Studies
| Study First Submitted Date | 2021-02-03 |
| Study First Posted Date | 2021-02-08 |
| Last Update Posted Date | 2022-07-11 |
| Start Month Year | January 2024 |
| Primary Completion Month Year | September 2025 |
| Verification Month Year | July 2022 |
| Verification Date | 2022-07-31 |
| Last Update Posted Date | 2022-07-11 |
Facilities
| Sequence: | 198467859 | Sequence: | 198467860 |
| Name | I-Motion Institute – Trousseau Hospital | Name | Universitätsklinikum Essen |
| City | Paris | City | Essen |
| Zip | 75012 | Zip | 45122 |
| Country | France | Country | Germany |
Conditions
| Sequence: | 51746915 |
| Name | Centronuclear Myopathy |
| Downcase Name | centronuclear myopathy |
Id Information
| Sequence: | 39820346 | Sequence: | 39820347 |
| Id Source | org_study_id | Id Source | secondary_id |
| Id Value | DYN101-C102 | Id Value | 2020-004608-32 |
| Id Type | EudraCT Number | ||
Countries
| Sequence: | 42219522 | Sequence: | 42219523 |
| Name | France | Name | Germany |
| Removed | False | Removed | False |
Design Groups
| Sequence: | 55167788 |
| Group Type | Experimental |
| Title | Cohort 1 |
| Description | Weekly infusions of DYN101 at the starting dose level |
Interventions
| Sequence: | 52068520 |
| Intervention Type | Drug |
| Name | DYN101 |
| Description | DYN101, is a constrained ethyl gapmer ASO directed against human DNM2 pre-mRNA |
Keywords
| Sequence: | 79173751 | Sequence: | 79173752 | Sequence: | 79173753 | Sequence: | 79173754 | Sequence: | 79173755 |
| Name | Muscular Diseases | Name | Myopathies, Structural, Congenital | Name | Musculoskeletal Diseases | Name | Neuromuscular Diseases | Name | Nervous System Diseases |
| Downcase Name | muscular diseases | Downcase Name | myopathies, structural, congenital | Downcase Name | musculoskeletal diseases | Downcase Name | neuromuscular diseases | Downcase Name | nervous system diseases |
Design Outcomes
| Sequence: | 176005791 | Sequence: | 176005792 | Sequence: | 176005793 | Sequence: | 176005794 |
| Outcome Type | primary | Outcome Type | secondary | Outcome Type | secondary | Outcome Type | secondary |
| Measure | Incidence of drug-related Treatment Emergent Adverse Events (TEAEs) | Measure | Measurement of DYN101 concentration in plasma | Measure | Maximum plasma drug concentration (Cmax) for DYN101 | Measure | Area under the Plasma Concentration versus Time Curve (AUC) of DYN101 |
| Time Frame | Baseline until Week 12 | Time Frame | Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion | Time Frame | Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion | Time Frame | Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion |
Browse Conditions
| Sequence: | 191777871 | Sequence: | 191777872 | Sequence: | 191777873 | Sequence: | 191777874 | Sequence: | 191777875 |
| Mesh Term | Muscular Diseases | Mesh Term | Myopathies, Structural, Congenital | Mesh Term | Musculoskeletal Diseases | Mesh Term | Neuromuscular Diseases | Mesh Term | Nervous System Diseases |
| Downcase Mesh Term | muscular diseases | Downcase Mesh Term | myopathies, structural, congenital | Downcase Mesh Term | musculoskeletal diseases | Downcase Mesh Term | neuromuscular diseases | Downcase Mesh Term | nervous system diseases |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 47925056 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | Dynacure |
Design Group Interventions
| Sequence: | 67633794 |
| Design Group Id | 55167788 |
| Intervention Id | 52068520 |
Eligibilities
| Sequence: | 30517697 |
| Gender | All |
| Minimum Age | 2 Years |
| Maximum Age | 17 Years |
| Healthy Volunteers | No |
| Criteria | 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: MFM20 score between 5% and 80% for subjects ≥2 and <6 years of age, or MFM32 score between 5% and 80% for subjects ≥6 years of age. 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. 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. Subject requires oxygen supplementation. For female subjects of childbearing potential: pregnant, breastfeeding, or planning to become pregnant during the trial. Current or relevant history of physical or psychiatric illness, and/or 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 investigational medicinal product (IMP) or procedures. Intake of any disallowed therapies by the subject within 12 weeks before the planned first IMP administration. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely related compounds. Parent(s) or legally authorized representative are legally incapacitated or have limited legal capacity, or have lack of mental capacity to fully understand the protocol requirements and ensure completion of all required trial procedures. |
| Adult | False |
| Child | True |
| Older Adult | False |
Calculated Values
| Sequence: | 254070224 |
| Number Of Facilities | 2 |
| Registered In Calendar Year | 2021 |
| Were Results Reported | False |
| Has Us Facility | False |
| Has Single Facility | False |
| Minimum Age Num | 2 |
| Maximum Age Num | 17 |
| Minimum Age Unit | Years |
| Maximum Age Unit | Years |
| Number Of Primary Outcomes To Measure | 1 |
| Number Of Secondary Outcomes To Measure | 3 |
Designs
| Sequence: | 30266723 |
| Allocation | N/A |
| Intervention Model | Single Group Assignment |
| Observational Model | |
| Primary Purpose | Treatment |
| Time Perspective | |
| Masking | None (Open Label) |
Responsible Parties
| Sequence: | 28646928 |
| Responsible Party Type | Sponsor |
Study References
| Sequence: | 51639122 |
| Pmid | 28589938 |
| Reference Type | background |
| Citation | Tasfaout H, Buono S, Guo S, Kretz C, Messaddeq N, Booten S, Greenlee S, Monia BP, Cowling BS, Laporte J. Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice. Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661. |