Selumetinib

Generic Name: Selumetinib

Brand Names: Koselugo

Drug Type: Small Molecule

Chemical Formula: C_₁₇H_₁₅BrClFN_₄O_₃

CAS Number: 606143-52-6

Unique Ingredient Identifier: 6UH91I579U

Background: Activation of the Raf-MEK-ERK signaling pathway is known to be implemented in several types of malignancies, thus, mitogen-activated protein kinase kinase (MEK) inhibitors such as selumetinib are important tools that can target the problematic overactivity of this pathway.

Results from clinical trials investigating earlier developed MEK inhibitors were underwhelming. However, selumetinib demonstrated impressive efficacy and tolerability in Phase I trials, leading to its continued investigation for the treatment of various types of tumors in Phase II trials.

Currently, the novel MEK 1 / 2 inhibitor, selumetinib, is approved solely for the treatment of Neurofibromatosis type 1 (NF-1) in a limited age group. NF-1 is considered rare with an estimated incidence of 1/3000 individuals. It is a genetic, autosomal dominant condition resulting from mutations of the NF1 gene, which can lead to various complications including the development of multiple tumors in the nervous system. Some patients with this disorder develop plexiform neurofibromas (PN); however, this is considered to be relatively uncommon compared to other variants of NF-1. Luckily, the use of selumetinib in patients with NF-1 has shown efficacy in shrinking associated tumors and is linked to other positive clinical outcomes.

Indication: Although selumetinib has been investigated for the treatment of several types of cancer, it is currently only indicated for the treatment of neurofibromatosis type 1 (NF1) in patients ≥2 years who have symptomatic, inoperable plexiform neurofibromas (PN).

Associated Conditions: Neurofibromatosis, type 1 (von Recklinghausen's disease)

Associated Therapies: -

Clinical Trials

Selumetinib in Treating Patients With Recurrent or Persistent Endometrial Cancer

Phase 2

Completed

Conditions: Endometrial Adenosquamous Carcinoma
Endometrial Adenocarcinoma
Recurrent Uterine Corpus Carcinoma
Endometrial Clear Cell Adenocarcinoma

Interventions: Other: Diagnostic Laboratory Biomarker Analysis
Drug: Selumetinib

First Posted Date: 2009-11-11

Last Posted Date: 2019-07-23

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 54

Registration Number: NCT01011933

Locations: 🇺🇸
University of Chicago Comprehensive Cancer Center, Chicago, Illinois, United States
🇺🇸
Smilow Cancer Hospital Care Center at Saint Francis, Hartford, Connecticut, United States
🇺🇸
Rush University Medical Center, Chicago, Illinois, United States

and more 69 locations

Selumetinib in Cancers With BRAF Mutations

Phase 2

Completed

Conditions: Adult Solid Neoplasm

Interventions: Drug: Selumetinib

First Posted Date: 2009-04-27

Last Posted Date: 2016-01-15

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 28

Registration Number: NCT00888134

Locations: 🇺🇸
Massachusetts General Hospital Cancer Center, Boston, Massachusetts, United States
🇺🇸
Massachusetts General Hospital, Charlestown, Massachusetts, United States
🇺🇸
Memorial Sloan-Kettering Cancer Center, New York, New York, United States

and more 2 locations

MEK Inhibitor AZD6244 in Treating Patients With Stage III or Stage IV Melanoma

Phase 2

Completed

Conditions: Stage III Skin Melanoma
Recurrent Melanoma
Stage IV Skin Melanoma

Interventions: Other: Laboratory Biomarker Analysis
Drug: Selumetinib

First Posted Date: 2009-03-20

Last Posted Date: 2015-08-11

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 167

Registration Number: NCT00866177

Locations: 🇺🇸
Memorial Sloan-Kettering Cancer Center, New York, New York, United States

Selumetinib in Treating Patients With Locally Advanced or Metastatic Liver Cancer

