Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This research study is investigating a new medicine called Tideglusib for individuals aged 6 to 45 who have a muscle condition known as Congenital or Childhood Onset Myotonic Dystrophy (DM1). This condition is present from birth or develops in childhood. The main goals of the study are to understand how safe the medicine is and how well it works. Some participants will have taken part in a previous study with this medicine, while others will be trying it for the first time. Participants will receive the medicine for about a year, with the possibility of taking it for longer. This study is an important step in finding better ways to help people living with DM1.
At a glance
What is this study about?
This study is all about a medicine called Tideglusib and how it might help children and young people who have a muscle condition called Myotonic Dystrophy type 1 (DM1). Specifically, it's for those who were born with the condition (Congenital DM1) or developed it in childhood (Childhood Onset DM1). Myotonic Dystrophy causes muscles to become weak and tight, and it can affect different parts of the body.
The researchers want to learn two main things: first, how safe Tideglusib is for people with DM1, and second, how well it works to reduce the symptoms of the condition. Finding new and effective treatments is really important for improving the lives of individuals with DM1. This study is an 'open-label' study, which means everyone involved, including the participants and the doctors, will know that Tideglusib is being given.
Participants will take Tideglusib for about a year, and there might be an option to continue taking it for longer after that. The amount of medicine given will depend on the person's weight. Some people in this study will have already been part of a previous study testing Tideglusib, while others will be trying it for the first time. This blend of participants helps researchers gather a lot of information about the medicine.
Key takeaways
- This study is testing a new medicine called Tideglusib for Congenital or Childhood Myotonic Dystrophy (DM1).
- It's for people aged 6 to 45 with a confirmed DM1 diagnosis.
- Participants will take the medicine daily for about a year, with regular check-ups.
- The study aims to understand the medicine's safety and how well it works.
- You'll know you are receiving the active medicine (Tideglusib).
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, participants generally need to be diagnosed with Congenital or Childhood Onset Myotonic Dystrophy (DM1), and this diagnosis must be confirmed by a genetic test. They need to be between 6 and 45 years old when the study starts. Also, a doctor needs to assess their DM1 symptoms and confirm they are at a certain level of severity.
If you haven't been in the previous study (AMO-02-MD-2-003) but are interested, you'll need to meet all these criteria. For all participants, a parent or legal guardian must agree to you joining the study, and if you're old enough, you'll also need to agree. Your caregiver must be able to support you throughout the study, and you must be willing to follow any special food instructions.
There are also a few reasons why someone might not be able to join. For example, if your body mass index (a measure of weight compared to height) is too low (under 13.5) or too high (over 40). Also, if you've recently started or changed certain medications or therapies, you might not be able to take part.
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.
- Do I have a confirmed genetic diagnosis of Congenital or Childhood Myotonic Dystrophy (DM1)?
- Am I between 6 and 45 years old?
- Am I able to commit to regular clinic visits and follow study instructions?
- Is my caregiver willing and able to support my participation throughout the study?
- Do I have a body mass index (BMI) within the healthy range (not too low or too high)?
- Have I recently started or changed other treatments, which might prevent me from joining?
What does participation involve?
If you decide to take part in this study, you will be taking a medicine called Tideglusib daily for about 52 weeks (roughly one year). The amount of medicine you receive will be carefully measured based on your weight. After this main treatment period, there's a chance you could continue taking the medicine for an extended time if the study allows.
Throughout the study, you'll have regular visits to the clinic so the doctors can check on your health and how the medicine is affecting you. These visits might include physical examinations, blood tests, and assessments of your muscle strength and daily activities. Your caregiver will need to help you attend these appointments and follow the study instructions, including any special rules about what you can eat.
Before you start, you'll have a screening visit to make sure you're a good fit for the study. The total time you're involved could be over a year, depending on whether you join the extended treatment period.
Potential risks and benefits
Locations (14)
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- University of California, Los Angeles (UCLA)Verified postcodeLos Angeles, United States· Enrolling by invitation
- Stanford UniversityVerified postcodePalo Alto, United States· Enrolling by invitation
- Lurie's Children's HospitalVerified postcodeChicago, United States· Recruiting
- University of Iowa Hospitals and ClinicsVerified postcodeIowa City, United States· Recruiting
- University of Rochester - Medical CenterVerified postcodeRochester, United States· Recruiting
- University of Pittsburgh Medical CenterVerified postcodePittsburgh, United States· Recruiting
- University of Utah Clinical Neurosciences CenterVerified postcodeSalt Lake City, United States· Recruiting
- Children's Hospital of The King's DaughtersVerified postcodeNorfolk, United States· Withdrawn
- Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research ProgramVerified postcodeRichmond, United States· Completed
- The Bright AllianceVerified postcodeRandwick, Australia· Recruiting
- Children's Hospital London Health Sciences Centre (LHSC)Verified postcodeLondon, Canada· Enrolling by invitation
Common questions
What is Myotonic Dystrophy (DM1)?
Myotonic Dystrophy type 1 (DM1) is a genetic condition that mainly causes muscle weakness and difficulty relaxing muscles. It can develop from birth (Congenital) or childhood.
What is Tideglusib?
Tideglusib is a new medicine being studied to see if it can help people with Myotonic Dystrophy. It's not yet approved for general use.
Will I know if I'm getting the actual medicine?
Yes, this is an 'open-label' study, which means everyone involved will know that participants are receiving Tideglusib.
How long will I be taking the medicine?
You will take the medicine for about 52 weeks (one year), with a possibility of continuing for longer if you wish and if the study allows.
What if I change my mind after joining?
You can leave the study at any time, for any reason, without it affecting your usual medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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