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New treatment option for Myotonic Dystrophy 1
Official title A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
ClinicalTrials.gov ID: NCT06667453
What this study is testing
What is PGN-EDODM1?
PGN-EDODM1 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for myotonic dystrophy 1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 65
You may be able to join if
- Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- Presence of myotonia
- Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
- Body Mass Index (BMI) of \< 35.0 kg/m\^2
You likely can't join if
- Congenital DM1
- 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
- Medications specific for the treatment of myotonia within 2 weeks prior to screening
- Percent predicted forced vital capacity (FVC) \<40%
- Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening Note...
The study team makes the final eligibility decision.
Where it's taking place
- Calgary, Alberta, Canada
- Ottawa, Ontario, Canada
- Chicoutimi, Quebec, Canada
- Montreal, Quebec, Canada
- Takapuna, Auckland, New Zealand
- Salford, England, United Kingdom
- London, UK, United Kingdom
- Newcastle upon Tyne, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Calgary, Alberta, Canada; Ottawa, Ontario, Canada; Chicoutimi, Quebec, Canada; Montreal, Quebec, Canada; Takapuna, Auckland, New Zealand; Salford, England, United Kingdom and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.