34. Neurofibromatosis Clinical trials / Disease details
Clinical trials : 137 / Drugs : 213 - (DrugBank : 76) / Drug target genes : 87 - Drug target pathways : 200
| 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 | EUCTR2020-005608-20-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 | ||
| 3 | NCT04544007 (ClinicalTrials.gov) | December 15, 2021 | 2/9/2020 | A Phase II Trial of Poly-ICLC for Low-Grade Gliomas | A Phase II Trial of Poly-ICLC for Progressive, Previously Treated Low-Grade Gliomas in Children and Young Adults With Neurofibromatosis Type 1 | NF1;Low-grade Glioma | Drug: Poly ICLC | University of Alabama at Birmingham | Children's Healthcare of Atlanta;Children's Hospital Los Angeles | Not yet recruiting | N/A | 22 Years | All | 20 | Phase 2 | United States |
| 4 | NCT05130866 (ClinicalTrials.gov) | December 1, 2021 | 10/11/2021 | Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas | A Parallel-group, Two-staged, Phase 2/3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of REC-2282 in Participants With Progressive NF2 Mutated Meningiomas | Neurofibromatosis Type 2 | Drug: REC-2282;Drug: Placebo | Recursion Pharmaceuticals Inc. | NULL | Not yet recruiting | 12 Years | N/A | All | 89 | Phase 2/Phase 3 | NULL |
| 5 | NCT04750928 (ClinicalTrials.gov) | November 29, 2021 | 10/2/2021 | Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas | A Phase I/II Study of the Cyclin-Dependent Kinase(CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type 1 (NF1) Related Atypical Neurofibromas | Neurofibromatosis 1 | Drug: Abemaciclib | National Cancer Institute (NCI) | NULL | Recruiting | 12 Years | N/A | All | 50 | Phase 1/Phase 2 | United States |
| 6 | 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 | 38 | Phase 1;Phase 2 | United States;Spain;Russian Federation;Netherlands;Germany;Italy | ||
| 7 | NCT04924608 (ClinicalTrials.gov) | November 19, 2021 | 14/5/2021 | 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 Plexiform Neurofibromas (KOMET) | Neurofibromatosis 1;Plexiform Neurofibroma (PN) | Drug: Selumetinib;Other: Placebo | AstraZeneca | Merck Sharp & Dohme Corp. | Recruiting | 18 Years | N/A | All | 146 | Phase 3 | United States;Australia;Brazil;Canada;China;France;Germany;Italy;Japan;Netherlands;Russian Federation;Spain;United Kingdom |
| 8 | JPRN-jRCT2041210036 | 19/10/2021 | 24/06/2021 | 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 Plexiform Neurofibromas | A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas. - KOMET | Neurofibromatosis 1, Plexiform Neurofibroma (PN) | Arm A: selumetinib 25 mg/m2 orally bid Arm B: placebo orally bid | Yamaji Shigeyuki | NULL | Pending | >= 18age old | Not applicable | Both | 15 | Phase 3 | Australia;Brasil;Canada;China;France;Germany;Italy;Netherland;Poland;Russia;Spain;USA;UK;Japan |
| 9 | 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 | 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 | ||
| 10 | NCT05028166 (ClinicalTrials.gov) | October 1, 2021 | 24/8/2021 | Individual Patient Compassionate Use of Mirdametinib | Neurofibromatosis Type 1-Associated Plexiform Neurofibromas;Histiocytic Neoplasm;Other MAP-K Pathway Driven Diseases | Drug: Mirdametinib (MEK Inhibitor) | SpringWorks Therapeutics, Inc. | NULL | Available | 2 Years | N/A | All | NULL | |||
| 11 | NCT05005845 (ClinicalTrials.gov) | September 20, 2021 | 2/8/2021 | NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) | A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2 Dose-Response Study to Determine Safety and Effectiveness of Two Concentrations of NFX-179 Gel in Subjects With Cutaneous Neurofibromas | Cutaneous Neurofibroma;Neurofibromatosis 1 | Drug: NFX-179 gel;Drug: Vehicle gel | NFlection Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 168 | Phase 2 | United States |
| 12 | 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 | ||
| 13 | NCT05011019 (ClinicalTrials.gov) | September 7, 2021 | 10/8/2021 | A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis | Phase Ib Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis (Neurofibromatosis and Malignant Peripheral Nerve Sheath Tumors) | Neurofibromatosis and Malignant Peripheral Nerve Sheath Tumors | Drug: AL2846 capsules | Chia Tai Tianqing Pharmaceutical Group Co., Ltd. | NULL | Recruiting | 18 Years | 75 Years | All | 192 | Phase 1/Phase 2 | China |
| 14 | NCT04954001 (ClinicalTrials.gov) | August 2021 | 17/6/2021 | Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 | A Multi-center, Open-label, Single-arm Phase I Dose-escalation and Phase II Dose-expansion Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 | Neurofibromatosis 1;Plexiform Neurofibroma;NF1 | Drug: FCN-159 | Shanghai Fosun Pharmaceutical Development Co, Ltd. | NULL | Not yet recruiting | 2 Years | 70 Years | All | 160 | Phase 1/Phase 2 | NULL |
| 15 | NCT05101148 (ClinicalTrials.gov) | July 21, 2021 | 16/6/2021 | Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Toxicity of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas | 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 Related Plexiform Neurofibromas | Neurofibromatosis Type 1 | Drug: Selumetinib | AstraZeneca | Merck Sharp & Dohme Corp. | Active, not recruiting | 12 Years | 17 Years | All | 24 | Phase 1 | United States;Poland;Russian Federation;Spain |
| 16 | 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 | ||
| 17 | NCT04730583 (ClinicalTrials.gov) | May 2021 | 12/1/2021 | Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas | Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas | Cutaneous Neurofibroma | Drug: Kybella;Device: 980nm laser;Device: 755nm Alexandrite Laser;Device: Radiofrequency | Massachusetts General Hospital | Johns Hopkins University | Not yet recruiting | 18 Years | 85 Years | All | 20 | Phase 1 | NULL |
