Ignite Creation Date:
2024-10-26 @ 3:39 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06582745
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-30
Brief Title:
Targeted Approach to Langerhans Cell Histiocytosis LCH Using MEK Inhibitor Trametinib
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Targeted Approach to Langerhans Cell Histiocytosis LCH Using MEK Inhibitor Trametinib
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this Phase II clinical trial is to establish the safety and effectiveness of trametinib a targeted therapy for the treatment of newly or recently diagnosed Langerhans Cell Histiocytosis LCH among pediatric patients
Detailed Description:
Langerhans cell histiocytosis LCH is a rare histiocytic disease derived from the mononuclear phagocytic system affecting between 2 and 10 cases per one million children under 15 years Controversy exists as to whether it is a true malignancy or a cancer-like disease given that the BRAFV600E mutation frequently found in LCH has been found in several cancers as well as in benign nevi Regardless among histiocytic disorders LCH is well-known to result from clonal proliferation of immature cells that can affect a single organ single system LCH which may be unifocal or multifocal or LCH may involve multiple organs which may be limited or widespread Notably involvement of specific organs such as the liver spleen and bone marrow is typically considered high risk A biopsy is a critical element for diagnosis as histiocytes with surface expression of CD207 langerin and CD1a are a defining characteristic of LCH In addition to a biopsy of either skin lesion lymph node or tumor standard imaging such as CT MRI and PET will be used to assess the extent of disease
This trial is designed to evaluate treatment of newly diagnosed or relapsed LCH patients with targeted therapy trametinib The investigators hypothesize that this will help establish a new treatment for these patients who have historically been treated with cytotoxic chemotherapy that can potentially be associated with serious adverse effects as well as relapse which have typically been noted within two years therefore justifying the rationale to treat for minimum of two years This clinical trial will provide an opportunity to assess for adverse events and toxicities associated with trametinib for the treatment of LCH among pediatric patients Additionally the investigators will critically analyze the effectiveness of genomic cancer testing through the use of liquid tumor and tumor-match next-generation sequencing NGS in patients with an LCH diagnosis
This trial is designed to evaluate treatment of newly diagnosed or relapsed LCH patients with targeted therapy trametinib The investigators hypothesize that this will help establish a new treatment for these patients who have historically been treated with cytotoxic chemotherapy that can potentially be associated with serious adverse effects as well as relapse which have typically been noted within two years therefore justifying the rationale to treat for minimum of two years This clinical trial will provide an opportunity to assess for adverse events and toxicities associated with trametinib for the treatment of LCH among pediatric patients Additionally the investigators will critically analyze the effectiveness of genomic cancer testing through the use of liquid tumor and tumor-match next-generation sequencing NGS in patients with an LCH diagnosis
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None