34. 神経線維腫症 Neurofibromatosis Clinical trials / Disease details
臨床試験数 : 133 / 薬物数 : 186 - (DrugBank : 67) / 標的遺伝子数 : 79 - 標的パスウェイ数 : 190
| 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 | EUCTR2020-005608-20-ES (EUCTR) | 16/02/2022 | 19/10/2021 | Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) | A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) - SPRINKLE | Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Not possible to specify | Product Name: Selumetinib granules in sprinkle capsules for opening 5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib granules in sprinkle capsules for opening 7.5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Trade Name: Koselugo 10mg Product Name: Selumetinib capsules 10mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Trade Name: Koselugo 25mg Product Name: Selumetinib capsules 25mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 38 | Phase 1;Phase 2 | United States;Spain;Russian Federation;Netherlands;Germany;Italy | ||
| 2 | EUCTR2020-005607-39-PL (EUCTR) | 25/01/2022 | 14/09/2021 | Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas | A Phase III, Multicentre, International Study with a Parallel, Randomised, Double blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiforn Neurofibromas (KOMET) - KOMET | Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Selumetinib 10mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib 25mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 146 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Brazil;Australia;Netherlands;Germany;China;Japan | ||
| 3 | EUCTR2020-005608-20-NL (EUCTR) | 24/01/2022 | 21/10/2021 | Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) | A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) - SPRINKLE | Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Not possible to specify | Product Name: Selumetinib granules in sprinkle capsules for opening 5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib granules in sprinkle capsules for opening 7.5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Trade Name: Koselugo 10mg Product Name: Selumetinib capsules 10mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Trade Name: Koselugo 25mg Product Name: Selumetinib capsules 25mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 38 | Phase 1;Phase 2 | United States;Spain;Russian Federation;Germany;Netherlands;Italy | ||
| 4 | EUCTR2020-005608-20-DE (EUCTR) | 24/11/2021 | 09/09/2021 | Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) | A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged = 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) - SPRINKLE | Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Not possible to specify | Product Name: Selumetinib granules in sprinkle capsules for opening 5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib granules in sprinkle capsules for opening 7.5 mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib capsules 10mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib capsules 25mg Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Spain;Russian Federation;Netherlands;Germany;Italy;Japan | ||
| 5 | EUCTR2020-005607-39-ES (EUCTR) | 05/10/2021 | 23/06/2021 | Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas | A Phase III, Multicentre, International Study with a Parallel, Randomised, Double blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiforn Neurofibromas (KOMET) - KOMET | Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Selumetinib 10mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib 25mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 146 | Phase 3 | United States;Spain;Russian Federation;Italy;France;Canada;Brazil;Poland;Australia;Germany;Netherlands;China;Japan | ||
| 6 | EUCTR2020-005607-39-IT (EUCTR) | 08/09/2021 | 18/10/2021 | Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas | A Phase III, Multicentre, International Study with a Parallel, Randomised, Double blind, Placebo controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiforn Neurofibromas (KOMET). - KOMET | Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Selumetinib 25mg capsule Product Code: [AZD6244] INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Product Name: Selumetinib 10mg capsule Product Code: [AZD6244] INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | ASTRAZENECA AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 146 | Phase 3 | United States;Spain;Russian Federation;Italy;France;Canada;Brazil;Poland;Australia;Germany;Netherlands;China;Japan | ||
| 7 | EUCTR2020-005648-52-PL (EUCTR) | 31/05/2021 | 26/04/2021 | Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Toxicity of Selumetinib in Adolescent Children with Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) | A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Toxicity and Pharmacokinetics of Selumetinib after Multiple Doses in Adolescent Children with Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) - Selumetinib Gastrointestinal Toxicity Study (Selumetinib GI Tox Study) | Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Not possible to specify | Trade Name: Koselugo 10mg Product Name: Selumetinib 10mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate Trade Name: Koselugo 25mg Product Name: Selumetinib 25mg capsule Product Code: AZD6244 INN or Proposed INN: Selumetinib Other descriptive name: selumetinib hyd-sulfate | AstraZeneca AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1 | United States;Spain;Poland;Russian Federation | ||
| 8 | 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) | Low Grade Astrocytoma;Low Grade Glioma;Metastatic Low Grade Astrocytoma;Metastatic Low Grade Glioma | Procedure: Biospecimen Collection;Drug: Carboplatin;Procedure: Magnetic Resonance Imaging;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 | 220 | Phase 3 | United States;Canada;Puerto Rico |
| 9 | 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;Procedure: Magnetic Resonance Imaging;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;Puerto Rico |
| 10 | 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 | Active, not recruiting | 12 Years | N/A | All | 21 | Phase 2 | United States |
| 11 | 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] | Product Name: Selumetinib INN or Proposed INN: selumetinib sulfate Other descriptive name: Selumetinib Hyd-sulphate | Great Ormond Street Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 38 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 12 | 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 |
| 13 | 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 Sulfate | National Cancer Institute (NCI) | NULL | Active, not recruiting | 18 Years | N/A | All | 24 | Phase 2 | United States |
| 14 | 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 |
| 15 | 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 | Active, not recruiting | 18 Years | N/A | All | 36 | Phase 2 | United States |
| 16 | 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 |
| 17 | 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; Hydrogen Sulfate in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | Neurofibromatosis 1;Neurofibromatosis Type 1;NF1;Neurofibroma, Plexiform | Drug: AZD6244 | National Cancer Institute (NCI) | NULL | Active, not recruiting | 2 Years | 18 Years | All | 99 | Phase 1/Phase 2 | United States |
| 18 | NCT00846430 (ClinicalTrials.gov) | October 2008 | 16/1/2009 | Medical Treatment of High-Risk Neurofibromas | Medical Treatment of High-Risk Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies | Neurofibromatosis 1 | Drug: Peg-Interferon alpha-2b;Drug: Celecoxib (Celebrex);Drug: Temozolomide (temodar);Drug: Vincristine Sulfate (Oncovin) | Spectrum Health Hospitals | NULL | Completed | 2 Years | 30 Years | All | 11 | Phase 2 | United States |
| 19 | NCT00352495 (ClinicalTrials.gov) | June 2006 | 13/7/2006 | Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma | A Phase I Study of Vinblastine in Combination With Carboplatin for Children With Newly Diagnosed and Recurrent Low-Grade Gliomas | Brain and Central Nervous System Tumors;Neurofibromatosis Type 1 | Drug: carboplatin;Drug: vinblastine sulfate | Children's Oncology Group | National Cancer Institute (NCI) | Completed | N/A | 21 Years | Both | 26 | Phase 1 | United States;Canada |