| 18 | EUCTR2020-000156-35-NL (EUCTR) | 18/03/2021 | 03/03/2020 | 89Zr-Bevacizumab PET/CT imaging of vestibular schwannomas for the prediction of bevacizumab treatment effect in patients with symptomatic neurofibromatosis type 2. | 89Zr-Bevacizumab PET/CT imaging of vestibular schwannomas for the prediction of bevacizumab treatment effect in patients with symptomatic neurofibromatosis type 2. - 89Zr-Bevacizumab PET/CT imaging in NF2 patients | Neurofibromatosis type 2;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: 89Zr-bevacizumab INN or Proposed INN: Unknown Other descriptive name: BEVACIZUMAB ZIRCONIUM ZR-89 | Leiden University Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 2 | Netherlands | ||
| 19 | NCT04590235 (ClinicalTrials.gov) | December 16, 2020 | 28/7/2020 | 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) | 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. | Active, not recruiting | 3 Years | 99 Years | All | 32 | Phase 1 | China |
| 20 | NCT04481048 (ClinicalTrials.gov) | December 15, 2020 | 15/5/2020 | Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1 | Antioxidant Therapy With N-acetylcysteine for Motor Behavior and/or Learning in Children With Neurofibromatosis Type 1 | Neurofibromatosis 1 | Drug: N-Acetyl cysteine;Other: Placebo | Children's Hospital Medical Center, Cincinnati | United States Department of Defense | Recruiting | 8 Years | 16 Years | All | 58 | Phase 2 | United States |
| 21 | 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 | |
| 22 | 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 | complete | 3 | 18 | BOTH | 9 | Phase 1 | Japan, North America |
| 23 | 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 | Active, not recruiting | 3 Years | 18 Years | All | 12 | Phase 1 | Japan |
| 24 | NCT04435665 (ClinicalTrials.gov) | August 21, 2020 | 8/6/2020 | NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) | A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2a Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of NFX-179 Gel in Subjects With Cutaneous Neurofibromas | Neurofibromatosis 1;Cutaneous Neurofibroma | Drug: NFX-179 Gel;Drug: Vehicle Gel | NFlection Therapeutics, Inc. | NULL | Completed | 18 Years | N/A | All | 48 | Phase 2 | United States |
| 25 | NCT04461886 (ClinicalTrials.gov) | July 8, 2020 | 3/7/2020 | A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I | A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I | Neurofibromatosis | Drug: NPC-12G gel | Nobelpharma | NULL | Active, not recruiting | 3 Years | N/A | All | 100 | Phase 3 | Japan |
| 26 | NCT04374305 (ClinicalTrials.gov) | June 20, 2020 | 30/4/2020 | Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 | Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 (INTUITT-NF2) | Neurofibromatosis Type 2;Vestibular Schwannoma;Non-vestibular Schwannoma;Meningioma;Ependymoma | Drug: Brigatinib | Scott R. Plotkin, MD, PhD | Takeda;The Children's Tumor Foundation | Recruiting | 12 Years | N/A | All | 80 | Phase 2 | United States |
| 27 | NCT04283669 (ClinicalTrials.gov) | February 18, 2020 | 22/2/2020 | Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas | Open-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas | Neurofibromatosis 2;Progressive Vestibular Schwannoma (VS) | Drug: Crizotinib | University of Alabama at Birmingham | Memorial Sloan Kettering Cancer Center | Active, not recruiting | 6 Years | N/A | All | 19 | Phase 2 | United States |
| 28 | 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 | 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 | 220 | Phase 3 | United States;Canada;Puerto Rico |
| 29 | NCT04201457 (ClinicalTrials.gov) | November 20, 2019 | 12/12/2019 | A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration | Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-associated Recurrent or Progressive Gliomas in Children and Young Adults | Low Grade Glioma (LGG) of Brain With BRAF Aberration;High Grade Glioma (HGG) of the Brain With BRAF Aberration;Low Grade Glioma of Brain With Neurofibromatosis Type 1 | Drug: Dabrafenib;Drug: Trametinib;Drug: Hydroxychloroquine | Pediatric Brain Tumor Consortium | NULL | Recruiting | 1 Year | 30 Years | All | 75 | Phase 1/Phase 2 | United States |
| 30 | NCT03975829 (ClinicalTrials.gov) | November 4, 2019 | 30/5/2019 | Pediatric Long-Term Follow-up and Rollover Study | An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib) | Diffuse Astrocytoma;Anaplastic Astrocytoma;Astrocytoma;Oligodendroglioma, Childhood;Anaplastic Oligodendroglioma;Glioblastoma;Pilocytic Astrocytoma;Giant Cell Astrocytoma;Pleomorphic Xanthoastrocytoma;Anaplastic Pleomorphic Xanthoastrocytoma;Angiocentric Glioma;Chordoid Glioma of Third Ventricle;Gangliocytoma;Ganglioglioma;Anaplastic Ganglioglioma;Dysplastic Gangliocytoma of Cerebrellum;Desmoplastic Infantile Astrocytoma and Ganglioglioma;Papillary Glioneuronal Tumor;Rosette-forming Glioneurona Tumor;Central Neurocytoma;Extraventricular Neurocytoma;Cerebellar Liponeurocytoma;Neurofibromatosis Type 1 | Drug: dabrafenib;Drug: trametinib | Novartis Pharmaceuticals | NULL | Recruiting | 1 Year | N/A | All | 250 | Phase 4 | United States;Canada;France;Spain;United Kingdom |
| 31 | EUCTR2019-001317-16-NL (EUCTR) | 30/10/2019 | 16/10/2019 | The TRAIN study: TRAmetinib In Neurofibromatosis type 1 related symptomatic plexiform neurofibromas | The TRAIN study: TRAmetinib In Neurofibromatosis type 1 related symptomatic plexiform neurofibromas - TRAIN study: Trametinib in NF1-PNF | Adult patients (age >17 years) with (mosaic) NF1 with inoperable symptomatic plexiform neurofibromas MedDRA version: 20.0;Level: PT;Classification code 10029267;Term: Neurofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 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] | Trade Name: Mekinist | ErasmusMC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Netherlands | ||
