Ignite Creation Date:
2025-12-25 @ 4:29 AM
Ignite Modification Date:
2026-03-01 @ 6:10 AM
Study NCT ID:
NCT02839720
Status:
COMPLETED
Last Update Posted:
2023-12-06
First Post:
2016-07-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma
Sponsor:
National Cancer Institute (NCI)
Organization:
Study Overview
Official Title:
Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF)
Status:
COMPLETED
Status Verified Date:
2023-12
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:
This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Detailed Description:
PRIMARY OBJECTIVE:
I. Determine if selumetinib sulfate (selumetinib) can result in shrinkage of cutaneous neurofibromas.
SECONDARY OBJECTIVE:
I. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s) excised prior treatment and on treatment with selumetinib for analysis of percent inhibition of phosphorylated ERK (pERK), and changes in phosphorylated AKT (pAKT).
EXPLORATORY OBJECTIVES:
I. Assess the effect of selumetinib on the development on new cutaneous neurofibromas while on treatment with selumetinib.
II. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s) excised prior treatment and on treatment with selumetinib for analysis of the tumor kinome.
III. Assess the effect of selumetinib skin related morbidity and pain using the Skindex, the Global Impression of Change Scale and Numeric Rating Scale, all of which are patient reported outcome measures.
IV. Quantify the development of new cutaneous neurofibromas on treatment with selumetinib.
V. Detailed pathologic analysis of cutaneous neurofibromas pretreatment and on treatment with selumetinib for changes in cell composition (including macrophage and mast cell infiltration).
VI. Investigate alterations that correlate with cutaneous neurofibroma (cNF) response to selumetinib treatment with pilot genomic, deoxyribonucleic acid (DNA) methylation, and transcriptomic studies.
OUTLINE:
Patients receive selumetinib sulfate orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients who experience a volume decrease in the target cutaneous neurofibromas may continue treatment for 12 additional cycles.
After completion of study treatment, patients are followed up every 4 months for 1 year.
I. Determine if selumetinib sulfate (selumetinib) can result in shrinkage of cutaneous neurofibromas.
SECONDARY OBJECTIVE:
I. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s) excised prior treatment and on treatment with selumetinib for analysis of percent inhibition of phosphorylated ERK (pERK), and changes in phosphorylated AKT (pAKT).
EXPLORATORY OBJECTIVES:
I. Assess the effect of selumetinib on the development on new cutaneous neurofibromas while on treatment with selumetinib.
II. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s) excised prior treatment and on treatment with selumetinib for analysis of the tumor kinome.
III. Assess the effect of selumetinib skin related morbidity and pain using the Skindex, the Global Impression of Change Scale and Numeric Rating Scale, all of which are patient reported outcome measures.
IV. Quantify the development of new cutaneous neurofibromas on treatment with selumetinib.
V. Detailed pathologic analysis of cutaneous neurofibromas pretreatment and on treatment with selumetinib for changes in cell composition (including macrophage and mast cell infiltration).
VI. Investigate alterations that correlate with cutaneous neurofibroma (cNF) response to selumetinib treatment with pilot genomic, deoxyribonucleic acid (DNA) methylation, and transcriptomic studies.
OUTLINE:
Patients receive selumetinib sulfate orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients who experience a volume decrease in the target cutaneous neurofibromas may continue treatment for 12 additional cycles.
After completion of study treatment, patients are followed up every 4 months for 1 year.
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 |
|---|---|---|---|---|
| NCI-2016-01087 | REGISTRY | CTRP (Clinical Trial Reporting Program) | View | |
| 9990 | OTHER | University of Alabama at Birmingham Cancer Center | View | |
| 9990 | OTHER | CTEP | View | |
| P30CA013148 | NIH | None | https://reporter.nih.gov/quic… | View |