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Single Dose of PGN-EDODM1 at Dose Level 3 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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 16
Awards & highlights

FREEDOM-DM1 Trial Summary

This trial is being conducted to see if a new medication, PGN-EDODM1, is safe and well-tolerated when given as a single IV dose to people with Myotonic

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.Check my eligibility
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.See study design
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.

FREEDOM-DM1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My leg muscles are strong enough to move against some resistance.
Select...
I experience muscle stiffness that doesn't relax quickly.
Select...
I have been diagnosed with DM1 due to a specific genetic marker.

FREEDOM-DM1 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 16
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 16 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Secondary outcome measures
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Area Under the Concentration-time Curve of PGN-EDODM1
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
+1 more

FREEDOM-DM1 Trial Design

4Treatment groups
Experimental Treatment
Placebo Group
Group I: Single Dose of PGN-EDODM1 at Dose Level 3Experimental Treatment1 Intervention
Group II: Single Dose of PGN-EDODM1 at Dose Level 2Experimental Treatment1 Intervention
Group III: Single Dose of PGN-EDODM1 at Dose Level 1Experimental Treatment1 Intervention
Group IV: Single Dose of PlaceboPlacebo Group1 Intervention

Find a Location

Who is running the clinical trial?

PepGen IncLead Sponsor
1 Previous Clinical Trials
10 Total Patients Enrolled

Frequently Asked Questions

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

Which individuals meet the criteria to be eligible for enrollment in this research investigation?

"Patients who are between the ages of 18 and 50 and have been diagnosed with myotonic dystrophy may be eligible to participate in this research project. Approximately 24 individuals will be enrolled for this study."

Answered by AI

How large is the participant pool for this particular clinical study?

"Indeed, the data provided by clinicaltrials.gov indicates that this particular clinical trial is currently in the recruitment phase. It was initially posted on December 12th, 2023 and last updated on January 3rd, 2024. The study aims to enroll a total of 24 participants from a single location."

Answered by AI

Is this clinical trial currently open for enrollment?

"Indeed, the information available on clinicaltrials.gov indicates that this particular clinical trial is presently accepting applications from potential participants. The trial was initially posted on December 12th, 2023 and has undergone its most recent update on January 3rd, 2024. For this study, a total of 24 individuals are being sought after at one designated research site."

Answered by AI

Are individuals who are above the age of 80 eligible to participate in this medical study?

"To be eligible for enrollment in this clinical trial, potential participants must be at least 18 years old but not exceed the age of 50."

Answered by AI

What are the potential risks associated with administering a single dose of PGN-EDODM1 at Dose Level 3 to patients?

"Considering the limited safety and efficacy data available for a Phase 1 trial, our team at Power rates the safety of a Single Dose of PGN-EDODM1 at Dose Level 3 as 1 on a scale from 1 to 3."

Answered by AI

Who else is applying?

What site did they apply to?
Rare Disease Research, LLC
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
2023. "Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06204809.
All > Paid Studies Atlanta
~16 spots leftby Apr 2025
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