Ignite Creation Date:
2025-12-24 @ 4:25 PM
Ignite Modification Date:
2025-12-27 @ 7:32 PM
Study NCT ID:
NCT06159166
Status:
RECRUITING
Last Update Posted:
2025-04-23
First Post:
2023-11-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Sponsor:
Johns Hopkins University
Organization:
Study Overview
Official Title:
A Phase 1/2a Open-label, Multicenter Dose Finding Study to Evaluate the Safety and Anti-tumor Activity of Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Status:
RECRUITING
Status Verified Date:
2025-04
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:
Mirda
Brief Summary:
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF.
In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
Detailed Description:
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. The study will be conducted in two phases:
Phase 1 will test the safety of multiple dose regimens of mirdametinib, in order to identify a recommended phase 2 dose(s) for phase 2a. The phase 1 portion will enroll participants in dose regimens 1 to 3 concurrently. Enrollment of participants to dose regimen 4 will be conducted only if no dose limiting toxicities (DLT) occurred in any of the three previous regimens within at least, the first three cycles. The study will continue until one of the following stopping conditions is met.
* The RP2D(s) have been identified with sufficient accuracy. The dose regimen(s) selected as the RP2D have been fully enrolled with at least 3 cycles of treatment completed for all participants)
* All dose regimens are deemed to have unacceptable safety
Phase 2a of the study will test the efficacy and safety of a maximum of two RP2Ds in adults with NF1 and cNF.
This study will be considered complete once all participants have ceased study treatment due to death, disease progression, intolerance, withdrawal of consent from the study, physician discretion, or completed a maximum of 48 cycles of treatment across both phase 1 and phase 2a. Each treatment cycle is 28 days.
In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at Johns Hopkins University.
Phase 1 will test the safety of multiple dose regimens of mirdametinib, in order to identify a recommended phase 2 dose(s) for phase 2a. The phase 1 portion will enroll participants in dose regimens 1 to 3 concurrently. Enrollment of participants to dose regimen 4 will be conducted only if no dose limiting toxicities (DLT) occurred in any of the three previous regimens within at least, the first three cycles. The study will continue until one of the following stopping conditions is met.
* The RP2D(s) have been identified with sufficient accuracy. The dose regimen(s) selected as the RP2D have been fully enrolled with at least 3 cycles of treatment completed for all participants)
* All dose regimens are deemed to have unacceptable safety
Phase 2a of the study will test the efficacy and safety of a maximum of two RP2Ds in adults with NF1 and cNF.
This study will be considered complete once all participants have ceased study treatment due to death, disease progression, intolerance, withdrawal of consent from the study, physician discretion, or completed a maximum of 48 cycles of treatment across both phase 1 and phase 2a. Each treatment cycle is 28 days.
In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at Johns Hopkins University.
Study Oversight
Has Oversight DMC:
True
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| J23129 | OTHER | SKCCC | View |