ENSAYO CLÍNICO

Reclutando

Fase III

Terapia celular y T-CARs, Trombosis y Anticoagulantes, Linfoma

Abril, 2026

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1. Age 16 to 65 years at the time of signing the informed consent/assent form
8. If being treated with testosterone, on a stable replacement dose for 30 days prior to screening
9. Participants must agree to follow protocol-specified highly effective contraception guidance as described in the protocol
11. Capable of giving signed informed consent/assent as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol and authorization to use health information that is confidential according to national and local privacy regulations
12. Willingness and ability of participant to comply with and tolerate scheduled visits, dosing administration plan, and study assessments over the duration of the study
2. Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size > 100. Historical results from clinical testing are acceptable
3. Clinically apparent myotonia equivalent to hand opening time of at least 3 seconds as determined by a central reading of vHOT (middle finger) values
4. Hand grip strength averaged from both sides 15% and 85% predicted of normal
5. 5×STS completion in 8 seconds without the use of assistive devices such as canes or walkers. The use of braces, such as ankle braces, and orthoses such as submalleolar orthoses, and inserts or supports that do not extend above the malleolus are permitted during testing.
6. Able to complete 10-MWRT and 5×STS without the use of assistive devices such as canes, walkers, or orthoses. The use of submalleolar orthoses and inserts or supports that do not extend above the malleolus are permitted during testing
7. Body mass index (BMI) < 35kg/m2

1. Previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that in the opinion of the Investigator could affect safety, make it unlikely that the dosing schedule and follow-up will be correctly completed, and/or impair the assessment and interpretation of study results
12. Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments
13. Uncontrolled diabetes mellitus or other serious medical illness that may complicate interpretation of safety or efficacy in the study, in the opinion of the Investigator
19. Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments
14. Individuals with a history or presence of second- or third-degree heart block, ventricular arrhythmias, ECG with the corrected QT interval by Fridericias Formula (QTcF) 450 ms in men and QTcF 460 ms in women, PR 240 ms; left bundle-branch block, or a conduction defect that is clinically significant in the opinion of the Investigator are excluded unless they have an implanted pacemaker or defibrillator that was implanted more than 2 weeks prior to screening
15. Individuals with known structural heart disease, evidence of noncompaction cardiomyopathy, or a known (at most recent imaging) left ventricular ejection fraction of < 40%
16. Individual has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of document or who, in the opinion of the Investigator, is at significant risk to commit suicide
17. Treatment with medications that can improve myotonia or clinical functional endpoints within a period of 5 half-lives of the medication prior to performing screening assessments. ay include, but not limited to, mexiletine, phenytoin, carbamazepine, procainamide, disopyramide, ranolazine, flecainide, lamotrigine, nifedipine, acetazolamide, clomipramine, imipramine, amitriptyline, taurine, or quinine
18. Current treatment with systemic immunosuppressive therapy
4. A known diagnosis of congenital DM1
2. History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator, or an expectation of a major surgical procedure during the Placebo-Controlled Period of the study (based on Investigator judgment).
10. Persistent systolic blood pressure < 90 mm Hg or signs or symptoms of hypotension or volume depletion/dehydration
3. History of DVT or PE within the last 5 years
5. History of clinically significant liver disease or ongoing treatment for liver disease or confirmed elevated ALT > 3× ULN at screening
6. History of clinically significant hematologic disease or have any of the following hematologic results at screening: platelets or hemoglobin below the lower limit of normal for age and sex
7. History of clinically significant kidney disease, ongoing treatment for kidney disease (treatment for hypertension is permitted) or eGFR < 60 mL/min as calculated with the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Cystatin C Equation
8. Acute kidney injury within 12 weeks prior to Screening, characterized by an increase in serum creatinine of 0.3 mg/dL within 48 hours, or of 1.5-fold within a week
9. Hematuria > 5 red blood cells per high power field (RBC/HPF) on urinalysis at screening
Inability to comply with physical activity requirements (eg., abstaining from strenuous exercise) from Screening through the end of the Placebo-Controlled Period
Any participant who is pregnant or breastfeeding or is planning to become pregnant during the study
20. Use of nephrotoxic medications within a period of 5 half-lives of the medication prior to performing screening assessments. These may include, but are not limited to, nonsteroidal anti-inflammatory drugs (NSAIDs), aminoglycosides (eg, gentamicin and streptomycin), bisphosphonates, and antiviral agents (eg, acyclovir and adefovir). Planned procedures that require contrast during the study are also exclusionary
11. Active malignancy or history within the last 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix thathat has been successfully excised and considered cured at least 1 year prior to Screening. Participants with cancers in remission for >5 years prior to Screening may be included after discussion with the Medical Monitor.
21. Receipt of any prior gene therapy, or receipt of another investigational drug, biologic agent, or device within 5 half-lives (if known) of the agent, or within 4 months prior to the start of Screening, whichever is longer. Individuals previously treated with oligonucleotide therapies (including small interfering RNA [siRNA]) may be eligible if the last dose of the investigational drug was received 3 years ago
22. Percent predicted forced vital capacity (FVC) < 50% (sitting position)
23. Recent physical inactivity (eg, immobilization of 3 days) if, in the opinion of the Investigator, this would hinder the participant from complying with study requirements
24. Inability to undergo venipuncture successfully or tolerate venous access
25. Inability, or impaired ability, to complete study procedures and/or complete the study, due to physical or cognitive impairment or illicit drug or alcohol abuse, in the judgment of the Investigator

To evaluate the efficacy of DYNE 101 compared with placebo as measured by improvement in muscle function

antagonistas de la vitamin k, bispecific antibody, vitamin k antagonist