Phase
Condition
Muscular Dystrophy
Treatment
Placebo
ARO-DM1 for Injection
Clinical Study ID
Ages 18-65 All Genders
Study Summary
Eligibility Criteria
Inclusion
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 contraceptionin addition to a condom during the study and for at least 90 days following the endof study or last dose of study drug, whichever is later. Subjects must not donatesperm or eggs during the study and for at least 90 days following the end of studyor last dose of study drug whichever is later.
Exclusion
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 toundergo 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 themedication prior to Screening.
Abnormal coagulation parameters at Screening including platelet count, InternationalNormalized Ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)
History or presence of any of the following: hypercoagulable state, nephrotic rangeproteinuria, antiphospholipid antibody syndrome or myeloproliferative diseases,inability to ambulate, use of hormone-based contraceptives and peri/post- menopausalhormone replacement therapy ≤ 16 weeks prior to Day 1
Note: Additional inclusion/exclusion criteria may apply per protocol
Study Design
Connect with a study center
Research Site
Birtinya, Queensland 4575
AustraliaActive - Recruiting
Research Site
Herston, Queensland 4006
AustraliaActive - Recruiting
Research Site
Melbourne, Victoria 3004
AustraliaActive - Recruiting
Research Site
Christchurch, 8011
New ZealandActive - Recruiting
Research Site
Taichung, 40447
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei, 10002
TaiwanActive - Recruiting
Research Site
Taipei, 112201
TaiwanActive - Recruiting
Research Site
Hat Yai, Songkhla 90110
ThailandActive - Recruiting
Research Site
Bangkok, 10700
ThailandActive - Recruiting
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