Health TrialsbyHealthPanel

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

ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorArrowhead Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • ARO-DM1 Intravenous (IV) Infusion

    Drug

    ARO-DM1 by intravenous (IV) infusion

  • Placebo Intravenous (IV) Infusion

    Drug

    0.9% NaCl calculated volume to match active treatment by IV infusion

  • ARO-DM1 subcutaneous (SC) injection

    Drug

    ARO-DM1 by subcutaneous (SC) injection(s)

  • Placebo Subcutaneous (SC) Injection

    Drug

    0.9% NaCl calculated volume to match active treatment by SC injection(s)

Treatment groups

78 Participants
are divided into 4 treatment groups
Group A: ARO-DM1 Intravenous (IV) InfusionExperimental treatment 1 intervention
Group B: Placebo by IV InfusionPlacebo comparator 1 intervention
Group C: ARO-DM1 Subcutaneous SC InjectionExperimental treatment 1 intervention
Group D: Placebo by SC InjectionPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)

Time frame
Single-dose phase (Part 1): Up to Day 90(EOS); multiple-dose phase (Part 2): Up to Day 180(EOS)

Secondary outcomes

1

Pharmacokinetics (PK) of ARO-DM1: Maximum Observed Plasma Concentration (Cmax)

Time frame
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
2

PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

Time frame
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
3

PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

Time frame
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
4

PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)

Time frame
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Arrowhead Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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