Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Launched by ARROWHEAD PHARMACEUTICALS · Nov 14, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ARO-DM1 for people with Type 1 Myotonic Dystrophy (DM1), a genetic condition that affects muscle function. The trial is in its early stages and aims to determine how safe the treatment is and how it works in the body. Participants will be randomly assigned to receive either ARO-DM1 or a placebo (a treatment that looks the same but has no active ingredients) over a series of doses.

To be eligible for this study, participants need to have a confirmed diagnosis of DM1, show certain symptoms of the condition, and be able to walk at least 10 meters on their own. The trial is open to both men and women aged 18 to 75. Participants will be carefully monitored throughout the study, and those who can become pregnant will need to use effective contraception during and for a period after the trial. This study is a valuable opportunity for those affected by DM1 to potentially access a new treatment while helping researchers learn more about this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Genetically confirmed diagnosis of DM1
• • Clinician-assessed signs of DM1 including clinically apparent myotonia
• • Onset of DM1 symptoms occurred after the age of 12 years
• • Walk for at least 10 meters independently at Screening
• • Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.
• Exclusion Criteria:
• • Inadequately controlled diabetes
• • Confirmed diagnosis of congenital DM1
• • Uncontrolled hypertension
• • History of thromboembolic events
• • History of Tibialis Anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period
• • Clinically significant cardiac, liver or renal disease
• • HIV infection (seropositive) at Screening
• • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at screening
• • Untreated or poorly controlled epilepsy
• • Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to Screening.
• • Abnormal coagulation parameters at Screening including platelet count, International Normalized Ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)
• • History or presence of any of the following: hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome or myeloproliferative diseases, inability to ambulate, use of hormone-based contraceptives and peri/post- menopausal hormone replacement therapy ≤ 16 weeks prior to Day 1
• • Note: Additional inclusion/exclusion criteria may apply per protocol