| 32 | JPRN-JapicCTI-194999 | 15/10/2019 | 14/10/2019 | BeatNF2 trial | A Randomized Double-blind Multicenter trial to Assess the Efficacy and Safety of Bevacizumab for Neurofibromatosis Type 2 | Neurofibromatosis type 2 | Intervention name : Bevacizumab (Genitical Recombination) INN of the intervention : Bevacizumab (Genitical Recombination) Dosage And administration of the intervention : 5mg/kg in a total volume of 100ml, div, every 2 week Control intervention name : Isotonic sodium chloride solution INN of the control intervention : - Dosage And administration of the control intervention : 100ml, div | Kiyoshi Saito | Masazumi Fujii, Masao Kobayakawa, Akihiro Inano, Jun Sakuma, Taku Sato, Akio Morita, Mitsuhiro Hasegawa, Takafumi Mitsuhara, Takashi Tamiya, Takeo Goro, Shigeru Yamaguchi, Hirofumi Nakatomi, Soichi Oya | pending | 18 | 64 | BOTH | 60 | Phase 2 | Japan |
| 33 | 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;Puerto Rico |
| 34 | 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 |
| 35 | 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 |
| 36 | NCT03962543 (ClinicalTrials.gov) | September 6, 2019 | 12/4/2019 | MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas | A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity | Plexiform Neurofibroma;Neurofibromatosis Type 1 (NF1) | Drug: Mirdametinib (PD-0325901) oral capsule or dispersible tablet | SpringWorks Therapeutics, Inc. | NULL | Active, not recruiting | 2 Years | N/A | All | 114 | Phase 2 | United States |
| 37 | NCT04085159 (ClinicalTrials.gov) | September 1, 2019 | 9/9/2019 | Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis | Immunotherapy Targeting Neurofibromatosis or Schwannomatosis | Cancer | Biological: Antigen-specific T cells CART/CTL and DCvac | Shenzhen Geno-Immune Medical Institute | Shenzhen Hospital of Southern Medical University;Shenzhen Children's Hospital | Recruiting | 1 Year | 80 Years | All | 100 | Phase 1/Phase 2 | China |
| 38 | NCT03741101 (ClinicalTrials.gov) | June 10, 2019 | 5/11/2018 | Treatment of NF1-related Plexiform Neurofibroma With Trametinib | Treatment of NF1-related Plexiform Neurofibroma With Trametinib; a Single Arm, Open-label Trial With the Goals of Volumetric Partial Remission and Pain Relief | Neurofibromatosis 1;Child;Neurofibroma, Plexiform | Drug: Trametinib | Region Skane | Novartis | Recruiting | 1 Year | 17 Years | All | 15 | Phase 2 | Sweden |
| 39 | NCT03826940 (ClinicalTrials.gov) | February 19, 2019 | 23/1/2019 | From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1 | Linking Inhibition From Molecular to Systems and Cognitive Levels: a Preclinical and Clinical Approach in Autism Spectrum Disorders and Neurofibromatosis. | Autism Spectrum Disorder;Neurofibromatosis 1 | Drug: Lovastatin 60 MG;Drug: Placebos | University of Coimbra | NULL | Completed | 16 Years | 65 Years | All | 16 | N/A | Portugal |
| 40 | NCT04481035 (ClinicalTrials.gov) | January 15, 2019 | 14/4/2020 | Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1 | Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1 | Neurofibromatosis 1 | Drug: N-acetylcysteine (NAC);Other: Placebo | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | 8 Years | 16 Years | All | 5 | Phase 2 | United States |
| 41 | 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 | ||
| 42 | EUCTR2018-001846-32-SE (EUCTR) | 18/09/2018 | 31/07/2018 | Treatment of symptomatic plexiform neurofibromas, a benign tumour associated with the disorder Neurofibromatosis type 1, in children with the drug trametinib | Treatment of NF1-related plexiform neurofibroma with trametinib; a single arm, open-label trial with the goals of volumetric partial remission and pain relief - plexifpc | NF1-related plexiform neurofibroma;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Mekinist Product Name: Mekinist | VO Barnmedicin, Skånes University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 2 | Sweden | ||
| 43 | 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 |
| 44 | NCT03688568 (ClinicalTrials.gov) | September 1, 2018 | 11/9/2018 | Study of Imatinib in Children With Neurofibromatosis and Airway Tumors | Phase 2 Study of Imatinib in Children With Neurofibromatosis and Airway Tumors | Neurofibroma, Plexiform | Drug: Imatinib Mesylate | Indiana University | NULL | Withdrawn | 6 Months | 12 Years | All | 0 | Phase 2 | United States |
| 45 | NCT03079999 (ClinicalTrials.gov) | June 11, 2018 | 6/3/2017 | Study of Aspirin in Patients With Vestibular Schwannoma | Prospective, Randomized, Placebo-Controlled Phase II Trial of Aspirin for Vestibular Schwannomas | Vestibular Schwannoma;Acoustic Neuroma;Neurofibromatosis 2 | Drug: Aspirin;Drug: Placebo | Massachusetts Eye and Ear Infirmary | United States Department of Defense;Massachusetts General Hospital | Recruiting | 12 Years | N/A | All | 300 | Phase 2 | United States |
| 46 | ChiCTR1800016040 | 2018-05-09 | 2018-05-08 | Clinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis | Clinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis | Type II Neurofibromatosis | Case series:Bevacizumab therapy; | Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of Medicine | NULL | Recruiting | 6 | Both | Case series:30; | China | ||
| 47 | NCT03513757 (ClinicalTrials.gov) | March 4, 2018 | 4/2/2018 | Dexmedetomidine and Propofol for Pediatric MRI Sedation | An Observer-blinded Randomized Study of Propofol Infusion vs Bolus Dexmedetomidine and Propofol Sedation for Pediatric Magnetic Resonance Imaging | Headache;Tumor;Seizure Disorder;Neurofibromatoses;Hydrocephalus;Abdominal Neoplasm;Spine Deformity | Drug: propofol;Drug: Dexmedetomidine;Drug: Glycopyrrolate;Drug: Lidocaine 1% Injectable Solution;Drug: Nitrous Oxide;Drug: Sevoflurane | Medical College of Wisconsin | NULL | Completed | 12 Months | 60 Months | All | 40 | Phase 4 | United States |
| 48 | EUCTR2013-003405-26-DK (EUCTR) | 22/02/2018 | 24/04/2017 | Treating learning disabilities in Neurofibromatosis 1 using lamotrigine | The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL) - Treating learning disabilities in Neurofibromatosis 1 using lamotrigine | Neurofibromatosis type 1 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE INN or Proposed INN: lamotrigine Other descriptive name: LAMOTRIGINE | Erasmus MC | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | Spain;Belgium;Denmark;Netherlands | ||
