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)
2026年7月30日 更新者：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
調査の概要
研究の種類
介入
入学 (推定)
105
段階
- フェーズ 3
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究連絡先
- 名前：Sabine Coppieters, MD
- 電話番号：857-350-4834
- メール：clinicaltrials@argenx.com
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
説明
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.
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：4倍
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Double-blinded treatment period (DBTP) - Adimanebart IV
Participants randomized to receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
|
プラセボコンパレーター：Double-blinded treatment period (DBTP) - Placebo IV
Participants randomized to receive Placebo IV
|
Intravenous infusion of Placebo
|
|
実験的：Open-label extension (OLE) - Adimanebart IV
Participants receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change from baseline at week 24 in 6MWT distance
時間枠：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
時間枠：up to 104 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 104 weeks
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change from baseline in 6MWT distance over time
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：up to 24 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 24 weeks
|
|
Adimanebart serum concentrations over time
時間枠：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
時間枠：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
時間枠：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Nab: neutralizing antibody(ies)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
スポンサー
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (推定)
2026年9月1日
一次修了 (推定)
2030年10月1日
研究の完了 (推定)
2030年10月1日
試験登録日
最初に提出
2026年7月30日
QC基準を満たした最初の提出物
2026年7月30日
最初の投稿 (実際)
2026年8月4日
学習記録の更新
投稿された最後の更新 (実際)
2026年8月4日
QC基準を満たした最後の更新が送信されました
2026年7月30日
最終確認日
2026年7月1日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- ARGX-119-17-CMS-3001
- 2025-524460-37-00 (Ctis)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
いいえ
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
はい
米国FDA規制機器製品の研究
いいえ
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