CLINICAL TRIAL

Tideglusib for Myotonic Dystrophy

EnrollingByInvitation · < 18 · All Sexes · Norfolk, VA

This study is evaluating whether a drug called AMO-02 can improve muscle strength and function in children and adolescents with congenital myotonic dystrophy.

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About the trial for Myotonic Dystrophy

Eligible Conditions
Myotonic Dystrophy · Myotonic Dystrophy, Congenital

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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Tideglusib
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tideglusib
Not yet FDA approved

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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The subject's caregiver must be willing and able to support the subject's participation in the study for the duration of the study. show original
The subjects who participated in the study through V11 have completed the study. show original
Before any study related procedures are conducted, the person involved must give written, voluntary, and informed consent show original
Participants in this study must agree to comply with the food intake restrictions as outlined in the study protocol. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 54 weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: 54 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 54 weeks.
View detailed reporting requirements
Trial Expert
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

10-meter walk-run test
52 WEEKS
The 10-meter walk/run test is a performance measure used to assess walking speed in meters per second over a short distance. It can be used as an assessment of functional mobility.
52 WEEKS
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
52 WEEKS
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
52 WEEKS
Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview
52 WEEKS
The Vineland Adaptive Behavior Scales require the clinician to measure personal and social skills needed for everyday living. It provides a targeted assessment of adaptive behavior via a semi-structured parent or caregiver interview.
52 WEEKS
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
52 WEEKS
CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.
52 WEEKS
Autism Behavior Inventory- Clinician (ABI-C)
52 WEEKS
ABI-C is a 14 item rating scale requires the clinician to assess the core features of Autism Spectrum Disorder as well as common associated behaviors.
52 WEEKS
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
54 WEEKS
The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.
54 WEEKS
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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...

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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