| 49 | EUCTR2016-005022-10-DE (EUCTR) | 13/02/2018 | 09/11/2017 | Improvement of synaptic plasticity and cognitive function in RAS pathway disorders | Improvement of synaptic plasticity and cognitive function in RAS pathway disorders | Noonan Syndrom and Neurofibromatosis Type 1 MedDRA version: 20.0;Level: PT;Classification code 10029748;Term: Noonan syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10029268;Term: Neurofibromatosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify | Product Name: Lovastatin INN or Proposed INN: Lovastatin Other descriptive name: LOVASTATIN Product Name: Lamotrigin beta INN or Proposed INN: Lamotrigin | Technische Universität München, Fakultät für Medizin | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | Germany | ||
| 50 | NCT03232892 (ClinicalTrials.gov) | February 13, 2018 | 14/7/2017 | Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung Cancer | Phase II Trial to Evaluate Trametinib in Patients With Advanced NF1-mutant Non-small Cell Lung Cancer | Non-small Cell Lung Cancer | Drug: Trametinib | University of California, San Francisco | Novartis Pharmaceuticals;American Cancer Society, Inc. | Terminated | 18 Years | N/A | All | 1 | Phase 2 | United States |
| 51 | NCT02700230 (ClinicalTrials.gov) | December 8, 2017 | 25/2/2016 | Vaccine Therapy in Treating Patients With Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery | Phase I Trial of Intratumoral Administration of a NIS-Expressing Derivative Manufactured From a Genetically Engineered Strain of Measles Virus in Patients With Unresectable or Recurrent Malignant Peripheral Nerve Sheath Tumor | Metastatic Malignant Peripheral Nerve Sheath Tumor;Neurofibromatosis Type 1;Recurrent Malignant Peripheral Nerve Sheath Tumor | Procedure: Computed Tomography;Other: Laboratory Biomarker Analysis;Biological: Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter;Other: Quality-of-Life Assessment;Procedure: Single Photon Emission Computed Tomography | Mayo Clinic | National Cancer Institute (NCI) | Recruiting | 18 Years | N/A | All | 30 | Phase 1 | United States |
| 52 | NCT03231306 (ClinicalTrials.gov) | November 28, 2017 | 21/7/2017 | Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas | A Phase II Study of Binimetinib in Children and Adults With NF1 Associated Plexiform Neurofibromas (PNOC010) | Neurofibromatosis Type 1;Plexiform Neurofibroma | Drug: Binimetinib | University of Alabama at Birmingham | Array BioPharma;Pacific Pediatric Neuro-Oncology Consortium | Active, not recruiting | 1 Year | N/A | All | 40 | Phase 2 | United States |
| 53 | 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 | ||
| 54 | EUCTR2013-003405-26-ES (EUCTR) | 16/10/2017 | 31/07/2017 | Treating learning disabilities in Neurofibromatosis 1 using lamotrigine | The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL) | Neurofibromatosis type 1 MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000012614;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE | Erasmus MC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Belgium;Spain;Netherlands | ||
| 55 | NCT03190915 (ClinicalTrials.gov) | October 6, 2017 | 16/6/2017 | Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia | A Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia;Neurofibromatosis Type 1 | Drug: Trametinib | National Cancer Institute (NCI) | Children's Oncology Group | Recruiting | 1 Month | 21 Years | All | 24 | Phase 2 | United States |
| 56 | NCT01968590 (ClinicalTrials.gov) | August 16, 2017 | 7/10/2013 | Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1) | A Phase II Trial on the Effect of Low-Dose Versus High-Dose Vitamin D Supplementation on Bone Mass in Adults With Neurofibromatosis Type 1 (NF1) | Neurofibromatosis Type 1 (NF1) | Drug: Cholecalciferol | University of Utah | U.S. Army Medical Research and Development Command;Universitätsklinikum Hamburg-Eppendorf;University of British Columbia;Children's Hospital Medical Center, Cincinnati | Recruiting | 25 Years | 40 Years | All | 320 | Phase 2 | United States;Canada;Germany |
| 57 | 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 |
| 58 | 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 |
| 59 | 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 |
| 60 | 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 |
| 61 | NCT03090971 (ClinicalTrials.gov) | February 15, 2017 | 8/3/2017 | Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1 | Clinical Assessment of the Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With Neurofibromatosis Type 1 | Neurofibromatosis 1;Cutaneous Neurofibroma | Drug: Diclofenac Sodium;Drug: Saline Solution | Fundação Educacional Serra dos Órgãos | NULL | Completed | 18 Years | N/A | All | 7 | Phase 2 | Brazil |
| 62 | NCT02964884 (ClinicalTrials.gov) | November 2016 | 9/11/2016 | Interventions for Reading Disabilities in NF1 | Neurobiology and Treatment of Reading Disability in NF1 | Neurofibromatosis Type 1;Learning Disability;Reading Disability;NF1 | Drug: Lovastatin;Behavioral: reading tutoring intervention;Drug: Placebo Oral Tablet;Behavioral: Other Academic sham tutoring | Vanderbilt University | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Recruiting | 8 Years | 20 Years | All | 120 | Phase 2 | United States |
| 63 | ChiCTR-DDD-16009551 | 2016-10-28 | 2016-10-23 | Comparative study of Sanger sequencing and specific panel high-throughput sequencing for detection of NF1 pathogenic mutations in Han Chinese | Comparative study of Sanger sequencing and specific panel high-throughput sequencing for detection of NF1 pathogenic mutations in Han Chinese | neurofibromatosis;M95400/1 | Gold Standard:;Index test:; | Shanghai 9th People's Hospital Affiliated to Shanghai Jiaotong University School of Medicine | NULL | Recruiting | 10 | 45 | Both | Target condition:0;Difficult condition:0 | China | |
