Ignite Creation Date:
2024-05-19 @ 5:33 PM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06411288
Status:
RECRUITING
Last Update Posted:
2024-06-12
First Post:
2024-05-08
Brief Title:
Global Study of Del-desiran for the Treatment of DM1
Sponsor:
Avidity Biosciences Inc
Organization:
Avidity Biosciences Inc
Study Overview
Official Title:
A Phase 3 Randomized Double-Blind Placebo-Controlled Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HARBOR
Brief Summary:
A Phase 3 Randomized Double-Blind Placebo-Controlled Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran abbreviated del-desiran formerly AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Detailed Description:
The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period The anticipated duration is approximately 60 weeks
Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses The final dose will occur at Week 48 followed by a final assessment at Week 54
After completion of Week 54 assessments eligible participants will have the option to enroll into an open label extension OLE study pending regulatory approval
An Independent Data Monitoring Committee IDMC comprised of members independent and external to the Sponsor will review safety tolerability and efficacy as needed data of this study at regular intervals
Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses The final dose will occur at Week 48 followed by a final assessment at Week 54
After completion of Week 54 assessments eligible participants will have the option to enroll into an open label extension OLE study pending regulatory approval
An Independent Data Monitoring Committee IDMC comprised of members independent and external to the Sponsor will review safety tolerability and efficacy as needed data of this study at regular intervals
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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