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

International Trial of Selumetinib in Combination With Dexamethasone for the Treatment of Acute Lymphoblastic Leukaemia

Phase 1

Terminated

Conditions: Acute Lymphoblastic Leukemia Recurrent
Acute Lymphoblastic Leukemia, in Relapse
Acute Lymphoblastic Leukemia
Acute Lymphoblastic Leukemia, Adult
Acute Lymphoblastic Leukemia, Pediatric

Interventions: Drug: Selumetinib
Drug: Dexamethasone

First Posted Date: 2018-10-15

Last Posted Date: 2023-05-11

Lead Sponsor: University of Birmingham

Target Recruit Count: 12

Registration Number: NCT03705507

Locations: 🇬🇧
King's College Hospital, London, United Kingdom
🇳🇱
Prinses Maxima Centrum Voor Kinderoncologie, Utrecht, Netherlands
🇬🇧
Birmingham Children's Hospital, Birmingham, United Kingdom

and more 14 locations

Durvalumab, Tremelimumab, and Selumetinib in Treating Participants With Recurrent or Stage IV Non-small Cell Lung Cancer

Phase 1

Active, not recruiting

Conditions: Recurrent Lung Non-Small Cell Carcinoma
Stage IV Lung Cancer AJCC v8
Stage IVB Lung Cancer AJCC v8
Stage IVA Lung Cancer AJCC v8

Interventions: Biological: Durvalumab
Drug: Selumetinib
Biological: Tremelimumab

First Posted Date: 2018-07-10

Last Posted Date: 2024-12-05

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 40

Registration Number: NCT03581487

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

Phase 2

Completed

Conditions: Malignant Peripheral Nerve Sheath Tumors
Neurofibromatosis 1

Interventions: Drug: Selumetinib
Drug: Sirolimus

First Posted Date: 2018-02-14

Last Posted Date: 2023-12-26

Lead Sponsor: Sarcoma Alliance for Research through Collaboration

Target Recruit Count: 21

Registration Number: NCT03433183

Locations: 🇺🇸
Children's National Medical Center, Washington, District of Columbia, United States
🇺🇸
National Cancer Institute, Bethesda, Maryland, United States
🇺🇸
Johns Hopkins University, Baltimore, Maryland, United States

and more 2 locations

A Phase 2 Study of Osimertinib in Combination With Selumetinib in EGFR Inhibitor naïve Advanced EGFR Mutant Lung Cancer

Phase 2

Active, not recruiting

Conditions: Non-small Cell Lung Cancer

Interventions: Drug: Osimertinib
Drug: Selumetinib

First Posted Date: 2018-01-05

Last Posted Date: 2025-01-01

Lead Sponsor: Dana-Farber Cancer Institute

Target Recruit Count: 25

Registration Number: NCT03392246

Locations: 🇺🇸
Dana Farber Cancer Institute, Boston, Massachusetts, United States
🇺🇸
Duke University, Durham, North Carolina, United States
🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States

and more 1 locations

Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours

Phase 1

Completed

Conditions: Neurofibromatosis Type 1
Plexiform Neurofibroma
Optic Nerve Glioma

Interventions: Drug: Selumetinib

First Posted Date: 2017-10-31

Last Posted Date: 2024-04-04

Lead Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Target Recruit Count: 14

Registration Number: NCT03326388

Locations: 🇬🇧
Great Ormond Street Hospital NHS Foundatin Trust, London, United Kingdom
🇬🇧
Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

Selumetinib and Azacitidine in High Risk Chronic Blood Cancers

Phase 1

Recruiting

Conditions: Chronic Myeloid Leukemia
Myelofibroses

Interventions: Drug: Azacitidine
Drug: Selumetinib

First Posted Date: 2017-10-31

Last Posted Date: 2024-01-29

Lead Sponsor: University of Chicago

Target Recruit Count: 18

Registration Number: NCT03326310

Locations: 🇺🇸
The University of Chicago, Chicago, Illinois, United States

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

Conditions: NF type1 With Inoperable Plexiform Neurofibromas

First Posted Date: 2017-08-24

Last Posted Date: 2020-05-01

Lead Sponsor: AstraZeneca

Registration Number: NCT03259633

Locations: 🇺🇸
Research Site, New Orleans, Louisiana, United States

Selumetinib Sulfate in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)

Phase 2

Active, not recruiting

Conditions: Advanced Malignant Solid Neoplasm
Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma
Refractory Primary Central Nervous System Neoplasm
Recurrent Childhood Central Nervous System Neoplasm
Recurrent Neuroblastoma
Recurrent Childhood Non-Hodgkin Lymphoma
Recurrent Malignant Solid Neoplasm
Refractory Neuroblastoma
Refractory Malignant Solid Neoplasm

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

First Posted Date: 2017-07-11

Last Posted Date: 2024-10-23

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 21

Registration Number: NCT03213691

Locations: 🇺🇸
Children's Healthcare of Atlanta - Arthur M Blank Hospital, Atlanta, Georgia, United States
🇺🇸
Children's Hospital of Alabama, Birmingham, Alabama, United States
🇺🇸
Banner Children's at Desert, Mesa, Arizona, United States

and more 108 locations

Selumetinib and Olaparib in Solid Tumors

Phase 1

Active, not recruiting

Conditions: Malignant Neoplasms of Digestive Organs
Malignant Neoplasms of Male Genital Organs
Malignant Neoplasms of Thyroid and Other Endocrine Glands
Malignant Neoplasm of Breast
Malignant Neoplasms of Female Genital Organs

Interventions: Drug: Selumetinib
Drug: Olaparib

First Posted Date: 2017-05-22

Last Posted Date: 2024-10-16

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 90

Registration Number: NCT03162627

Locations: 🇺🇸
University of Texas MD Anderson Cancer Center, Houston, Texas, United States

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

Phase 2

Active, not recruiting

Conditions: Advanced Malignant Solid Neoplasm
Juvenile Xanthogranuloma
Langerhans Cell Histiocytosis
Malignant Glioma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Ewing Sarcoma
Recurrent Osteosarcoma
Recurrent Primary Central Nervous System Neoplasm
Refractory Hepatoblastoma
Refractory Medulloblastoma

Interventions: Procedure: Biopsy
Procedure: Biospecimen Collection
Other: Laboratory Biomarker Analysis
Procedure: Bone Marrow Aspiration and Biopsy
Procedure: Mutation Carrier Screening
Drug: Larotrectinib Sulfate
Procedure: Bone Scan
Other: Pharmacological Study
Procedure: Computed Tomography
Drug: Olaparib
Drug: Palbociclib
Drug: Tipifarnib
Drug: Erdafitinib
Drug: Tazemetostat
Drug: Samotolisib
Drug: Selumetinib Sulfate
Drug: Ensartinib
Drug: Vemurafenib
Drug: Ulixertinib
Procedure: Magnetic Resonance Imaging
Drug: Selpercatinib
Procedure: Radionuclide Imaging
Procedure: Positron Emission Tomography
Procedure: X-Ray Imaging

First Posted Date: 2017-05-16

Last Posted Date: 2025-01-08

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 2316

Registration Number: NCT03155620

Locations: 🇺🇸
Children's Hospital of Alabama, Birmingham, Alabama, United States
🇺🇸
Providence Alaska Medical Center, Anchorage, Alaska, United States
🇺🇸
Banner Children's at Desert, Mesa, Arizona, United States

and more 169 locations

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