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Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies


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


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


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


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


Sequence: 52068520
Intervention Type Drug
Name DYN101
Description DYN101, is a constrained ethyl gapmer ASO directed against human DNM2 pre-mRNA


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


Sequence: 47925056
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Dynacure

Design Group Interventions

Sequence: 67633794
Design Group Id 55167788
Intervention Id 52068520


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


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.