ATX-01 for Myotonic Dystrophy
(ArthemiR Trial)
Recruiting in Palo Alto (17 mi)
+8 other locations
Age: 18 - 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: ARTHEx Biotech S.L.
Must not be taking: Mexiletine, others
Disqualifiers: Congenital DM1, others
Approved in 2 Jurisdictions
Trial Summary
What is the purpose of this trial?The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo.
ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.
Will I have to stop taking my current medications?
The trial requires that you stop using mexiletine or any other medication for myotonia at least 21 days before screening.
How is the drug ATX-01 different from other treatments for myotonic dystrophy?
ATX-01 is a novel treatment being explored for myotonic dystrophy, a condition with limited existing therapies. Unlike other treatments, ATX-01 may offer a new approach, potentially involving unique mechanisms or formulations, as current options like tideglusib, mexiletine, or metformin are repurposed drugs.
12345Eligibility Criteria
This trial is for people with myotonic dystrophy type 1 (DM1) who can walk 10 meters without help (except ankle-foot braces), have a specific genetic marker in their blood, and experience grip myotonia lasting more than three seconds.Inclusion Criteria
I can walk 10 meters without help, except for ankle-foot braces.
Presence for >3 seconds of grip myotonia as confirmed by a central reader
I have been diagnosed with DM1, confirmed by a specific genetic test.
Exclusion Criteria
I was born with myotonic dystrophy type 1.
I cannot move my ankle upwards well or have severe muscle loss in the front of my lower leg.
I haven't taken mexiletine or similar drugs for myotonia in the last 21 days or 5 half-lives, whichever is longer.
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Single-Ascending Dose
Participants receive one dose of ATX-01 or placebo
1 week
Multiple-Ascending Dose
Participants receive three doses of ATX-01 or placebo
3 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
16 weeks
Participant Groups
The study tests ATX-01, an experimental drug designed to inhibit a microRNA linked to DM1. Participants will receive either one dose of ATX-01 or placebo in the first part, followed by three doses in the second part.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ATX-01Experimental Treatment1 Intervention
ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide
Group II: PlaceboPlacebo Group1 Intervention
Placebo to ATX-01
ATX-01 is already approved in United States, European Union for the following indications:
🇺🇸 Approved in United States as ATX-01 for:
- None approved; Orphan Drug Designation for Myotonic Dystrophy Type 1 (DM1)
🇪🇺 Approved in European Union as ATX-01 for:
- None approved; Orphan Drug Designation for Myotonic Dystrophy Type 1 (DM1)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of Kansas Medical Center, Department of NeurologyFairway, KS
University of FloridaGainesville, FL
Virginia Commonwealth UniversityRichmond, VA
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-JeanChicoutimi, Canada
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Who Is Running the Clinical Trial?
ARTHEx Biotech S.L.Lead Sponsor
References
Characteristics of myotonic dystrophy patients in the national registry of Japan. [2022]Myotonic dystrophies (DM) are inherited autosomal dominant disorders affecting multiple organs. Currently available therapeutics for DM are limited; therefore, a patient registry is essential for therapeutic development and success of clinical trials targeting the diseases. We have developed a nationwide DM registry in Japan under the Registry of Muscular Dystrophy (Remudy). The registration process was patient-initiated; however, physicians certified the clinical information. The dataset includes all Naarden and TREAT-NMD core datasets and additional items covering major DM clinical features. As of March 2020, we enrolled 976 patients with genetically confirmed DM. The majority (99.9%) of these patients had DM1, with 11.4% having the congenital form. However, 1 patient had DM2. Upon classifying 969 symptomatic DM1 patients based on their age at onset, an earlier onset was associated with a longer CTG repeat length. Myotonia was the most frequent symptom, followed by hand disability, fatigue, and daytime sleepiness. The frequency of hand disabilities, constipation, and visual disturbances was higher for patients with congenital DM. According to a multiple regression analysis of objective clinical measurements related to prognosis and activities of daily living, CTG repeat length strongly influenced the grip strength, forced vital capacity, and QRS time in an electrocardiogram. However, the grip strength was only modestly related to disease duration. This report will shed light on the Japanese national DM registry, which has recruited a significant number of patients. The registry will provide invaluable data for planning clinical trials and improving the standard of care for patients.
[Correlation of muscular impairment rating scale with myopathological changes in myotonic dystrophy type 1]. [2014]To explore the relationship between muscular impairment rating scale (MIRS) and myopathological changes of myotonic dystrophy type 1 (DM1).
Myotonic dystrophy type 1 drug development: A pipeline toward the market. [2022]Myotonic dystrophy type 1 (DM1) is a multisystemic neuromuscular genetic disease with an estimated prevalence of approximately at least half a million individuals based on its vast ethnic variation. Building upon a well-known physiopathology and several proof-of-concept therapeutic approaches, herein we compile a comprehensive overview of the most recent drug development programs under preclinical and clinical evaluation. Specifically, close to two dozen drug developments, eight of which are already in clinical trials, explore a diversity of new chemical entities, drug repurposing, oligonucleotide, and gene therapy-based approaches. Of these, repurposing of tideglusib, mexiletine, or metformin appear to be therapies with the most potential to receive marketing authorization for DM1.
Assessing upper extremity capacity as a potential indicator of needs related to household activities for rehabilitation services in people with myotonic dystrophy type 1. [2015]This study aimed to assess upper extremity capacity as a potential indicator of needs related to household activities for rehabilitation services in people with myotonic dystrophy type 1 (DM1). A cross-sectional study was set in an outpatient neuromuscular clinic where 200 adults with a confirmed diagnosis of DM1 (121 women; mean age: 47 y) were selected from the registry of a neuromuscular clinic to participate. Housing-related activities were assessed using the "housing" section of the Assessment of Life Habits Questionnaire (LIFE-H). The upper extremity assessment included grip strength (Jamar dynamometer), lateral pinch strength (pinch gauge), gross dexterity (Box and Block Test) and fine dexterity (Purdue Pegboard Test). Correlations with the LIFE-H item "housing" were stronger for grip and lateral strength (r = 0.62; 0.61). When difficulties were present in "housing", the cut-off score associated with lateral pinch strength was 4.8 kg (sensitivity: 75.6%; specificity: 79.2%). Grip strength presented cut-off scores that clinically differed by gender. In conclusion, potential indicator of needs related to household activities for rehabilitation services with valid assessment tools were developed for people with DM1 who experience difficulties in housing-related activities. These criteria will assist health professionals in their attempt to refer DM1 patients to rehabilitation services at the appropriate time.
Functioning and disability in adults with myotonic dystrophy type 1. [2015]To provide a comprehensive description of functioning and disability with regard to stages of disease progression in adults with myotonic dystrophy type 1 (DM1). Further to explore associations of measures of manual dexterity and of walking capacity with measures of activities of daily living (ADL) and participation in social and lifestyle activities.