Our Clinical Trials

Patient needs are what drive us to innovate.

At ARTHEx, we recognize that persons living with DM are eager to see potential drugs studied in clinical trials to give the community hope for a novel therapy.  Patients are at the center of what we do at ARTHEx and we are working hard to advance our investigational therapies toward the clinic to improve patient outcomes and quality of life.
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We are actively recruiting patients to participate in a clinical trial to evaluate our therapeutic candidate, ATX-01, for DM1. If you believe you or a loved one might be a good candidate for our trial, we encourage you to reach out directly through the link below.
Arthex Clinical Trial
ArthemiR™ Trial
First in Human Study in DM1 Persons
A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study in Persons with Classic Myotonic Dystrophy Type 1 (DM1)
Objectives
PRIMARY OBJECTIVES
‍Safety and tolerability of single and multiple ascending doses of ATX-01 in DM1 Persons
SECONDARY OBJECTIVES
‍Target engagement at the muscle level via biomarkers
(MBNL levels, splicing index)
EXPLORATORY OBJECTIVES
‍Clinical endpoints will include measures related to muscle function and patient-reported outcomes
ArthemiR trial is co-funded by EIC Accelerator program under the Grant Agreement Nº 190181217

Clinical Sites Now Open

For further information on the study and which trial sites are open, click the link below.
CLINICALTRIALS.GOV

Key Enrollment Criteria

Inclusion Criteria
  • Male or Female; Age 18-64
  • Diagnosis of DM1 >150 CTG repeats
  • Ambulatory
  • Myotonia (vHOT>3 secs)
  • Signed Informed Consent
Exclusion Criteria
  • Congenital DM1
  • Contraindication to muscle biopsies
  • On anti-myotonia medication
  • Moderate-advanced cardiac disease
  • Other health conditions that might put participation at risk

Trial Design

Trial Design

Find Out More Information

CLINICALTRIALS.GOV