24 Participants Needed

PGN-EDODM1 for Muscular Dystrophy

(FREEDOM2-DM1 Trial)

Recruiting at 3 trial locations
P
Overseen ByPepGen
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking medications specifically for the treatment of myotonia at least 2 weeks before screening. For other medications, the protocol does not specify, so it's best to discuss with the study team.

What data supports the effectiveness of the drug PGN-EDODM1 for Muscular Dystrophy?

Research on similar treatments, like eteplirsen, shows that increasing dystrophin production can help slow down muscle decline in Duchenne muscular dystrophy. Additionally, studies on utrophin modulation suggest that enhancing related proteins can improve muscle function and stability, which might be relevant for PGN-EDODM1.12345

How is the drug PGN-EDODM1 different from other treatments for muscular dystrophy?

PGN-EDODM1 is unique because it targets the prostaglandin D2 (PGD2) pathway, which is involved in muscle necrosis in Duchenne muscular dystrophy (DMD). This approach is different from other treatments that focus on gene therapy or exon skipping, as it aims to reduce inflammation and muscle damage by inhibiting the enzyme responsible for PGD2 production.678910

What is the purpose of this trial?

The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.

Eligibility Criteria

This trial is for people with myotonic dystrophy type 1, who have myotonia and a confirmed genetic diagnosis. Participants should be able to move their tibialis anterior muscles against moderate pressure and have a BMI of less than 32 kg/m2.

Inclusion Criteria

I can move my lower legs fully and resist some pressure.
Body Mass Index (BMI) of < 32.0 kg/m^2
I have been diagnosed with DM1 with a specific genetic marker.
See 1 more

Exclusion Criteria

I was born with myotonic dystrophy type 1.
Known history or presence of any clinically significant conditions that may interfere with study safety assessments
Abnormal laboratory tests at screening considered clinically significant by the Investigator
See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either PGN-EDODM1 or placebo once every 4 weeks for 12 weeks

12 weeks
3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • PGN-EDODM1
Trial Overview The study tests PGN-EDODM1, an experimental drug for DM1. It will compare the safety and tolerability of multiple doses of PGN-EDODM1 against a placebo in participants.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: PGN-EDODM1Experimental Treatment1 Intervention
Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks
Group II: PlaceboPlacebo Group1 Intervention
Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

Find a Clinic Near You

Who Is Running the Clinical Trial?

PepGen Inc

Lead Sponsor

Trials
4
Recruited
80+

Findings from Research

In a long-term study of patients with Duchenne muscular dystrophy (DMD) treated with eteplirsen, 100% of patients showed successful skipping of exon 51, confirming the drug's mechanism of action.
Eteplirsen treatment resulted in significant increases in dystrophin production, with an 11.6-fold increase in dystrophin content and a 7.4-fold increase in the percentage of dystrophin-positive fibers compared to untreated controls, indicating its potential efficacy in restoring dystrophin levels in muscle cells.
Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production.Charleston, JS., Schnell, FJ., Dworzak, J., et al.[2019]
In a phase 3 study involving 79 patients aged 7-16 with Duchenne muscular dystrophy, eteplirsen treatment for 96 weeks resulted in a significant increase in dystrophin production (7-fold) and exon skipping (18.7-fold), indicating its efficacy in addressing the underlying cause of the disease.
The study also demonstrated a favorable safety profile, with most adverse events being mild to moderate and unrelated to the treatment, while showing a notable slowing of disease progression compared to untreated controls.
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.McDonald, CM., Shieh, PB., Abdel-Hamid, HZ., et al.[2022]
In a 48-week study involving 57 DMD patients, the 6-minute walk distance (6MWD) was confirmed as a strong predictor of ambulation decline, with a baseline distance of less than 350 meters indicating a higher risk of functional decline.
The study found that only 2.3% of subjects who could stand from a supine position lost their ability to walk, highlighting the importance of baseline functional assessments in predicting outcomes in DMD patients.
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.McDonald, CM., Henricson, EK., Abresch, RT., et al.[2022]

References

Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. [2019]
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial. [2022]
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. [2022]
Second-generation compound for the modulation of utrophin in the therapy of DMD. [2022]
Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression. [2022]
Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. [2021]
A prostaglandin D2 metabolite is elevated in the urine of Duchenne muscular dystrophy patients and increases further from 8 years old. [2022]
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. [2021]
[Hematopoietic prostaglandin D synthase inhibitors for the treatment of duchenne muscular dystrophy]. [2022]
Highly sensitive screening of antisense sequences for different types of DMD mutations in patients' urine-derived cells. [2021]
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