| 64 | NCT02831257 (ClinicalTrials.gov) | August 31, 2016 | 7/7/2016 | AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas | A Single Arm Phase 2 Study of the Dual mTORC1/mTORC2 Inhibitor AZD2014 Provided on an Intermittent Schedule for Neurofibromatosis 2 Patients With Progressive or Symptomatic Meningiomas | Neurofibromatosis 2;Meningioma | Drug: AZD2014 | Massachusetts General Hospital | AstraZeneca;United States Department of Defense | Active, not recruiting | 18 Years | N/A | All | 18 | Phase 2 | United States |
| 65 | NCT02728388 (ClinicalTrials.gov) | August 2016 | 25/3/2016 | Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II | Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II | NEUROFIBROMATOSIS 1 | Drug: aminolevulinic acid | Harry T Whelan, MD | NULL | Recruiting | 14 Years | 30 Years | All | 30 | Phase 2 | United States |
| 66 | NCT02934256 (ClinicalTrials.gov) | July 2016 | 13/10/2016 | Icotinib Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors | Icotinib Hydrochloride Tablets Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors | Vestibular Schwannoma;Neurofibromatosis Type 2 | Drug: Icotinib | Li Peng | Betta Pharmaceuticals Co., Ltd. | Completed | 16 Years | 50 Years | All | 10 | Phase 2 | China |
| 67 | JPRN-UMIN000021030 | 2016/03/16 | 22/02/2016 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 | Neurofibromatosis type 1 | 0.2% Sirolimus gel twice daily 24 weeks topical application 0.4% Sirolimus gel twice daily 24 weeks topical application Placebo gel twice daily 24 weeks topical application | Osaka University Hospital | NULL | Complete: follow-up complete | 16years-old | 70years-old | Male and Female | 18 | Phase 2 | Japan | |
| 68 | NCT02718131 (ClinicalTrials.gov) | March 2016 | 7/3/2016 | A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1) | A Study of INFUSE Bone Graft (Recombinant Human Bone Morphogenetic Protein-2/Absorbable Collagen Sponge) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis 1 (NF1) | NF1;Congenital Pseudarthrosis of Tibia | Device: INFUSE Bone Graft (BMP-2);Procedure: Control Group | University of Alabama at Birmingham | The Children's Tumor Foundation;Medtronic | Active, not recruiting | 2 Years | 18 Years | All | 54 | N/A | United States;Australia |
| 69 | 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 |
| 70 | 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 |
| 71 | NCT02282917 (ClinicalTrials.gov) | September 2015 | 31/10/2014 | Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma | Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma | Vestibular Schwannoma;Meningioma;Acoustic Neuroma;Neurofibromatosis Type 2 | Drug: AR-42 | Massachusetts Eye and Ear Infirmary | Johns Hopkins University;Mayo Clinic;Stanford University;Ohio State University;Nationwide Children's Hospital | Active, not recruiting | 18 Years | N/A | All | 5 | Early Phase 1 | United States |
| 72 | NCT02415153 (ClinicalTrials.gov) | July 14, 2015 | 13/4/2015 | Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors | A Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS Tumors | Neurofibromatosis Type 1;Recurrent Childhood Brain Stem Glioma;Recurrent Childhood Visual Pathway Glioma;Recurrent Primary Central Nervous System Neoplasm;Refractory Primary Central Nervous System Neoplasm | Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Pomalidomide | National Cancer Institute (NCI) | NULL | Active, not recruiting | 3 Years | 20 Years | All | 29 | Phase 1 | United States |
| 73 | NCT01345136 (ClinicalTrials.gov) | July 1, 2015 | 21/4/2011 | Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma | A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma | Neurofibromatosis Type 2;Neuroma, Acoustic | Drug: RAD001, everolimus | Jonsson Comprehensive Cancer Center | Novartis Pharmaceuticals | Active, not recruiting | 16 Years | 65 Years | All | 4 | Phase 2 | United States |
| 74 | EUCTR2013-003405-26-BE (EUCTR) | 30/04/2015 | 30/03/2015 | Treating learning disabilities in Neurofibromatosis 1 using lamotrigine | The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL) | Neurofibromatosis type 1 MedDRA version: 17.1;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lamotrigine dispers | Erasmus MC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Belgium;Netherlands | ||
| 75 | NCT02390752 (ClinicalTrials.gov) | April 29, 2015 | 17/3/2015 | PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) | Phase I/II Trial of PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) | Neurofibroma, Plexiform;Precursor Cell Lymphoblastic Leukemia-Lymphoma;Leukemia, Promyelocytic, Acute;Sarcoma | Drug: Turalio | National Cancer Institute (NCI) | NULL | Recruiting | 3 Years | 35 Years | All | 81 | Phase 1/Phase 2 | United States |
| 76 | NCT02332902 (ClinicalTrials.gov) | February 2015 | 12/12/2014 | Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T | Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T | Neurofibromatosis 1 | Drug: Everolimus | The University of Texas Health Science Center, Houston | Texas Neurofibromatosis Foundation | Completed | 18 Years | N/A | All | 24 | Phase 2 | United States |
| 77 | NCT02211768 (ClinicalTrials.gov) | December 8, 2014 | 6/8/2014 | Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1 | Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: Clinical, Histopathologic, and Genomic Analysis | Neurofibromatosis;MPNST | Procedure: MRI, FDG-PET/CT scans;Drug: [18F]-FLT-PET/CT scans | National Cancer Institute (NCI) | NULL | Completed | 10 Years | N/A | All | 10 | Phase 1 | United States |
