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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06138743




Registration number
NCT06138743
Ethics application status
Date submitted
14/11/2023
Date registered
18/11/2023
Date last updated
1/10/2024

Titles & IDs
Public title
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Scientific title
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are =18 to = 65 Years
Secondary ID [1] 0 0
2024-513579-42
Secondary ID [2] 0 0
ARODM1-1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myotonic Dystrophy 1 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARO-DM1 for Injection
Treatment: Drugs - Placebo

Experimental: ARO-DM1 - ARO-DM1 for Injection

Placebo comparator: Placebo - (0.9% NaCl)


Treatment: Drugs: ARO-DM1 for Injection
single or multiple doses of ARO-DM1 by intravenous (IV) infusion

Treatment: Drugs: Placebo
calculated volume to match active treatment by IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
Timepoint [1] 0 0
Single dose phase (Part 1): Up to Day 90(EOS); Multiple dose phase (Part 2): Up to Day 180(EOS)
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of ARO-DM1: Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [2] 0 0
PK of ARO-DM1: Time to Maximum Observed Plasma Concentration (Tmax)
Timepoint [2] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [3] 0 0
PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Timepoint [3] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [4] 0 0
PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Timepoint [4] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [5] 0 0
PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Timepoint [5] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [6] 0 0
PK of ARO-DM1: Elimination half-life (t1/2)
Timepoint [6] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [7] 0 0
PK of ARO-DM1: Apparent Systemic Clearance (CL/F)
Timepoint [7] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose
Secondary outcome [8] 0 0
PK of ARO-DM1:m Apparent Terminal-phase Volume of Distribution (Vz/F)
Timepoint [8] 0 0
Single dose phase (Part 1): Up 24 hours post-dose; Multiple dose phase (Part 2): Through 24 hours post first and second dose

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/03/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2025
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Research Site - Birtinya
Recruitment hospital [2] 0 0
Research Site - Herston
Recruitment hospital [3] 0 0
Research Site - Melbourne
Recruitment postcode(s) [1] 0 0
4575 - Birtinya
Recruitment postcode(s) [2] 0 0
4006 - Herston
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06138743
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Monitor
Address 0 0
Country 0 0
Phone 0 0
626-304-3400
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06138743