34. Neurofibromatosis
120 clinical trials, 182 drugs (DrugBank: 72 drugs), 84 drug target genes, 194 drug target pathways
Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04590235 (ClinicalTrials.gov) | November 12, 2020 | 28/7/2020 | A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | Neurofibromatosis 1;Neurofibroma Plexiform | Drug: Selumetinib | AstraZeneca | Merck Sharp & Dohme Corp. | Not yet recruiting | 3 Years | 99 Years | All | 32 | Phase 1 | China |
| 2 | ChiCTR2000036770 | 2020-10-01 | 2020-08-25 | Establishment of prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsy | Establishment of prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsy | Plexiform Neurofibromatosis type 1 | Gold Standard:Clinical outcomes;Index test:Prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsy; | Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine | NULL | Pending | 2 | 65 | Both | Target condition:32;Difficult condition:0 | China | |
| 3 | JPRN-JapicCTI-205422 | 01/9/2020 | 20/08/2020 | A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN) | A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN) | Neurofibromatosis Type 1 and Inoperable and Symptomatic Plexiform Neurofibroma | Intervention name : Selumetinib INN of the intervention : - Dosage And administration of the intervention : Selumetinib 25 mg/m2 BID Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | AstraZeneca KK | NULL | recruiting | 3 | 18 | BOTH | 9 | Phase 1 | Japan, North America |
| 4 | NCT04495127 (ClinicalTrials.gov) | August 31, 2020 | 29/7/2020 | Selumetinib Paediatric NF1 Japan Study | A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN) | Neurofibromatosis Type 1 | Drug: Selumetinib | AstraZeneca | NULL | Recruiting | 3 Years | 18 Years | All | 12 | Phase 1 | Japan |
| 5 | NCT04166409 (ClinicalTrials.gov) | January 3, 2020 | 15/11/2019 | A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma | A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1) | Astrocytoma;Low Grade Glioma | Drug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib Sulfate;Drug: Vincristine Sulfate | National Cancer Institute (NCI) | NULL | Recruiting | 2 Years | 21 Years | All | 200 | Phase 3 | United States;Canada |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03871257 (ClinicalTrials.gov) | October 4, 2019 | 11/3/2019 | A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma | A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) | Low Grade Glioma;Neurofibromatosis Type 1;Visual Pathway Glioma | Drug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib Sulfate;Drug: Vincristine Sulfate | National Cancer Institute (NCI) | NULL | Recruiting | 2 Years | 21 Years | All | 290 | Phase 3 | United States;Canada |
| 7 | NCT03433183 (ClinicalTrials.gov) | October 2, 2019 | 29/1/2018 | SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors | SARC031: A Phase 2 Trial of the MEK Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Combination With the mTOR Inhibitor Sirolimus for Patients With Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors | Malignant Peripheral Nerve Sheath Tumors;Neurofibromatosis 1 | Drug: Selumetinib;Drug: Sirolimus | Sarcoma Alliance for Research through Collaboration | United States Department of Defense;AstraZeneca | Recruiting | 12 Years | N/A | All | 21 | Phase 2 | United States |
| 8 | NCT03326388 (ClinicalTrials.gov) | September 26, 2019 | 8/9/2017 | Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours | A Paediatric Phase I/II Study Of Intermittent Dosing Of The Mek-1 Inhibitor Selumetinib In Children With Neurofibromatosis Type-1 And Inoperable Plexiform Neurofibroma And/Or Progressive Optic Pathway Glioma | Neurofibromatosis Type 1;Plexiform Neurofibroma;Optic Nerve Glioma | Drug: Selumetinib | Great Ormond Street Hospital for Children NHS Foundation Trust | AstraZeneca | Recruiting | 3 Years | 18 Years | All | 30 | Phase 1;Phase 2 | United Kingdom |
| 9 | EUCTR2017-002635-41-GB (EUCTR) | 26/09/2018 | 19/06/2019 | A PAEDIATRIC PHASE I/II STUDY OF INTERMITTENT DOSING OF THE MEK-1 INHIBITOR SELUMETINIB IN CHILDREN WITH NEUROFIBROMATOSIS TYPE-1 AND INOPERABLE PLEXIFORM NEUROFIBROMA AND/OR PROGRESSIVE OPTIC PATHWAY GLIOMA | A paediatric phase I/II study of intermittent dosing of the MEK-1 inhibitor selumetinib in children with neurofibromatosis type-1 and inoperable plexiform neurofibroma and/or progressive optic pathway glaiom - Intermittent Selumetinib dosing Phase I/II study Childhood NF1 tumours | Neurofibromatosis type 1 associated plexiform neurofibromas Neurofibromatosis type 1 associated progressive or relapsed optic pathway glioma MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10065866;Term: Plexiform neurofibroma;System Organ Class: 100000004864 MedDRA version: 20.0;Classification code 10030935;Term: Optic nerve glioma;System Organ Class: 100000004864 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Great Ormond Street Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 38 | Phase 1;Phase 2 | United Kingdom | |||
