What side effects are associated with this type of intervention?

Common treatments for Myotonic Dystrophy include medications like mexiletine, which can cause gastrointestinal issues and dizziness, and physical therapy to manage symptoms. For treatments similar to Tideglusib, a GSK-3β inhibitor, side effects may include gastrointestinal disturbances, fatigue, and potential liver enzyme elevations. It is important to monitor these side effects closely and discuss them with a healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of Myotonic Dystrophy in the provided context. Tideglusib, a GSK-3β inhibitor, is currently being studied in a phase 2/3 trial for Congenital Myotonic Dystrophy. Generally, treatment for Myotonic Dystrophy focuses on managing symptoms and may include medications for muscle stiffness, pain, and other related symptoms, but no disease-modifying treatments have been highlighted in the context provided.

What other types of research exist?

Promising novel alternatives to AMO-02 (Tideglusib) for Myotonic Dystrophy patients include antisense oligonucleotide therapies, such as those targeting DMPK mRNA, and small molecule modulators of RNA splicing. These therapies are in advanced stages of clinical development and have shown potential in improving muscle function and reducing disease symptoms.

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Glycogen Synthase Kinase-3 Beta Inhibitor

Tideglusib for Myotonic Dystrophy (REACH CDM X Trial)

Phase 2 & 3

Recruiting

Research Sponsored by AMO Pharma Limited

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1

Subjects must be male or female aged ≥6 years to ≤45 years at Screening

Must not have

Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to week 132

Awards & highlights

No Placebo-Only Group

Summary

This trial tests tideglusib, a medication, in children and adolescents with Congenital or Childhood Onset Myotonic Dystrophy. It aims to improve muscle function by targeting specific body processes. The study includes both those who participated in a previous study and those who have not been treated before.

Who is the study for?

This trial is for individuals aged 6 to 45 with Congenital or Childhood Onset Myotonic Dystrophy, who can follow food intake rules and have a caregiver's support. It's open to those who completed the AMO-02-MD-2-003 study or are treatment naïve. Participants need confirmed genetic diagnosis and a CGI-S score of ≥3.

What is being tested?

The trial tests Tideglusib's safety and effectiveness in treating Myotonic Dystrophy. It includes people from an earlier study on the same drug as well as new participants, all receiving Tideglusib in an open-label phase 2/3 setting.

What are the potential side effects?

While specific side effects of Tideglusib aren't listed here, common ones may include allergic reactions (as there's mention of hypersensitivity), especially since it contains strawberry components.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I was diagnosed with DM1 as a child or it is a congenital condition.

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I am between 6 and 45 years old.

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My diagnosis was confirmed through genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My BMI is either below 13.5 or above 40.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to week 132

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to week 132

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to week 132 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

Safety (Adverse Events)

Safety (Adverse Events) - With Optional Expanded Access

Secondary study objectives

10-meter walk-run test

Autism Behavior Inventory- Clinician (ABI-C)

Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)

+11 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: TideglusibExperimental Treatment1 Intervention

Weight adjusted tideglusib, orally, once daily

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tideglusib

2016

Completed Phase 3

~160

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Tideglusib, a GSK-3β inhibitor, works by inhibiting glycogen synthase kinase 3 beta, an enzyme involved in various cellular processes including inflammation and cell death. This inhibition can help reduce muscle degeneration and improve muscle function in Myotonic Dystrophy patients. Other common treatments include antisense oligonucleotides, which target and modify the expression of specific genes involved in the disease, and small molecule drugs that aim to correct the underlying genetic defects or improve muscle function. These treatments are crucial for Myotonic Dystrophy patients as they address the root causes of muscle weakness and degeneration, potentially improving quality of life and disease outcomes.