| 78 | NCT02256124 (ClinicalTrials.gov) | October 2014 | 17/9/2014 | Effect of Lamotrigine on Cognition in NF1 | The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial (NF1-EXCEL) | Neurofibromatosis Type 1 | Drug: Lamotrigine;Drug: Placebo | Erasmus Medical Center | Universitaire Ziekenhuizen Leuven;ZonMw: The Netherlands Organisation for Health Research and Development;Hospital Sant Joan de Deu | Terminated | 12 Years | 18 Years | All | 41 | Phase 2/Phase 3 | Belgium;Netherlands;Spain |
| 79 | JPRN-UMIN000015081 | 2014/09/08 | 07/09/2014 | Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 | neurofibromatosis type1 | 1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks. 2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks. 3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks. | Department of Dermatology Graduate School of Medicine, Osaka University | NULL | Recruiting | 20years-old | Not applicable | Male and Female | 3 | Phase 2,3 | Japan | |
| 80 | EUCTR2013-003405-26-NL (EUCTR) | 03/09/2014 | 20/09/2013 | Treating learning disabilities in Neurofibromatosis 1 using lamotrigine | The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL) | Neurofibromatosis type 1 MedDRA version: 17.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lamotrigine dispers INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE INN or Proposed INN: lamotrigine Other descriptive name: LAMOTRIGINE | Erasmus MC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Netherlands | ||
| 81 | NCT02101736 (ClinicalTrials.gov) | June 2014 | 14/3/2014 | Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults | A Phase II Study of Cabozantinib (XL184) for Plexiform Neurofibromas in Subjects With Neurofibromatosis Type 1 in Children and Adults | NF1;Neurofibromatosis;Plexiform Neurofibromas | Drug: Cabozantinib | University of Alabama at Birmingham | NULL | Active, not recruiting | 3 Years | N/A | All | 45 | Phase 2 | United States |
| 82 | NCT02177825 (ClinicalTrials.gov) | June 2014 | 25/6/2014 | Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas | Phase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas | Plexiform Neurofibromas | Drug: Imatinib Mesylate | St. Justine's Hospital | NULL | Terminated | 2 Years | 21 Years | All | 5 | Phase 2 | Canada |
| 83 | NCT02096471 (ClinicalTrials.gov) | June 2014 | 12/3/2014 | MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1 | A Phase 2 Trial of the MEK Inhibitor PD-0325901 in Adolescents and Adults With NF1-Associated Morbid Plexiform Neurofibromas | Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN | Drug: PD-0325901 | University of Alabama at Birmingham | NULL | Completed | 16 Years | N/A | All | 19 | Phase 2 | United States |
| 84 | NCT02129647 (ClinicalTrials.gov) | April 2014 | 29/4/2014 | Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas | Phase II Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas | Neurofibromatosis Type 2;Vestibular Schwannomas | Drug: Axitinib | NYU Langone Health | NULL | Completed | 18 Years | N/A | All | 13 | Phase 2 | United States |
| 85 | NCT02104323 (ClinicalTrials.gov) | January 2014 | 26/3/2014 | Endostatin Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors | Recombinant Human Endostatin Injection Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors by Continuous Intravenous Pumping | Vestibular Schwannoma;Neurofibromatosis Type 2 | Drug: Endostatin | Beijing Tiantan Hospital | Shandong Simcere-Medgenn Bio-pharmaceutical Co., Ltd | Completed | 16 Years | 30 Years | All | 20 | Phase 2 | China |
| 86 | EUCTR2012-003005-10-DK (EUCTR) | 06/06/2013 | 08/05/2013 | LOW-GRADE GLIOMA | PHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA - Vinilo | Children, adolescents and young adults with refractory or recurrent low-grade gliomas, and children, adolescents and young adults with neurofibromatosis type 1 and previously untreated low-grade gliomas MedDRA version: 20.0;Level: PT;Classification code 10065443;Term: Malignant glioma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.0;Classification code 10038111;Term: Recurrent cancer;Classification code 10070308;Term: Refractory cancer;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Cancer [C04] | Product Name: Nilotinib Product Code: AMN107 Other descriptive name: NILOTINIB HYDROCHLORIDE MONOHYDRATE Trade Name: Tasigna 150mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB Trade Name: Tasigna 200mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB Product Name: Vinblastine Other descriptive name: VINBLASTINE SULPHATE Trade Name: Tasigna 50 mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB | Gustave Roussy | NULL | Not Recruiting | Female: yes Male: yes | 160 | Phase 2 | France;Spain;Denmark;Netherlands;Switzerland | ||
| 87 | NCT01880749 (ClinicalTrials.gov) | June 2013 | 11/6/2013 | Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas | Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas | Neurofibromatosis Type 2;Vestibular Schwannomas;Meningiomas | Drug: RAD001 | NYU Langone Health | NULL | Completed | 18 Years | N/A | All | 5 | Early Phase 1 | United States |
| 88 | NCT01767792 (ClinicalTrials.gov) | May 15, 2013 | 7/1/2013 | Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular Schwannomas | Open-label, Phase 2 Study of Bevacizumab in Children and Young Adults With Neurofibromatosis 2 and Progressive Vestibular Schwannomas That Are Poor Candidates for Standard Treatment With Surgery or Radiation | Neurofibromatosis Type 2;Progressive Vestibular Schwannomas | Drug: Bevacizumab | University of Alabama at Birmingham | Genentech, Inc. | Active, not recruiting | 6 Years | N/A | All | 22 | Phase 2 | United States |
| 89 | EUCTR2012-005742-38-GB (EUCTR) | 02/05/2013 | 05/04/2013 | Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1 | Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1 - SimvAstatin in Neurofibromatosis Type 1-Autism (SANTA) | Neurofibromatosis Type 1 MedDRA version: 14.1;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Simvastatin 20mg/5ml Oral Suspension Product Name: Simvastatin INN or Proposed INN: Simvastatin | Central Manchester University Hospitals NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 0 | Phase 2 | United Kingdom | ||
