FREEDOM-DM1 Study

What is the FREEDOM-DM1 study?

The FREEDOM-DM1 study is a Phase 1 study that is exploring whether a single dose of the investigational drug, called PGN-EDODM1, is safe and tolerable for people with DM1 compared to a placebo (a substance that has no active drug). FREEDOM-DM1 will enroll approximately 24 people with DM1.

Who can join the FREEDOM-DM1 study?

You may be able to join the study if you meet the following requirements:

• Age 18-50
• Have myotonic dystrophy type 1 (DM1)
• Willing to have a total of 3 needle muscle biopsies (a sample of your leg tissue will be collected) over several months.

Other study requirements will apply.

Participants will be compensated for their study visit time and reimbursed for travel expenses.

What will happen during the study?

If you are interested in participating, the study team will schedule a screening visit to see if you qualify for the study.

At this visit you will:

• Be asked about your medical history and medications
• Have blood and urine tests
• Have your heart and lungs tested
• Do some simple muscle exercises
• Fill out some questionnaires.
• If you are eligible to be in the study, you will also have a muscle biopsy done.

If you are qualified and enroll in the study, you will be randomized to receive a single infusion of PGN-EDODM1 or placebo. You will have a 3 out of 4 chances of receiving PGN-EDODM1 instead of placebo. Neither you nor the study staff will know which you have received. After your infusion, you will stay for observation and safety tests.

Participation in the FREEDOM-DM1 study lasts about 5 months and involves up to 6 visits to the study site. You will have a total of 3 muscle biopsies over the course of the study if you complete the entire study.

Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.