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 uppdaterad 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
Studieöversikt
Status
Har inte rekryterat ännu
Betingelser
Intervention / Behandling
Studietyp
Interventionell
Inskrivning (Beräknad)
105
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studiekontakt
- Namn: Sabine Coppieters, MD
- Telefonnummer: 857-350-4834
- E-post: clinicaltrials@argenx.com
Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Nej
Beskrivning
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
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Fyrdubbla
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Double-blinded treatment period (DBTP) - Adimanebart IV
Participants randomized to receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
|
Placebo-jämförare: Double-blinded treatment period (DBTP) - Placebo IV
Participants randomized to receive Placebo IV
|
Intravenous infusion of Placebo
|
|
Experimentell: Open-label extension (OLE) - Adimanebart IV
Participants receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Change from baseline at week 24 in 6MWT distance
Tidsram: 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
Tidsram: up to 104 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 104 weeks
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Change from baseline in 6MWT distance over time
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: up to 24 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 24 weeks
|
|
Adimanebart serum concentrations over time
Tidsram: 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
Tidsram: 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
Tidsram: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Nab: neutralizing antibody(ies)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Samarbetspartners och utredare
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Sponsor
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart (Beräknad)
1 september 2026
Primärt slutförande (Beräknad)
1 oktober 2030
Avslutad studie (Beräknad)
1 oktober 2030
Studieregistreringsdatum
Först inskickad
30 juli 2026
Först inskickad som uppfyllde QC-kriterierna
30 juli 2026
Första postat (Faktisk)
4 augusti 2026
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
4 augusti 2026
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
30 juli 2026
Senast verifierad
1 juli 2026
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- ARGX-119-17-CMS-3001
- 2025-524460-37-00 (Ctis)
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
NEJ
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Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Ja
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Nej
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