| 90 | NCT02584413 (ClinicalTrials.gov) | April 16, 2013 | 20/10/2015 | Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma | Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1 | Neurofibromatosis Type 1 | Device: Magnetic resonance imaging;Drug: Gadolinium contrast | Washington University School of Medicine | St. Louis Children's Hospital | Terminated | 1 Year | 7 Years | All | 29 | N/A | United States |
| 91 | NCT01661283 (ClinicalTrials.gov) | September 2012 | 7/8/2012 | SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath Tumors | Phase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors | Malignant Peripheral Nerve Sheath Tumors;MPNST;Sarcoma | Drug: everolimus;Drug: bevacizumab | Sarcoma Alliance for Research through Collaboration | Novartis Pharmaceuticals;Genentech, Inc.;United States Department of Defense | Completed | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 92 | NCT01365468 (ClinicalTrials.gov) | April 2012 | 27/5/2011 | Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1) | A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1) | Plexiform Neurofibroma Associated With Neurofibromatosis Type 1 | Drug: Everolimus (RAD001) | Novartis Pharmaceuticals | NULL | Terminated | 6 Years | N/A | All | 9 | Phase 2 | Israel |
| 93 | NCT01553149 (ClinicalTrials.gov) | March 19, 2012 | 10/3/2012 | Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma | A Phase II Randomized Trial of Lenalidomide (NSC # 703813) in Pediatric Patients With Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas | Neurofibromatosis Type 1;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Childhood Visual Pathway Glioma | Drug: Lenalidomide;Other: Pharmacological Study | National Cancer Institute (NCI) | NULL | Active, not recruiting | N/A | 21 Years | All | 75 | Phase 2 | United States;Australia;Canada;New Zealand |
| 94 | NCT01552434 (ClinicalTrials.gov) | March 16, 2012 | 7/3/2012 | Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease | A Phase I Trial of Bevacizumab, Temsirolimus Alone and in Combination With Valproic Acid, or Cetuximab in Patients With Advanced Malignancy and Other Indications | Advanced Malignant Neoplasm;Castleman Disease;Digestive System Carcinoma;Erdheim-Chester Disease;Lip and Oral Cavity Carcinoma;Lymphangioleiomyomatosis;Malignant Endocrine Neoplasm;Malignant Female Reproductive System Neoplasm;Malignant Male Reproductive System Neoplasm;Malignant Neoplasm;Malignant Respiratory Tract Neoplasm;Malignant Thoracic Neoplasm;Malignant Urinary System Neoplasm;Mesothelial Neoplasm;Metastatic Malignant Neoplasm;Metastatic Urothelial Carcinoma;Neurofibromatosis Type 2;Recurrent Adult Soft Tissue Sarcoma;Recurrent Breast Carcinoma;Recurrent Childhood Soft Tissue Sarcoma;Recurrent Digestive System Carcinoma;Recurrent Female Reproductive System Carcinoma;Recurrent Male Reproductive System Carcinoma;Recurrent Malignant Neoplasm;Recurrent Pharyngeal Carcinoma;Recurrent Thyroid Gland Carcinoma;Refractory Malignant Neoplasm;Soft Tissue Neoplasm;Stage III Breast Cancer AJCC v7;Stage III Pharyngeal Cancer;Stage IIIA Breast Cancer AJCC v7;Stage IIIB Breast Cancer AJCC v7;Stage IIIC Breast Cancer AJCC v7;Stage IV Breast Cancer AJCC v6 and v7;Stage IV Pharyngeal Cancer;Stage IVA Pharyngeal Cancer;Stage IVB Pharyngeal Cancer;Stage IVC Pharyngeal Cancer;Thyroid Gland Neoplasm | Biological: Bevacizumab;Biological: Cetuximab;Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Temsirolimus;Drug: Valproic Acid | M.D. Anderson Cancer Center | National Cancer Institute (NCI) | Active, not recruiting | N/A | N/A | All | 155 | Phase 1 | United States |
| 95 | NCT01682811 (ClinicalTrials.gov) | March 12, 2012 | 5/9/2012 | Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1) | Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red Light | Neurofibromatoses | Drug: Part 1 Levulan injection;Drug: Part 1 Levulan surface application;Drug: Part 1 Levulan surface application twice;Drug: Part 1 Levulan surface application twice with microneedling;Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1;Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2;Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3 | Harry T Whelan, MD | NULL | Completed | 18 Years | 90 Years | All | 20 | Phase 1 | United States |
| 96 | NCT01402817 (ClinicalTrials.gov) | March 2012 | 25/7/2011 | Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas | A Pilot Study of Sutent®/Sunitinib (SU11248), an Oral Multi-Targeted Tyrosine Kinase Inhibitor in Subjects With NF-1 Plexiform Neurofibromas | Neurofibromatosis;NF1;Plexiform Neurofibromas | Drug: Sutent®/Sunitinib | Indiana University | United States Department of Defense;Pfizer | Terminated | 3 Years | 65 Years | All | 19 | Phase 2 | United States |
| 97 | NCT01140360 (ClinicalTrials.gov) | February 2012 | 8/6/2010 | Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas | Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas | Neurofibromatosis;Neurofibromas | Drug: Gleevec | Kent Robertson | NULL | Completed | 3 Years | 65 Years | All | 21 | Phase 1/Phase 2 | United States |
| 98 | NCT01490476 (ClinicalTrials.gov) | January 2012 | 30/11/2011 | Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients | A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma | Neurofibromatosis 2 | Drug: RAD001 | Assistance Publique - Hôpitaux de Paris | NULL | Completed | 15 Years | N/A | All | 10 | Phase 2 | France |
| 99 | NCT01419639 (ClinicalTrials.gov) | October 2011 | 17/8/2011 | Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2 | Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2 | Neurofibromatosis Type II | Drug: Everolimus (RAD001) , Afinitor® | New York University School of Medicine | Novartis Pharmaceuticals;The Children's Tumor Foundation | Completed | 3 Years | N/A | All | 10 | Phase 2 | United States |