Phase 2

Completed

Conditions: Adult Primary Hepatocellular Carcinoma
Advanced Adult Primary Liver Cancer
Localized Unresectable Adult Primary Liver Cancer
Recurrent Adult Primary Liver Cancer

Interventions: Drug: selumetinib
Other: pharmacological study

First Posted Date: 2008-01-30

Last Posted Date: 2014-05-28

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 19

Registration Number: NCT00604721

Locations: 🇺🇸
Emory University, Atlanta, Georgia, United States
🇺🇸
Arthur G. James Cancer Hospital and Solove Research Institute at Ohio State University Medical Center, Columbus, Ohio, United States
🇺🇸
H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, United States

and more 3 locations

Selumetinib in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia

Phase 2

Completed

Conditions: Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
Adult Acute Promyelocytic Leukemia (M3)
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms
Recurrent Adult Acute Myeloid Leukemia
Secondary Acute Myeloid Leukemia

Interventions: Drug: selumetinib

First Posted Date: 2008-01-09

Last Posted Date: 2015-08-05

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 47

Registration Number: NCT00588809

Locations: 🇺🇸
University of Chicago Comprehensive Cancer Center, Chicago, Illinois, United States

Selumetinib in Treating Patients With Papillary Thyroid Cancer That Did Not Respond to Radioactive Iodine

Phase 2

Completed

Conditions: Stage I Thyroid Gland Papillary Carcinoma
Stage III Thyroid Gland Papillary Carcinoma
Stage IV Thyroid Gland Papillary Carcinoma
Stage II Thyroid Gland Papillary Carcinoma
Recurrent Thyroid Gland Carcinoma

Interventions: Other: Laboratory Biomarker Analysis
Drug: Selumetinib

First Posted Date: 2007-11-19

Last Posted Date: 2017-01-30

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 39

Registration Number: NCT00559949

Locations: 🇺🇸
Moffitt Cancer Center, Tampa, Florida, United States
🇺🇸
University of Chicago Comprehensive Cancer Center, Chicago, Illinois, United States
🇺🇸
UNC Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina, United States

and more 3 locations

Selumetinib in Treating Patients With Biliary Cancer That Cannot Be Removed By Surgery

Phase 2

Completed

Conditions: Recurrent Extrahepatic Bile Duct Cancer
Liver and Intrahepatic Biliary Tract Cancer
Unresectable Extrahepatic Bile Duct Cancer

Interventions: Drug: selumetinib
Other: laboratory biomarker analysis

First Posted Date: 2007-11-04

Last Posted Date: 2016-04-22

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 29

Registration Number: NCT00553332

Locations: 🇺🇸
University of North Carolina, Chapel Hill, North Carolina, United States
🇺🇸
Vanderbilt University, Nashville, Tennessee, United States
🇺🇸
Ohio State University Medical Center, Columbus, Ohio, United States

and more 4 locations

Selumetinib Sulfate in Treating Woman With Recurrent Low-Grade Ovarian Cancer or Peritoneum Cancer

Phase 2

Completed

Conditions: Primary Peritoneal Carcinoma
Micropapillary Serous Carcinoma
Borderline Ovarian Serous Tumor
Low Grade Ovarian Serous Adenocarcinoma
Primary Peritoneal Low Grade Serous Adenocarcinoma
Recurrent Borderline Ovarian Surface Epithelial-Stromal Tumor

Interventions: Other: Laboratory Biomarker Analysis
Other: Pharmacological Study
Drug: Selumetinib
Drug: Selumetinib Sulfate

First Posted Date: 2007-10-30

Last Posted Date: 2020-12-03

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 52

Registration Number: NCT00551070

Locations: 🇺🇸
Maine Medical Center-Bramhall Campus, Portland, Maine, United States
🇺🇸
Brigham and Women's Hospital, Boston, Massachusetts, United States
🇺🇸
Massachusetts General Hospital Cancer Center, Boston, Massachusetts, United States

and more 46 locations

Drug Development Updates

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