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 oppdatert av: 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
Studieoversikt
Status
Har ikke rekruttert ennå
Forhold
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Antatt)
105
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Sabine Coppieters, MD
- Telefonnummer: 857-350-4834
- E-post: clinicaltrials@argenx.com
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Nei
Beskrivelse
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.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Firemannsrom
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: Double-blinded treatment period (DBTP) - Adimanebart IV
Participants randomized to receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
|
Placebo komparator: Double-blinded treatment period (DBTP) - Placebo IV
Participants randomized to receive Placebo IV
|
Intravenous infusion of Placebo
|
|
Eksperimentell: Open-label extension (OLE) - Adimanebart IV
Participants receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Change from baseline at week 24 in 6MWT distance
Tidsramme: 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
Tidsramme: up to 104 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 104 weeks
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Change from baseline in 6MWT distance over time
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: up to 24 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 24 weeks
|
|
Adimanebart serum concentrations over time
Tidsramme: 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
Tidsramme: 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
Tidsramme: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Nab: neutralizing antibody(ies)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Antatt)
1. september 2026
Primær fullføring (Antatt)
1. oktober 2030
Studiet fullført (Antatt)
1. oktober 2030
Datoer for studieregistrering
Først innsendt
30. juli 2026
Først innsendt som oppfylte QC-kriteriene
30. juli 2026
Først lagt ut (Faktiske)
4. august 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
4. august 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
30. juli 2026
Sist bekreftet
1. juli 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- ARGX-119-17-CMS-3001
- 2025-524460-37-00 (Ctis)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .