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
2026년 4월 23일 업데이트: 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.
연구 개요
상세 설명
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.
연구 유형
중재적
등록 (추정된)
28
단계
- 2 단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 연락처
- 이름: Patrick Round
- 전화번호: 888-209-5458
- 이메일: clinicaltrials@rycarma.com
연구 장소
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Gelderland
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Nijmegen, Gelderland, 네덜란드, 6500 HB
- Radboud Universitair Medisch Centrum
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Baden-Wurttemberg
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Ulm, Baden-Wurttemberg, 독일, 89081
- Universitätsklinikum Ulm
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State of Berlin
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Berlin, State of Berlin, 독일, 13125
- Charité - Campus Berlin Buch
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Barcelona
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Barcelona, Barcelona, 스페인, 8035
- Hospital Universitario Vall d'Hebron - PPDS
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Guipúzcoa
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San Sebastián, Guipúzcoa, 스페인, 20014
- Hospital Universitario de Donostia
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London, 영국, WC1N 3BG
- University College Hospital - PPDS
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Shropshire
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Oswestry, Shropshire, 영국, SY10 7AG
- The Robert Jones and Agnes Hunt Orthopaedic Hospital
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Paris, 프랑스, 73013
- Institut de Myologie - Hôpital de La Pitié-Salpétrière
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Bouches-du-Rhône
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Marseille, Bouches-du-Rhône, 프랑스, 13385
- AP-HM- Hôpital de La Timone
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
아니
설명
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
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 크로스오버 할당
- 마스킹: 더블
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
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실험적: 그룹 A
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300 mg administered once a day
다른 이름들:
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위약 비교기: 그룹 B
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administered once a day
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
|---|---|
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Change from baseline in the 1-minute sit-to-stand test (1-MSST)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change from baseline in the 6-Minute Walk Test (6-MNWT)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline in the Timed Up and Go Test (TUG)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline in the 4-Stair Climb Test (4-SCT)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline Quantitative Muscle Assessment (QMA)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline Manual Muscle Testing (MMT)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline in Patient-Reported Outcomes Measurement Information System-fatigue (PROMIS-F)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Change from baseline in Patient-Reported Outcomes Measurement Information System-physical fatigue (PROMIS-PF)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Chage in International Physical Activity Questionnaire (IPAQ)
기간: Day 1 to day 28 [approximately]
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Day 1 to day 28 [approximately]
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Number of Adverse Events
기간: Day 1 to end of study [approximately 68 weeks]
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in systolic blood pressure
기간: Day 1 to end of study [approximately 68 weeks]
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Systolic blood pressure will be measured in millimeters of mercury (mmHg)
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in diastolic blood pressure
기간: Day 1 to end of study [approximately 68 weeks]
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Diastolic blood pressure will be measured in millimeters of mercury (mmHg)
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in temperature
기간: Day 1 to end of study [approximately 68 weeks]
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Temperature will be measured in degrees Centigrade (°C)
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in hemoglobin
기간: Day 1 to end of study [approximately 68 weeks]
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Hemoglobin concentration will be measured in grams per liter (g/L) using standard hematology laboratory methods
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in white blood cell count
기간: Day 1 to end of study [approximately 68 weeks]
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White blood cell count will be measured in ×10⁹/L using standard hematology laboratory methods
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in platelet count
기간: Day 1 to end of study [approximately 68 weeks]
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Platelet count will be measured in ×10⁹/L using standard hematology laboratory methods
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in serum creatinine
기간: Day 1 to end of study [approximately 68 weeks]
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Serum creatinine will be measured in micromoles per liter (µmol/L) using standard clinical chemistry methods
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in estimated glomerular filtration rate
기간: Day 1 to end of study [approximately 68 weeks]
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Estimated glomerular filtration rate (eGFR) will be calculated from serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration equation
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in serum glucose
기간: Day 1 to end of study [approximately 68 weeks]
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Serum glucose concentration will be measured in millimoles per liter (mmol/L) using standard clinical chemistry methods
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in QTc interval
기간: Day 1 to end of study [approximately 68 weeks]
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Corrected QT interval (QTcF) will be measured in milliseconds using triplicate 12-lead electrocardiograms
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Day 1 to end of study [approximately 68 weeks]
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Change from baseline in urine protein
기간: Day 1 to end of study [approximately 68 weeks]
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Urine protein will be measured in milligrams per liter (mg/L) using standard urinalysis methods
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Day 1 to end of study [approximately 68 weeks]
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (추정된)
2026년 4월 15일
기본 완료 (추정된)
2027년 8월 27일
연구 완료 (추정된)
2028년 8월 27일
연구 등록 날짜
최초 제출
2026년 4월 7일
QC 기준을 충족하는 최초 제출
2026년 4월 23일
처음 게시됨 (실제)
2026년 4월 30일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 4월 30일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 4월 23일
마지막으로 확인됨
2026년 4월 1일
추가 정보
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