Tideglusib for Myotonic Dystrophy
This study is evaluating whether a drug called AMO-02 can improve muscle strength and function in children and adolescents with congenital myotonic dystrophy.
See full descriptionAbout the trial for Myotonic Dystrophy
Treatment Groups
This trial involves 2 different treatments. Tideglusib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 & 3 and have had some early promising results.
About The Treatment
Eligibility
This trial is for patients born any sex aged 18 and younger. There are 4 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Tideglusib will improve 1 primary outcome and 8 secondary outcomes in patients with Myotonic Dystrophy. Measurement will happen over the course of 52 weeks.
Patient Q & A Section
How many people get myotonic dystrophy a year in the United States?
Around 100,000 are diagnosed with DM1 each year. The average age is 45 years at time of diagnosis. The disease has a carrier rate of 60%. Myotonic dystrophy affects males more than females.
Have there been other clinical trials involving tideglusib?
Our preliminary results show that tideglusib in patients with myotonic dystrophy type 1 is well-tolerated and has the potential for future clinical development; however, additional clinical trial data are urgently required prior to widespread recommendation in myotonic dystrophy. This trial was registered at clinicaltrials.gov as NCT03611351.
What are the signs of myotonic dystrophy?
Symptoms related to myotonic dystrophy are found early, even before the diagnosis of the disease and they tend to evolve throughout the course of the illness. Symptoms associated with ocular abnormalities tend to occur later in the disease course, especially in the more severe forms.
Can myotonic dystrophy be cured?
The study supports the notion that in some cases, myotonic dystrophy can be treated. The degree of improvement varies drastically from patient to patient. It is best not to try using conventional treatments as a sole tactic, but to use these strategies in combination.
What causes myotonic dystrophy?
We have found no evidence for a genetic association for DM, and instead have found associations between the risk of DM and environmental factors such as smoking, and some of the genetic associations between DM. It remains to be proven whether these environmental risk factors increase the risk of DM, or whether DM itself causes the risk factors. We speculate we may find evidence that DM may be influenced by a small set of common environmental risk factors, which we are not currently assessing.
What are common treatments for myotonic dystrophy?
There is a dearth of peer-reviewed information on the management of DM2. Patients need education in their disease management, but they also need information on their own risks and benefits. Physicians need to learn to assess the risks before initiating treatments.
What is myotonic dystrophy?
Myotonic dystrophy is a rare muscular disorder with an approximate incidence rate of approximately 1 in 15000. It occurs later in life, after a mean age of 42 for the adult onset form. This is a disease that has a variety of systemic symptoms and is characterised initially by atrophy of muscles. We have demonstrated a link between the MYCN locus and myotonic dystrophy by performing a LOH analysis for this gene on the patient material. In addition we have confirmed that MYCN is a modifier in other myotonic dystrophies. In a recent study, findings will further the progress in understanding myotonic dystrophy.
How serious can myotonic dystrophy be?
Results from a recent paper, patients diagnosed with MD-1D had fewer total births and a significantly lower live birth rate than sporadic-MD patients. The most prominent feature of DMD is early-onset severe muscle weakness, which leads to early dependence/physical disability and often leads to death. Although both groups reported fatigue and muscle pain, the severity and degree of symptoms in patients diagnosed with DMD appear to be more severe and disabling than in age- and gender-matched patients with sporadic DMD. Results from a recent paper, in conjunction with previously published clinical data on the outcomes of patients with DMD, suggests that this form of MD is a progressive disorder with significant life-threatening symptoms.
What is tideglusib?
Tideglusib is an oral tyrosine kinase inhibitor effective against a small subset of BCL-2 related malignancies. Tideglusib has shown activity in advanced solid tumors and hematologic malignancies harboring aberrant BCL-2 translocation...
Is tideglusib safe for people?
Tideglusib is well tolerated, and has demonstrated a high safety profile to date. Given their safety and excellent efficacy profile, further studies with longer follow-up are indicated to establish the safety of tideglusib in the longer term.
What are the latest developments in tideglusib for therapeutic use?
This article highlights the most recent updates and research underway on the recently approved drug, tiroteglutide, to address the most important clinical questions, particularly those on the efficacy of the drug in DMD. Key points include current use and the evidence for dose responses, tolerability, and other end points.
What are the common side effects of tideglusib?
In this exploratory study, the most common adverse events associated with the investigational drug tideglusib included fatigue, headache, fatigue, dizziness, abdominal pain, vomiting, constipation, and cough. Other common adverse effects included abnormal taste sensations and decreased sensation in the hands, feet, lips, and tongue. These adverse events were generally mild to moderate in severity and experienced by all patients randomized to treatment. On the basis of these observations, tideselusib can be considered to be generally well tolerated by patients.