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A Trial to Compare Treatment With Surlorian (ARM210, S48168) to Placebo in Effects on Muscle Strength and Safety in Adults With Autosomal Dominant RYR1-related Myopathy

2026年7月13日 更新者：RyCarma Therapeutics, Inc.

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Efficacy and Safety of Surlorian (ARM210, S48168) in Adults With Autosomal Dominant RYR1-Related Myopathy

This study is testing a medicine called surlorian in adults who have a genetic muscle condition known as autosomal dominant RYR1-related myopathy (RYR1-RM). The goal is to find out whether surlorian improves muscle weakness, and whether it is safe and well tolerated.

調査の概要

状態

募集

条件

介入・治療

詳細な説明

The study is taking place at several medical centers with doctors who specialize in treating people with RYR1-RM. Everyone in the study will receive both surlorian and a placebo (a "dummy" treatment) at different times, but neither the participants nor the study staff will know which one they are getting during each period.

During Treatment period 1, participants will be randomly assigned to receive either surlorian or a placebo, which will be followed up by a washout period. Following the washout, in Treatment period 2, participants will switch and receive the opposite treatment from what they received in the first period. This main part of the study lasts about 16 weeks.

After finishing the main, placebo-controlled part of the study participants may be able to join an open-label extension lasting approximately 12 months. In this extension, everyone receives surlorian.

研究の種類

介入

入学 (推定)

28

段階

  • フェーズ2

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

研究場所

  • イギリス
      • London、イギリス、WC1N 3BG
        • 募集
        • University College Hospital - PPDS
    • Shropshire
      • Oswestry、Shropshire、イギリス、SY10 7AG
        • まだ募集していません
        • The Robert Jones and Agnes Hunt Orthopaedic Hospital
  • オランダ
    • Gelderland
      • Nijmegen、Gelderland、オランダ、6500 HB
        • まだ募集していません
        • Radboud Universitair Medisch Centrum
  • スペイン
    • Barcelona
      • Barcelona、Barcelona、スペイン、8035
        • まだ募集していません
        • Hospital Universitario Vall d'Hebron - PPDS
    • Guipúzcoa
      • San Sebastián、Guipúzcoa、スペイン、20014
        • まだ募集していません
        • Hospital Universitario de Donostia
  • ドイツ
    • Baden-Wurttemberg
      • Ulm、Baden-Wurttemberg、ドイツ、89081
        • まだ募集していません
        • Universitätsklinikum Ulm
    • State of Berlin
      • Berlin、State of Berlin、ドイツ、13125
        • 募集
        • Charité - Campus Berlin Buch
  • フランス
      • Paris、フランス、73013
        • まだ募集していません
        • Institut de Myologie - Hôpital de La Pitié-Salpétrière
    • Bouches-du-Rhône
      • Marseille、Bouches-du-Rhône、フランス、13385
        • まだ募集していません
        • AP-HM- Hôpital de La Timone

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

  • 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:

  • Can understand the written informed consent, provides signed and witnessed written informed consent, and agrees to comply with all protocol requirements
  • Confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation
  • Clinical evidence of weakness affecting any proximal muscle group(s) as assessed by the Investigator
  • Can walk 10 m with or without a cane (no other walking aid allowed)
  • Is either a female of non-childbearing potential or male or female, who agrees to use highly effective contraception/preventive exposure measures from the time of first dose of IP (for a male participant) or the signing of the informed consent form (ICF) (for a female participant) during the trial, and until 7 days after the last dose of IP.

Exclusion Criteria:

