Ignite Creation Date:
2024-05-06 @ 3:29 PM
Last Modification Date:
2024-10-26 @ 1:50 PM
Study NCT ID:
NCT04641442
Status:
RECRUITING
Last Update Posted:
2024-06-25
First Post:
2020-11-20
Brief Title:
Study to Evaluate the Efficacy Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases Including NLRC4-GOF XIAP Deficiency or CDC42 Mutations
Sponsor:
Novartis Pharmaceuticals
Organization:
Novartis
Study Overview
Official Title:
A Three-period Multicenter Study With a Randomized-withdrawal Double-blind Placebo-controlled Design to Evaluate the Clinical Efficacy Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases Including NLRC4-GOF XIAP Deficiency or CDC42 Mutations
Status:
RECRUITING
Status Verified Date:
2024-09
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:
MASter-1
Brief Summary:
This study is a Phase 2 trial designed to evaluate the clinical efficacy safety and tolerability of MAS825 in patients with NLRC4-GOF XIAP deficiency or CDC42 mutations
Detailed Description:
This is a three-period study with an open-label single-arm active treatment in Period 1 followed by a randomized-withdrawal double-blinded placebo-controlled design in Period 2 and an open label long-term safety follow-up in Period 3 The total study duration is approximately 3 - 4 years
Patients who enter Period 2 will be randomized to MAS825 or matching placebo in a 11 ratio
Cohort 1 patients will complete all periods of the study which will take approximately 4 years
Cohort 2 Patients who are receiving MAS825 in a Novartis Managed Access Program with a diagnosis of NLRC4-GOF XIAP deficiency or CDC42 mutation who meet criteria will be eligible to directly enter into Period 3 for open-label long-term safety follow-up Cohort 2 patients will be in the study for approximately 3 years
Patients who enter Period 2 will be randomized to MAS825 or matching placebo in a 11 ratio
Cohort 1 patients will complete all periods of the study which will take approximately 4 years
Cohort 2 Patients who are receiving MAS825 in a Novartis Managed Access Program with a diagnosis of NLRC4-GOF XIAP deficiency or CDC42 mutation who meet criteria will be eligible to directly enter into Period 3 for open-label long-term safety follow-up Cohort 2 patients will be in the study for approximately 3 years
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