Phase 1/2a study of ATX-01 in participants with DM1
- Conditions
- Myotonic dystrophy type 1 (DM1)MedDRA version: 20.0Level: PTClassification code: 10068871Term: Myotonic dystrophy Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-505363-37-00
- Lead Sponsor
- Arthex Biotech S.L.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 56
1. Participant must be 18 to 64 years of age inclusive, at the time of signing the informed consent., 2. Participants with a documented clinical diagnosis of DM1 (CTG expansion of >150 repeats in DMPK gene measured in peripheral blood mononuclear cells [PBMCs])., 3. Ambulatory, defined as able to complete a 10-meter walk/run test (10MWRT) at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses., 4. Presence for >3 seconds of grip myotonia as confirmed by a central reader., 5. BMI <35 kg/m²., 6. Male and female participants., 7. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol., 8. The participant agrees not to post any personal medical data related to the study or information related to the study on any website or social media (e.g., Facebook, Twitter) or discuss the study publicly until the entire study has been completed., 9. The participant is able to have muscle biopsies as per the Schedule of Activities.
1. Participants with congenital DM1., 18. Contraindication to a muscle biopsy defined as: use of an anticoagulant, platelet count < 50,000, or other bleeding disorder., 19. Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening., 2. Prior or ongoing medical condition, medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results., 20. Exposure to another investigational drug within 3 months prior to start of study treatment., 21. Use of medications which are sensitive substrates of CCI, CCI, or CCI., 3. Medical Research Council Muscle Scale score of = 3 on ankle dorsiflexion (either ankle) or significant tibialis anterior atrophy that prevents a muscle biopsy., 4. Active COVID-19 infection., 5. History of pacemaker or implantable cardioverter-defibrillator; atrial fibrillation or flutter; ventricular tachycardia; any other sustained atrial or ventricular arrhythmias; undiagnosed syncope in the last year; congestive heart failure; myocardial infarction; coronary artery disease; congenital heart disease; moderate or severe valvular heart disease. Participants with pacemakers implanted for solely prophylactic reasons can be included after confirmation from the medical monitor., 6. Participants with a family history of sudden cardiac death, unexplained death, long QT syndrome, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member that is not related to an underlying DM1 diagnosis., 7. Participants with serum electrolyte abnormalities that cannot be corrected., 10. Breast cancer within the past 10 years., 8. Participants receiving concomitant QTc prolonging medications unless on stable doses., 9. Lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years., 22. Use of medications which are CCI, including regular use of CCI., 23. Participants with planned procedures requiring an CCI during the study., 24. Ongoing participation in any other interventional therapeutic clinical trial., 25. Screening systolic blood pressure > 160 mmHg systolic and/or diastolic blood pressure > 100 mmHg. Blood pressure measurements may be repeated up to 3 times if anxiety is thought to be responsible for an elevation of blood pressure., 26. Non-sinus rhythm, any second or third degree heart block, PR interval > 220 ms, QRS duration > 110 ms, QTcF > 450 ms (males and females), on a 12-lead ECG at screening., 27. On a 24-hour ambulatory (Holter) ECG with at least 18 hours of interpretable recordings: any sustained (> 30 seconds) of atrial or ventricular arrhythmias, non- sustained ventricular tachycardia (3 or more beats at > 100 beats per minute [BPM]), Mobitz type II 2nd or 3rd-degree heart block, mean heart rate < 50 BPM in waking hours, any rate pauses > 3.0 seconds in waking hours. For entry into Part 2 (MAD), the 24-hour Holter ECG is required for treatment naïve participants, or if the participant was in the SAD and 6 months have elapsed from the SAD screening Holter assessment., 28. Transthoracic Echocardiogram ejection fraction < 45% at Screening or if the participant has had an
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method