Not yet recruitingPHASE2INTERVENTIONAL

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

This study is looking into a new medicine called Surlorian for adults who have a specific genetic muscle condition known as autosomal dominant RYR1-related myopathy (RYR1-RM). The main goal is to find out if Surlorian can make muscles stronger and if it's safe and doesn't cause too many side effects. Participants will receive both Surlorian and a 'dummy' medicine (placebo) at different times, but no one will know which one they're getting at any particular moment. After this main part, there might be an option to continue receiving only Surlorian for a longer period. This trial is in an early stage (Phase 2), meaning it's still primarily focused on safety and initial effectiveness.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

RyCarma Therapeutics, Inc.

Enrolment target

Start

15 Apr 2026

Estimated completion

27 Aug 2028

Autosomal Dominant RYR1-Related Myopathy

What is this study about?

Imagine your muscles aren't working as well as they should, and you have a condition called autosomal dominant RYR1-related myopathy. This study is trying to find a new medicine, called Surlorian, that might help. Doctors want to see if Surlorian can make your muscles stronger and if it's a safe medicine to use. It's like a scientific detective mission to understand if this new treatment can make a real difference for people with this condition.

During the study, you wouldn't know if you're getting the actual Surlorian medicine or a 'dummy' treatment called a placebo. This is a common and important way to fairly test new medicines, ensuring that any improvements are truly due to the medicine and not just other factors. Everyone eventually gets both the real medicine and the 'dummy' one, just at different times.

After the main part of the study, which lasts about 16 weeks, there might be an option to continue receiving only Surlorian for about a year. This longer period helps doctors see how the medicine works over time. The study is taking place in special medical centers with doctors who know a lot about RYR1-RM, making sure you're in good hands.

Key takeaways

This study is testing a new medicine called Surlorian for a genetic muscle condition.
It aims to see if Surlorian improves muscle strength and is safe.
Participants will receive both the real medicine and a 'dummy' medicine at different times.
The main study lasts around 16 weeks, with an optional longer extension.
Strict rules apply for who can and cannot join the study.
You can stop participating at any time if you wish.

Who may be eligible?

To join this study, you must be between 18 and 65 years old and have a confirmed diagnosis of RYR1-related myopathy due to a specific gene change (autosomal dominant mutation). You should also show some muscle weakness and be able to walk at least 10 metres, even if you need a cane. It's important to be able to understand the study information and agree to follow all the rules.

There are also some things that would mean you couldn't join. For instance, if you have other serious health problems that might affect the study, or if you're pregnant, breastfeeding, or planning to get pregnant. You also can't have certain other conditions like severe breathing or heart problems, a history of seizures not related to your muscle condition, or some specific infections like HIV or Hepatitis.

Both men and women who could have children must agree to use reliable birth control during the study and for a short time after. This is to make sure the medicine doesn't affect a potential pregnancy.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 65 years old?
Do you have a confirmed genetic diagnosis of RYR1-related myopathy?
Can you walk at least 10 metres, possibly with a cane?
Are you able to understand the study details and follow instructions?
Are you not pregnant, breastfeeding, or planning to become pregnant?
Do you not have other serious health problems that might complicate the study?

Answer every question to see your result.

What does participation involve?

If you join, the main part of the study lasts about 16 weeks. During this time, you'll be given either Surlorian or a dummy treatment (placebo). There will be a 'washout' period in between, which acts like a break before you swap over to the other treatment. This means you will try both the study medicine and the placebo at different times.

Neither you nor the study team will know which treatment you are on at any given time. This helps to keep the study fair and accurate. After the 16 weeks, you might be able to join an 'open-label' extension, where everyone receives Surlorian for approximately 12 months. This means you will know you are getting the active medicine.

Throughout the study, you'll have regular visits to the medical centre for check-ups. These visits will include tests to see how your muscles are doing, and to check your general health and safety. These might include blood tests, physical examinations, and other assessments to track how you're responding to the treatment.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit if Surlorian proves to be an effective medicine for your condition, as you would be among the first to receive it. However, because this is a new medicine, there are also potential risks, including unknown side effects. You might also receive the placebo and not the active drug for part of the study. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (9)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

AP-HM- Hôpital de La Timone
Verified postcode
Marseille, France
Institut de Myologie - Hôpital de La Pitié-Salpétrière
Verified postcode
Paris, France
Universitätsklinikum Ulm
Verified postcode
Ulm, Germany
Charité - Campus Berlin Buch
Verified postcode
Berlin, Germany
Radboud Universitair Medisch Centrum
Verified postcode
Nijmegen, Netherlands
Hospital Universitario Vall d'Hebron - PPDS
Verified postcode
Barcelona, Spain
Hospital Universitario de Donostia
Verified postcode
San Sebastián, Spain
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Verified postcode
Oswestry, United Kingdom
University College Hospital - PPDS
Verified postcode
London, United Kingdom

Common questions

What is RYR1-related myopathy?

It's a rare muscle condition caused by a change in a specific gene (RYR1), leading to muscle weakness.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active drug. It helps researchers fairly test a new medicine.

Will I know if I'm getting the real medicine or the placebo?

During the main part of the study, neither you nor the doctors will know. This is to ensure fair testing. In the optional longer part, everyone gets the real medicine.

How long will I be in the study?

The main part lasts about 16 weeks. There's an option for a further 12 months in a different part of the study where everyone gets the active medicine.

Can I leave the study at any time?

Yes, you can choose to leave the study at any point without it affecting your usual medical care.

How to find out more

Patrick Round

clinicaltrials@rycarma.com 888-209-5458 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.