What is the mechanism of action of this treatment?

The most common treatments for Myotonic Dystrophy Type 1 (DM1) often focus on modulating gene expression or protein function to address the underlying genetic defects. These treatments aim to correct the abnormal RNA splicing caused by the expanded CTG repeats in the DMPK gene, which leads to the production of toxic RNA. By targeting this toxic RNA, therapies can reduce its accumulation and mitigate its harmful effects on muscle and other tissues. This approach is crucial for DM1 patients as it directly addresses the root cause of the disease, potentially improving muscle function, reducing symptoms, and enhancing overall quality of life.

What side effects are associated with this type of intervention?

Common treatments for Myotonic Dystrophy Type 1 (DM1) that modulate gene expression or protein function include antisense oligonucleotides, small molecule inhibitors, and gene therapy approaches. Known side effects of these treatments can include injection site reactions, flu-like symptoms, and potential off-target effects leading to unintended gene modulation. Additionally, there may be risks of immune responses to the therapeutic agents. For example, in trials involving antisense oligonucleotides, patients have reported mild to moderate adverse effects such as headache, fatigue, and gastrointestinal disturbances.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Myotonic Dystrophy Type 1 (DM1) that modulate gene expression or protein function. Current management primarily focuses on symptomatic treatment, such as medications for muscle stiffness, pain, and cardiac issues. The trial for PGN-EDODM1 represents an investigational approach aimed at addressing the underlying genetic causes of DM1, which is a novel direction in the treatment landscape for this condition.

What other types of research exist?

Promising alternatives to PGN-EDODM1 for Myotonic Dystrophy Type 1 patients include: 1) GDF11/myostatin inhibitors like GDF11 propeptide-Fc, which increase muscle mass and strength. 2) NF-κB inhibitors such as edasalonexent and CAT-1041, which have shown efficacy in ameliorating dystrophic processes. 3) PGC-1α overexpression, which protects against muscle damage and improves muscle dystrophy. These therapies have shown potential in preclinical models and could be considered for further investigation in DM1 treatment.

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PGN-EDODM1 for Myotonic Dystrophy (FREEDOM-DM1 Trial)

Phase 1

Recruiting

Research Sponsored by PepGen Inc

Eligibility Criteria Checklist

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

Must have

Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles

Presence of myotonia

Must not have

Congenital DM1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing the safety and tolerability of a drug called PGN-EDODM1, given through an IV, in people with Myotonic Dystrophy Type 1 (DM1). The drug aims to address the underlying issues causing muscle problems in these patients. The study includes initial assessments followed by a period of receiving the drug and monitoring.

Who is the study for?

This trial is for individuals with Myotonic Dystrophy Type 1. Participants must be adults who meet specific health criteria during the screening period, which lasts up to 30 days before starting treatment.

What is being tested?

The study tests the safety and tolerability of a new drug called PGN-EDODM1 given through an IV compared to a placebo in people with Myotonic Dystrophy Type 1 over a period of 16 weeks.

What are the potential side effects?

Since this is a study primarily focused on safety and tolerability, potential side effects are being investigated but may include reactions at the infusion site, fatigue, muscle symptoms or other issues related to DM1.

Eligibility Criteria

Inclusion Criteria

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

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My leg muscles are strong enough to move against some resistance.

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I experience muscle stiffness that doesn't relax quickly.

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I have been diagnosed with DM1 due to a specific genetic marker.

Exclusion Criteria

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

Select...

I was born with myotonic dystrophy type 1.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PGN-EDODM1Experimental Treatment1 Intervention

PGN-EDODM1 for infusion

Group II: PlaceboPlacebo Group1 Intervention

0.9% NaCl

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

The most common treatments for Myotonic Dystrophy Type 1 (DM1) often focus on modulating gene expression or protein function to address the underlying genetic defects. These treatments aim to correct the abnormal RNA splicing caused by the expanded CTG repeats in the DMPK gene, which leads to the production of toxic RNA. By targeting this toxic RNA, therapies can reduce its accumulation and mitigate its harmful effects on muscle and other tissues. This approach is crucial for DM1 patients as it directly addresses the root cause of the disease, potentially improving muscle function, reducing symptoms, and enhancing overall quality of life.