The FREEDOM2-DM1 is a Phase 2 study investigating whether multiple doses of a potential new medication, called PGN-EDODM1, is safe and tolerable for people living with DM1 compared to a placebo (a substance that has no active drug in it)
Study participants may be able to join the study if they meet the following requirements:
16-60 years old (inclusive)
Confirmed diagnosis of myotonic dystrophy type 1 (DM1)
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Willing to have a total of 2 needle muscle biopsies (a sample of your leg tissue will be collected) over several months
Available to attend all study visits for the duration of the study
*Other study requirements will apply.
Participants will be compensated for study visit time and reimbursed for travel-related expenses.
Participation in the FREEDOM2-DM1 study lasts about 5.5 months and involves up to 10 visits to the study site. Study assessments include receiving the study drug, blood and urine tests, questionnaires, muscle tests, a heart test (ECG), breathing tests, and 2 muscle biopsies if you complete the entire study.
Participation in a clinical trial is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
Take the pre-screener to see if you may qualify!
Take the Pre-ScreenerPGN-EDODM1 is an investigational medication designed to address the root cause of DM1 and restore the body’s ability to correctly process the proteins affected by DM1. This restoration may slow, halt, or reverse DM1 symptoms. The study medication (either PGN-EDODM1 or placebo) will be given as an IV infusion (through a needle in a vein) every 4 weeks. In this study there will be a total of 4 doses. Each infusion takes about an hour.
Participants will be randomized as a 3 out of 4 chance, where:
· 3 out of every 4 participants will receive the investigational medication (PGN-EDODM1)
· 1 out of every 4 will receive a placebo (no active ingredients)
Neither you nor the study staff will know which you have received.
An investigational drug is a substance that is being tested in clinical research studies and may or may not be approved by the regulatory authorities (e.g. U.S. Food and Drug Administration, Medicines and Healthcare products Regulatory Agency or Health Canada) for treatment of this condition. Researchers compare the investigational drug to a placebo. A placebo is a substance that has no active drug in it.
Take the pre-screener to see if you may qualify!
Take the Pre-ScreenerOur genes provide instructions (genetic code) to our cells to make proteins that play important roles in our bodies. People with myotonic dystrophy type 1 (DM1), also known as Steinert Disease) have a mutation (or error) in their genetic code in a gene called myotonic dystrophy protein kinase (DMPK).
This mutation prevents the body from correctly processing certain proteins that are important for muscle, respiratory, cardiac health, and brain function. Many types of proteins can be affected, accounting for the wide variety of possible DM1 symptoms.
Here are some common questions and answers about study participation.
Clinical research studies, or trials, help scientists and doctors explore whether a medical strategy, device, or medication is safe and effective for people. Before any medication can be approved and made available to the public, it must go through several phases of clinical research.
Before enrolling in a clinical trial, you must sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive. If you are under the age of consent you will provide Assent on a similar form, and your legal guardian will sign the consent form.
Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study.
Participation in clinical research studies is your choice, and you may stop at any time.
If you have additional questions about participating in a clinical trial, contact a study clinic near you.
You will play an active role in your own health care, will receive frequent health check-ups as part of your participation, and may help others by contributing to what we know about DM1.
You do not have to pay for participation in a clinical research study. This includes the investigational drug, study supplies, study visits, or tests that are part of the study. You will be compensated for your study visit time and travel expenses.
If you have additional questions about participating in a clinical research study, contact a study clinic near you.
To learn more about FREEDOM2-DM1 study and for study clinic locations, visit clinicaltrials.gov.