel: Open-label extension (OLE) - Adimanebart IV
Participants receive Adimanebart IV
Biologisch: Adimanebart IV
Intravenous infusion of Adimanebart

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from baseline at week 24 in 6MWT distance
Tijdsspanne: Up to 24 weeks
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Up to 24 weeks
Incidence of AEs and SAEs
Tijdsspanne: up to 104 weeks
AE: adverse event; SAE: serious adverse event
up to 104 weeks

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from baseline in 6MWT distance over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PROMIS PF-10b T-score over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The PROMIS PF-10b T-score (Patient-Reported Outcomes Measurement Information System Physical Function 10b) is a 10- item, participant-reported short-form questionnaire designed to assess physical function. The questionnaire asks the participant to rate the items on a 5-point Likert scale of 5 (without any difficulty) to 1 (unable to do)
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in QMG key component composite score over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG. Minimum value: 0 (no disease severity); Maximum value: 15 (highest disease severity).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in 6MWT cadence over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Cadence is the number of steps taken per unit of time (steps/min) and is a measure of functional mobility.
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in the QMG key component raw values and scores over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG. Minimum value: 0 (no disease severity); Maximum value: 15 (highest disease severity).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PROMIS PF-WMA-SF T-score over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The PROMIS PF-WMA-SF T-score (Patient-Reported Outcomes Measurement Information System Physical Function With Mobility Aid Short Form) is an 11-item, participant-completed questionnaire that assesses lower and upper extremity function and associated activities of daily living. The questionnaire asks the participant to rate the items on a 5-point scale of 5 (without any difficulty) to 1 (unable to do).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in Neuro-QoL Short Form-Fatigue T-score over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Neuro-QoL Short Form-Fatigue (Quality of Life in Neurological Disorders Short Form - Fatigue) is a participant-completed short-form questionnaire designed to provide a measurement of fatigue in patients with neurological conditions and the impact of their fatigue on their quality of life and daily activities.
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in FVC over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
FVC: forced vital capacity to assess pulmonary function
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in the Actigraphy measures over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Participants will be asked to wear a wrist-worn actigraphy device to assess physical behaviour and mobility.
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PGI-C over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Patient Global Impression of Change (PGI-C) is a patient-reported rating scale to assess the change in symptom severity and functional problems since the first IMP administration. Participants are asked to rate their change in symptom severity and functional problems on a 7- point Likert scale from 1 (much improved) to 7 (much worse).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PGI-S over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Patient Global Impression of Severity (PGI-S) scale is a patient-reported rating scale to assess symptom severity and functional problems. Participants are asked to rate their symptom severity and functional problems on a 7-point Likert scale from 1 (no problems) to 7 (unable to do).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in CGI-C over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Clinical Global Impression of Change (CGI-C) scale is a clinician-reported rating scale to assess changes in a participant's symptom severity and functional problems since the first IMP administration. Clinicians are asked to rate the change in the participant's symptom severity and functional problems on a 7-point Likert scale from 1 (much improved) to 7 (much worse)
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in CGI-S over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The Clinical Global Impression of Severity (CGI-S scale) is a clinician-reported rating scale to assess a participant's symptom severity and functional problems. The scale comprises a single item. Based on clinical judgment, clinicians rate the participant's symptom severity and functional problems on a 4-point Likert scale from 0 (no problems) to 4 (unable to do).
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in EQ-5D-5L over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
The participant-completed EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status.
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of AEs and SAEs
Tijdsspanne: up to 24 weeks
AE: adverse event; SAE: serious adverse event
up to 24 weeks
Adimanebart serum concentrations over time
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
PK=pharmacokinetic(s)
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of ADA against adimanebart
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
ADA: Anti-drug antibodies
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of NAb against adimanebart
Tijdsspanne: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Nab: neutralizing antibody(ies)
up to 24 weeks (DBTP) + up to 104 weeks (OLE)

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

argenx

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 september 2026

Primaire voltooiing (Geschat)

1 oktober 2030

Studie voltooiing (Geschat)

1 oktober 2030

Studieregistratiedata

Eerst ingediend

30 juli 2026

Eerst ingediend dat voldeed aan de QC-criteria

30 juli 2026

Eerst geplaatst (Werkelijk)

4 augustus 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

4 augustus 2026

Laatste update ingediend die voldeed aan QC-criteria

30 juli 2026

Laatst geverifieerd

1 juli 2026

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • ARGX-119-17-CMS-3001
  • 2025-524460-37-00 (Ctis)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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