Ignite Creation Date:
2024-05-06 @ 7:48 PM
Last Modification Date:
2024-10-26 @ 3:13 PM
Study NCT ID:
NCT06131983
Status:
RECRUITING
Last Update Posted:
2024-03-15
First Post:
2023-11-08
Brief Title:
Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Sponsor:
Arrowhead Pharmaceuticals
Organization:
Arrowhead Pharmaceuticals
Study Overview
Official Title:
A Phase12a Dose-Escalating Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular 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:
None
Brief Summary:
The purpose of this study is to evaluate the safety tolerability pharmacokinetics PK and pharmacodynamics PD of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 FSHD1 In Part 1 of the study participants will receive one dose of ARO-DUX4 or placebo In Part 2 of the study participants will receive 4 doses of ARO-DUX4 or placebo Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2 All participants will undergo pre- and post-dose MRI-guided muscle biopsies a total of 2 biopsies Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies
Detailed Description:
None
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?:
True
Is an FDA AA801 Violation?:
None