Ignite Creation Date:
2024-05-06 @ 3:45 PM
Last Modification Date:
2024-10-26 @ 1:56 PM
Study NCT ID:
NCT04743557
Status:
WITHDRAWN
Last Update Posted:
2022-07-11
First Post:
2021-02-03
Brief Title:
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
Sponsor:
Dynacure
Organization:
Dynacure
Study Overview
Official Title:
A Phase 12 Multicenter Open-label Dose-confirmation Trial to Evaluate the Safety and Preliminary Efficacy of DYN101 in Participants 2 to 17 Years of Age With Centronuclear Myopathy Caused by Mutations in MTM1 or DNM2
Status:
WITHDRAWN
Status Verified Date:
2022-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The first Clinical trial with DYN101 UNITE-CNM was early terminated As a consequence Dynacure decided to not perform this study
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
DyNaMic
Brief 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 MTM1The 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2020-004608-32 | EUDRACT_NUMBER | None | None |