  • Unable or unwilling to understand and comply with protocol requirements or unlikely to complete the study as planned, as judged by the Investigator
  • Any clinically significant medical condition that, in the opinion of the Investigator, would interfere with the study
  • Females who are pregnant, breastfeeding or intend to become pregnant, or of childbearing potential not using adequate contraceptive methods
  • Participants with severe pulmonary dysfunction at screening, or evidence of pulmonary exacerbation (defined as an acute worsening of respiratory symptoms that result from a decline in lung function)
  • Cardiac disease by history or at screening that, in the Investigator's opinion, is likely to worsen overall performance of efficacy measures during the study
  • History of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM
  • History of chronic orthopedic issues, acute injury, or expected surgery during the study that may affect the ability to complete study assessments
  • Positive test results at screening for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV)
  • Participants with screening alanine aminotransferase (ALT) levels >3 × upper limit of normal (ULN) or screening aspartate aminotransferase (AST) levels >5 × ULN (isolated elevations of total bilirubin <2 × ULN with direct bilirubin below the ULN will be included)
  • History within the past year of alcohol or other drug substance abuse
  • Known hypersensitivity to the investigational product of related compounds
  • Treatment with statins, proton pump inhibitors or H2 blockers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of the study drug
  • Treatment with sensitive or narrow therapeutic index CYP3A4 substrates within 7 days or 5 half-lives, whichever is longer, prior to first dose of the study drug
  • Treatment with strong or moderate CYP2C8 inhibitor or inducers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of the study drug
  • Currently enrolled in another study or received treatment with any other investigational drug within 30 days or > 5 half-lives, whichever is longer, prior to screening
  • Has reported any suicide ideation of Category 4 or 5 on the C-SSRS within 6 months prior to screening or any suicidal behavior in the last two years prior to screening as indicated by any 'yes' answers on the suicidal behavior section of the C-SSRS

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：クロスオーバー割り当て
  • マスキング：ダブル

武器と介入

参加者グループ / アーム
介入・治療
実験的：グループA
薬：Surlorian
300 mg administered once a day
他の名前：
  • ARM210 (S48168)
プラセボコンパレーター：グループB
他の：Placebo
administered once a day

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
時間枠
Change from baseline in the 1-minute sit-to-stand test (1-MSST)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]

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 the 6-Minute Walk Test (6-MNWT)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the Timed Up and Go Test (TUG)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the 4-Stair Climb Test (4-SCT)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Quantitative Muscle Assessment (QMA)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Manual Muscle Testing (MMT)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-fatigue (PROMIS-F)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-physical fatigue (PROMIS-PF)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Chage in International Physical Activity Questionnaire (IPAQ)
時間枠：Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Number of Adverse Events
時間枠：Day 1 to end of study [approximately 68 weeks]
Day 1 to end of study [approximately 68 weeks]
Change from baseline in systolic blood pressure
時間枠：Day 1 to end of study [approximately 68 weeks]
Systolic blood pressure will be measured in millimeters of mercury (mmHg)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in diastolic blood pressure
時間枠：Day 1 to end of study [approximately 68 weeks]
Diastolic blood pressure will be measured in millimeters of mercury (mmHg)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in temperature
時間枠：Day 1 to end of study [approximately 68 weeks]
Temperature will be measured in degrees Centigrade (°C)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in hemoglobin
時間枠：Day 1 to end of study [approximately 68 weeks]
Hemoglobin concentration will be measured in grams per liter (g/L) using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in white blood cell count
時間枠：Day 1 to end of study [approximately 68 weeks]
White blood cell count will be measured in ×10⁹/L using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in platelet count
時間枠：Day 1 to end of study [approximately 68 weeks]
Platelet count will be measured in ×10⁹/L using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum creatinine
時間枠：Day 1 to end of study [approximately 68 weeks]
Serum creatinine will be measured in micromoles per liter (µmol/L) using standard clinical chemistry methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in estimated glomerular filtration rate
時間枠：Day 1 to end of study [approximately 68 weeks]
Estimated glomerular filtration rate (eGFR) will be calculated from serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration equation
Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum glucose
時間枠：Day 1 to end of study [approximately 68 weeks]
Serum glucose concentration will be measured in millimoles per liter (mmol/L) using standard clinical chemistry methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in QTc interval
時間枠：Day 1 to end of study [approximately 68 weeks]
Corrected QT interval (QTcF) will be measured in milliseconds using triplicate 12-lead electrocardiograms
Day 1 to end of study [approximately 68 weeks]
Change from baseline in urine protein
時間枠：Day 1 to end of study [approximately 68 weeks]
Urine protein will be measured in milligrams per liter (mg/L) using standard urinalysis methods
Day 1 to end of study [approximately 68 weeks]

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

RyCarma Therapeutics, Inc.

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7月1日

一次修了 (推定)

2027年8月27日

研究の完了 (推定)

2028年8月27日

試験登録日

最初に提出

2026年4月7日

QC基準を満たした最初の提出物

2026年4月23日

最初の投稿 (実際)

2026年4月30日

学習記録の更新

投稿された最後の更新 (実際)

2026年7月15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13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

キーワード

その他の研究ID番号

  • CL2-210-02
  • 2025-522343-18-00 (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

いいえ

米国FDA規制機器製品の研究

いいえ

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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