BridgeMD
New treatment option for Myotonic Dystrophy 1
Official title Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
ClinicalTrials.gov ID: NCT06138743
What this study is testing
What is ARO-DM1 Intravenous (IV) Infusion?
ARO-DM1 Intravenous (IV) Infusion 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
- This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of ARO-DM1 or 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, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- 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
- people of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days...
You likely can't join if
- Inadequately controlled diabetes
- Confirmed diagnosis of congenital DM1
- Uncontrolled hypertension
- 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
See the full eligibility 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
- people 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...
- Inadequately controlled diabetes
- Confirmed diagnosis of congenital DM1
- Uncontrolled hypertension
- 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) Note: Additional inclusion/exclusion...
The study team makes the final eligibility decision.
Where it's taking place
- Liverpool, New South Wales, Australia
- Birtinya, Queensland, Australia
- Herston, Queensland, Australia
- Melbourne, Victoria, Australia
- Christchurch, New Zealand
- Taichung, Taiwan
- Taipei, Taiwan
- Bangkok, Bangkok, Thailand
- Hat Yai, Changwat Songkhla, Thailand
- Lampang, Thailand
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, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Liverpool, New South Wales, Australia; Birtinya, Queensland, Australia; Herston, Queensland, Australia; Melbourne, Victoria, Australia; Christchurch, New Zealand; Taichung, Taiwan and 4 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.