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 and Erlotinib Hydrochloride in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer

Phase 2

Completed

Conditions: Adenocarcinoma of the Pancreas
Recurrent Pancreatic Cancer
Stage III Pancreatic Cancer
Stage IV Pancreatic Cancer

Interventions: Drug: erlotinib hydrochloride
Drug: selumetinib
Other: laboratory biomarker analysis

First Posted Date: 2010-10-18

Last Posted Date: 2020-07-31

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 46

Registration Number: NCT01222689

Locations: 🇺🇸
UCSF-Mount Zion, San Francisco, California, United States
🇺🇸
Ohio State University Medical Center, Columbus, Ohio, United States

Selumetinib and Cetuximab in Treating Patients With Refractory Solid Tumors

Phase 1

Completed

Conditions: Recurrent Colon Cancer
Stage IVA Colon Cancer
Stage IVB Colon Cancer
Recurrent Rectal Cancer
Stage IVA Rectal Cancer
Stage IVB Rectal Cancer
Unspecified Adult Solid Tumor, Protocol Specific

Interventions: Drug: selumetinib
Biological: cetuximab
Other: laboratory biomarker analysis
Other: pharmacological study

First Posted Date: 2010-10-08

Last Posted Date: 2014-05-29

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 33

Registration Number: NCT01217450

Locations: 🇺🇸
National Cancer Institute Medicine Branch, Bethesda, Maryland, United States
🇺🇸
Wisconsin Clinical Cancer Center, Milwaukee, Wisconsin, United States
🇺🇸
University of Wisconsin Hospital and Clinics, Madison, Wisconsin, United States

Selumetinib With or Without Temsirolimus in Treating Patients With Metastatic, Recurrent, or Locally Advanced Soft Tissue Sarcoma That Cannot Be Removed By Surgery

Phase 2

Completed

Conditions: Stage III Adult Soft Tissue Sarcoma
Recurrent Adult Soft Tissue Sarcoma
Stage IV Adult Soft Tissue Sarcoma

Interventions: Other: Laboratory Biomarker Analysis
Drug: Selumetinib
Drug: Temsirolimus

First Posted Date: 2010-09-21

Last Posted Date: 2015-10-05

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 70

Registration Number: NCT01206140

Locations: 🇺🇸
City of Hope Comprehensive Cancer Center, Duarte, California, United States
🇺🇸
Tower Cancer Research Foundation, Beverly Hills, California, United States
🇺🇸
USC / Norris Comprehensive Cancer Center, Los Angeles, California, United States

and more 5 locations

Temsirolimus/AZD 6244 for Treatment-naive With BRAF Mutant Unresectable Stage IV

Phase 2

Terminated

Conditions: Mucosal Melanoma
Recurrent Melanoma
Stage IV Melanoma

Interventions: Drug: temsirolimus
Drug: selumetinib
Other: laboratory biomarker analysis

First Posted Date: 2010-07-20

Last Posted Date: 2014-05-15

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 4

Registration Number: NCT01166126

Locations: 🇺🇸
H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, United States

Fulvestrant With or Without AZD6244 in Treating Patients With Advanced Breast Cancer That Progressed After Aromatase Inhibitor Therapy

Phase 2

Completed

Conditions: Breast Cancer

Interventions: Drug: fulvestrant
Drug: selumetinib

First Posted Date: 2010-07-12

Last Posted Date: 2019-05-15

Lead Sponsor: Swiss Group for Clinical Cancer Research

Target Recruit Count: 46

Registration Number: NCT01160718

Locations: 🇨🇭
Kantonsspital Aarau, Aarau, Switzerland
🇨🇭
Kantonsspital Baden, Baden, Switzerland
🇨🇭
City Hospital Triemli, Zurich, Switzerland

and more 14 locations

Temozolomide or Selumetinib in Treating Patients With Metastatic Melanoma of the Eye

Phase 2

Completed

Conditions: Iris Melanoma
Recurrent Uveal Melanoma
Stage IV Uveal Melanoma
Medium/Large Size Posterior Uveal Melanoma
Ocular Melanoma With Extraocular Extension
Small Size Posterior Uveal Melanoma

Interventions: Drug: Dacarbazine
Other: Laboratory Biomarker Analysis
Other: Quality-of-Life Assessment
Drug: Selumetinib
Drug: Temozolomide

First Posted Date: 2010-06-14

Last Posted Date: 2017-07-26

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 120

Registration Number: NCT01143402

Locations: 🇨🇦
University Health Network-Princess Margaret Hospital, Toronto, Ontario, Canada
🇺🇸
Mount Sinai Medical Center, Miami Beach, Florida, United States
🇺🇸
Emory University/Winship Cancer Institute, Atlanta, Georgia, United States

and more 30 locations

A Phase I Study of AZD6244 in Combination With Capecitabine and Radiotherapy in Locally Advanced Adenocarcinoma of the Rectum

Phase 1

Terminated

Conditions: Non-Metastatic Adenocarcinoma of the Rectum

Interventions: Procedure: Radiation Therapy
Drug: Capecitabine
Drug: AZD6244

First Posted Date: 2010-06-02

Last Posted Date: 2021-12-01

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 1

Registration Number: NCT01134601

Locations: 🇺🇸
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

Phase 1

Active, not recruiting

Conditions: Recurrent Childhood Pilocytic Astrocytoma
Refractory Neurofibromatosis Type 1
Refractory Visual Pathway Glioma
Low Grade Glioma
Recurrent Neurofibromatosis Type 1
Recurrent Visual Pathway Glioma

Interventions: Procedure: Biospecimen Collection
Drug: Selumetinib

First Posted Date: 2010-03-18

Last Posted Date: 2024-10-23

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 220

Registration Number: NCT01089101

Locations: 🇺🇸
Children's Healthcare of Atlanta - Arthur M Blank Hospital, Atlanta, Georgia, United States
🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸
Lucile Packard Children's Hospital Stanford University, Palo Alto, California, United States

and more 14 locations

AZD6244 (Selumetinib) in Treating Patients With Multiple Myeloma

Phase 2

Completed

Conditions: Refractory Plasma Cell Myeloma
Recurrent Plasma Cell Myeloma

Interventions: Other: Laboratory Biomarker Analysis
Drug: Selumetinib

First Posted Date: 2010-03-11

Last Posted Date: 2015-08-19

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 37

Registration Number: NCT01085214

Locations: 🇺🇸
Billings Clinic Cancer Center, Billings, Montana, United States
🇺🇸
University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States
🇺🇸
Virginia Commonwealth University/Massey Cancer Center, Richmond, Virginia, United States

and more 6 locations

Selumetinib and Cixutumumab in Treating Patients With Advanced Solid Malignancies

Phase 1

Completed

Conditions: Adult Solid Neoplasm

Interventions: Biological: Cixutumumab
Other: Laboratory Biomarker Analysis
Other: Pharmacological Study
Drug: Selumetinib

First Posted Date: 2010-02-03

Last Posted Date: 2016-01-25

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 30

Registration Number: NCT01061749

Locations: 🇺🇸
Johns Hopkins University/Sidney Kimmel Cancer Center, Baltimore, Maryland, United States

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