| 10 | NCT03649165 (ClinicalTrials.gov) | September 5, 2018 | 24/8/2018 | A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants | A Phase I, Open-label, Single-center Relative Bioavailability and Food Effect Randomized Crossover Study of New Granule and Capsule Formulations of Selumetinib (AZD6244) in Healthy Male Subjects | Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs);Healthy Participants | Drug: Treatment A;Drug: Treatment B;Drug: Treatment C;Drug: Treatment D;Drug: Acetaminophen | AstraZeneca | NULL | Completed | 18 Years | 45 Years | Male | 24 | Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03259633 (ClinicalTrials.gov) | October 30, 2017 | 21/8/2017 | An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 | An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN) | NF type1 With Inoperable Plexiform Neurofibromas | Drug: Selumetinib | AstraZeneca | NULL | Approved for marketing | 2 Years | 130 Years | All | United States | ||
| 12 | NCT03095248 (ClinicalTrials.gov) | May 8, 2017 | 8/3/2017 | Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors | Phase 2 Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors | Neurofibromatosis 2;Vestibular Schwannoma;Meningioma;Ependymoma;Glioma | Drug: Selumetinib | Children's Hospital Medical Center, Cincinnati | AstraZeneca | Recruiting | 3 Years | 45 Years | All | 34 | Phase 2 | United States |
| 13 | NCT03105258 (ClinicalTrials.gov) | May 1, 2017 | 23/3/2017 | Selumetinib Pilot Study for Cutaneous Neurofibromas | Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (cNF) | Neurofibromatosis Type 1;Cutaneous Neurofibroma | Drug: Selumetinib | University of Alabama at Birmingham | National Cancer Institute (NCI) | Not yet recruiting | 18 Years | N/A | All | 24 | Phase 2 | United States |
| 14 | NCT02839720 (ClinicalTrials.gov) | April 11, 2017 | 20/7/2016 | Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma | Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF) | Cutaneous Neurofibroma;Neurofibromatosis Type 1;Optic Nerve Glioma | Other: Laboratory Biomarker Analysis;Drug: Selumetinib;Drug: Selumetinib Sulfate | National Cancer Institute (NCI) | NULL | Recruiting | 18 Years | N/A | All | 24 | Phase 2 | United States |
| 15 | NCT03109301 (ClinicalTrials.gov) | April 7, 2017 | 11/4/2017 | Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST) | A Phase II Trial of the Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Patients With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST) | Neoplasms, Nerve Tissue;Neurofibromatosis 1;Heredodegenerative Disorders, Nervous System;Peripheral Nervous System Diseases | Drug: Selumetinib (AZD6244 hyd sulfate) 50mg/dose;Drug: Selumetinib (AZD6244 hyd sulfate) 25mg/m2 | National Cancer Institute (NCI) | NULL | Withdrawn | 3 Years | 99 Years | All | 0 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT02407405 (ClinicalTrials.gov) | January 7, 2016 | 2/4/2015 | MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas | Phase II Trial of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas | Neurofibromatosis 1 (NF1);Plexiform Neurofibromas (PN) | Drug: Selumetinib | National Cancer Institute (NCI) | NULL | Recruiting | 18 Years | N/A | All | 60 | Phase 2 | United States |
| 17 | NCT02644512 (ClinicalTrials.gov) | December 2015 | 31/12/2015 | MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas | Phase II Trial of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas | Neurofibromatosis 1;Plexiform Neurofibromas | Drug: Selumetinib | National Cancer Institute (NCI) | NULL | Recruiting | 18 Years | 99 Years | Both | 50 | Phase 2 | United States |
| 18 | NCT01362803 (ClinicalTrials.gov) | September 21, 2011 | 27/5/2011 | AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors | A Phase I/II Study of the Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor Selumetinib (AZD6244; HYD Sulfate) in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | Neurofibromatosis 1;Neurofibromatosis Type 1;NF 1;Neurofibroma, Plexiform | Drug: AZD6244 | National Cancer Institute (NCI) | NULL | Active, not recruiting | 2 Years | 18 Years | All | 99 | Phase 1;Phase 2 | United States |
| 19 | NCT01089101 (ClinicalTrials.gov) | April 19, 2010 | 17/3/2010 | Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma | A Phase 1 and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma | Low Grade Glioma;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Neurofibromatosis Type 1;Recurrent Visual Pathway Glioma;Refractory Neurofibromatosis Type 1;Refractory Visual Pathway Glioma | Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Selumetinib | National Cancer Institute (NCI) | NULL | Recruiting | 3 Years | 21 Years | All | 220 | Phase 1;Phase 2 | United States |