BridgeMD
Compares treatment options for Autosomal Dominant RYR1-Related Myopathy
Official title 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
ClinicalTrials.gov ID: NCT07560020
What this study is testing
What is Surlorian?
Surlorian is an investigational medicine, given as an once-daily, being studied as a potential treatment for autosomal dominant ryr1-related myopathy.
Also referred to as ARM210 (S48168).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- Can understand the written informed consent, provides signed and witnessed written informed consent, and agrees to comply with all protocol...
- 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...
You likely can't join if
- 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...
- Cardiac disease by history or at screening that, in the Investigator's opinion, is likely to worsen overall performance of how well it works measures...
- History of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM
See the full eligibility 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...
- 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 how well it works 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Marseille, Bouches-du-Rhône, France
- Paris, France
- Ulm, Baden-Wurttemberg, Germany
- Berlin, State of Berlin, Germany
- Nijmegen, Gelderland, Netherlands
- Barcelona, Barcelona, Spain
- San Sebastián, Guipúzcoa, Spain
- Oswestry, Shropshire, United Kingdom
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Marseille, Bouches-du-Rhône, France; Paris, France; Ulm, Baden-Wurttemberg, Germany; Berlin, State of Berlin, Germany; Nijmegen, Gelderland, Netherlands; Barcelona, Barcelona, Spain and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.