Muscle illness
Conditions
Interventions
Name of Investigational Medicinal Product: DYN101
Sponsors
Dynacure
Eligibility
Age
16 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male or female aged >= 16 years of age* on the date of signing the main ICF. The first subject (i.e. the sentinel subject) in each cohort must be >= 18 years of age. *for Germany, the subject must be >= 18 years of age in accordance with local and national regulations. 2. Have a documented mutation in DNM2 or MTM1. 3. Platelet count > 150,000/µL, 4. Have a symptomatic CNM in the opinion of the investigator, at least mild to moderately affected, i.e. showing clinical symptoms in at least 2 of the 4 relevant domains that will be investigated in this trial (respiratory function, muscle strength, muscle function, dysphagia), and be ambulatory, i.e. being able to walk 10 steps, if needed with support/assisted. If a subject is non-ambulatory but highly functioning in the view of the investigator, he/she may be included following discussion with the sponsor. 5. Have an understanding, ability and willingness to fully comply with visit frequency, trial procedures and restrictions, including contraceptive requirements. 6. Able to provide written, signed and dated informed consent/assent to participate in the trial. Parental consent (one or both parents) and an assent for subjects
Exclusion criteria
Exclusion criteria: 1. Clinically significant liver disease. 2. Clinically significant renal disease 3. Presence of significant co-morbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, laboratory testing, vital signs or ECG recording for which, in the opinion of the investigator and the medical monitor, participation would not be in the best interest of the subject (e.g. compromise the safety or well-being) or that could prevent, limit, or confound the protocol-specified assessments (e.g. taking a muscle biopsy). 4. For female subjects of child-bearing potential: pregnant or breastfeeding, or planning to become pregnant during the trial. 5. Current or past abuse of alcohol or recreational/narcotic drugs (with the exception of caffeine and nicotine), which in the investigator*s opinion would compromise the subject*s safety and/or compliance with the trial procedures. 6. Currently enrolled in any interventional trial or scheduled to participate in another trial whilst participating in this trial. Subjects are allowed to participate in registry studies. 7. Current or relevant history of physical or psychiatric illness, any medical disorder that may require treatment or make the subject unlikely to fully complete the trial, or any condition that presents undue risk from the IMP or procedures. 8. Intake of any disallowed therapies as noted in the protocol within 12 weeks before the planned first IMP administration. 9. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely-related compounds, or history of a significant allergic reaction to IMP ingredients as determined by the investigator, such as anaphylaxis requiring hospitalization. 10. Legally incapacitated or have limited legal capacity. Lack of mental capacity to fully understand the protocol requirements and complete all study required procedures.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine safety and tolerability of SAD and MAD of DYN101, assessed by the number and severity of AEs and SAEs reported from signing of the main ICF onwards until the last trial visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| • To determine PK parameters of DYN101 in plasma following SAD and MAD of DYN101. • To assess effects of DYN101 (including DNM2 mRNA levels and DYN101 concentration) in muscle biopsies. | — |
Countries
Netherlands
Outcome results
None listed