Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronucle… (NCT04743557) | Clinical Trial Compass
WithdrawnPhase 1/2
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
Stopped: The first Clinical trial with DYN101 (UNITE-CNM) was early terminated. As a consequence, Dynacure decided to not perform this study.
France, Germany0Started 2024-01
Plain-language summary
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of DYN101 in participants 2 to 17 years of age with CNM caused by mutations in DNM2 or MTM1.The trial will consist of a pre-screening consent, a screening period, a run-in period (if applicable), and a Part 1 of 12 weeks with weekly infusion of DYN101 to evaluate safety and tolerability as well as PK, PD and preliminary efficacy. The dose level may need adjustment based on the Part 1 results of the current study and available data from the Unite-CNM study (DYN101-C101, NCT04033159). If a dose adjustment is needed, Part 2 will be conducted in the same participants and the newly selected dose level will be used to assess whether efficacy is seen after an additional 12 weeks of treatment. As this trial is investigational, there is no defined, expected benefit for subjects who participate in this trial except a better knowledge of their disease.
Who can participate
Age range
2 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female aged ≥2 to \<18 years on the date the main ICF is signed.
. Have a clinically symptomatic CNM, with a documented MTM1 or DNM2 mutation.
. Have impaired muscle function as evidenced by:
. Have sufficient skeletal muscle (vastus lateralis, gastrocnemius, or biceps brachii as last resort) to perform 2 open muscle biopsies during the trial, as determined by ultrasound imaging at screening.
. Subject must have platelet count \>150,000/µL at screening.
. Parent(s) or legally-authorized representative must be able to provide written, signed and dated informed consent for their child to participate in the trial. Informed assent can be obtained from the child according to local regulations.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of drug-related Treatment Emergent Adverse Events (TEAEs)
. Parent(s) or legally-authorized representative must be at or above the age of legal consent in the jurisdiction of the country in which the trial is taking place.
. Subject, parent(s), and/or legally-authorized representative must have an understanding, ability, and willingness to fully comply with visit frequency, trial procedures, videorecording of assessments where applicable, and restrictions, including contraceptive requirements.
Exclusion criteria
. Subject has evidence of clinically significant liver disease.
. Subject has evidence of clinically significant renal disease.
. Presence of significant comorbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, clinical laboratory evaluation, vital signs, or ECG recording for which, in the opinion of the investigator and/or 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).
. Subject currently enrolled in any interventional trial or scheduled to participate in such a trial whilst participating in the current trial.
. Subject has previously received gene therapy for CNM.
. Subject has severe muscle contractures that would preclude the ability to show improvement in the MFM32 assessment, in the opinion of the investigator.
. Subject has severe airway malacia which could impact the capacity to wean off ventilatory support.