| 100 | 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;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 |
| 101 | EUCTR2011-001789-16-GB (EUCTR) | 26/08/2011 | 17/05/2011 | Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas - Sorafenib in NF2 | Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas - Sorafenib in NF2 | Neurofibromatosis, type 2 (acoustic neurofibromatosis) MedDRA version: 14.0;Level: LLT;Classification code 10029271;Term: Neurofibromatosis, type 2 (acoustic neurofibromatosis);System Organ Class: 10010331 - Congenital, familial and genetic disorders | Trade Name: Nexavar INN or Proposed INN: sorafenib (as tosylate) Other descriptive name: Nexavar | Plymouth Hospital NHS Trust | NULL | Not Recruiting | Female: yes Male: yes | 14 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | United Kingdom | ||
| 102 | EUCTR2010-023508-28-GB (EUCTR) | 14/04/2011 | 30/11/2010 | A CLINICAL TRIAL OF THE INTRA-TUMOURAL CONCENTRATION AND ACTIVITY OF NILOTINIB IN INTRA-CUTANEOUS SCHWANNOMAS - PHNT NilotinibNF2 | A CLINICAL TRIAL OF THE INTRA-TUMOURAL CONCENTRATION AND ACTIVITY OF NILOTINIB IN INTRA-CUTANEOUS SCHWANNOMAS - PHNT NilotinibNF2 | In vivo investigation of the intra-tumoural concentration and activity of nilotinib in cutaneous schwannomas (CS) in patienst with Neurofibromatosis 2 | Trade Name: Tasigna INN or Proposed INN: NILOTINIB | Plymouth Hospital NHS Trust | NULL | Not Recruiting | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | United Kingdom | ||
| 103 | NCT01412892 (ClinicalTrials.gov) | April 2011 | 8/8/2011 | Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas | A Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by Surgery | Neurofibromatosis Type 1;Plexiform Neurofibroma;Neurofibromatoses | Drug: RAD001: Everolimus | Assistance Publique - Hôpitaux de Paris | Novartis | Completed | 18 Years | 60 Years | Both | 30 | Phase 2 | France |
| 104 | EUCTR2010-019759-23-GB (EUCTR) | 12/01/2011 | 13/12/2010 | Evaluation of [11C]-methionine positron emission computerised tomography (PET CT) in diagnosing neurofibromatosis 1(NF1) - malignant peripheral nerve sheath tumours (MPNST) - Methionine PET | Evaluation of [11C]-methionine positron emission computerised tomography (PET CT) in diagnosing neurofibromatosis 1(NF1) - malignant peripheral nerve sheath tumours (MPNST) - Methionine PET | The diagnosis of malignant peripheral nerve sheath tumours in patients with neurofibromatosis 1 MedDRA version: 12;Level: LLT;Classification code 10029268;Term: Neurofibromatosis 1 associated malignant peripheral nerve sheath tumour | Product Name: [11C]-methionine INN or Proposed INN: 2-amino-4-(methyl[11C]sulfanyl)butanoic acid Other descriptive name: Methionine | Guys' and St. Thomas' NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | United Kingdom | ||||
| 105 | NCT01275586 (ClinicalTrials.gov) | January 2011 | 11/1/2011 | Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas | Pilot Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas | Neurofibromatosis;NF1;Neurofibromas | Drug: Tasigna | Indiana University | Novartis | Completed | 18 Years | N/A | All | 6 | Early Phase 1 | United States |
| 106 | NCT01207687 (ClinicalTrials.gov) | October 2010 | 1/9/2010 | Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2) | Phase 2 Study of Bevacizumab in Children and Adults With Neurofibromatosis Type 2 and Symptomatic Vestibular Schwannoma | Vestibular Schwannoma;Neurofibromatosis Type 2 | Biological: bevacizumab;Other: laboratory biomarker analysis;Procedure: quality-of-life assessment | National Cancer Institute (NCI) | NULL | Completed | 12 Years | N/A | All | 14 | Phase 2 | United States |
| 107 | NCT01125046 (ClinicalTrials.gov) | July 2010 | 7/5/2010 | Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas | Phase II Trial of Bevacizumab in Patients With Recurrent or Progressive Meningiomas | Acoustic Schwannoma;Adult Anaplastic Meningioma;Adult Ependymoma;Adult Grade I Meningioma;Adult Grade II Meningioma;Adult Meningeal Hemangiopericytoma;Adult Papillary Meningioma;Neurofibromatosis Type 1;Neurofibromatosis Type 2;Recurrent Adult Brain Tumor | Biological: bevacizumab | Northwestern University | NULL | Unknown status | 18 Years | N/A | All | 50 | Phase 2 | United States |
| 108 | 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 | Active, not recruiting | 3 Years | 21 Years | All | 220 | Phase 1/Phase 2 | United States |
| 109 | EUCTR2009-016922-15-ES (EUCTR) | 04/02/2010 | 19/11/2009 | ENSAYO PILOTO CON IMATINIB PARA PACIENTES CON NEUROFIBROMA PLEXIFORME ASOCIADO A LA NEUROFIBROMATOSIS TIPO I. PILOT STUDY WITH IMATINIB FOR PLEXIFORM NEUROFIBROMA IN NEUROFIBROMATOSIS TYPE I PATIENTS | ENSAYO PILOTO CON IMATINIB PARA PACIENTES CON NEUROFIBROMA PLEXIFORME ASOCIADO A LA NEUROFIBROMATOSIS TIPO I. PILOT STUDY WITH IMATINIB FOR PLEXIFORM NEUROFIBROMA IN NEUROFIBROMATOSIS TYPE I PATIENTS | Tratamiento de los neurofibromas plexiformes de alto riesgo no abordables quirúrgicamente en pacientes con neurofibromatosis tipo I | Trade Name: GLIVEC 100 mg comprimidos recubiertos con película INN or Proposed INN: IMATINIB Other descriptive name: IMATINIB Trade Name: GLIVEC 400 mg comprimidos recubiertos con película INN or Proposed INN: IMATINIB Other descriptive name: IMATINIB | HOSPITAL SANT JOAN DE DÉU | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Spain | ||||
| 110 | NCT01031901 (ClinicalTrials.gov) | December 2009 | 10/12/2009 | Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) | Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1 | Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma | Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin | The University of Texas Health Science Center, Houston | Society for Pediatric Dermatology | Completed | 13 Years | N/A | Both | 52 | Phase 1 | United States |
| 111 | EUCTR2009-010965-22-NL (EUCTR) | 29/09/2009 | 21/09/2009 | The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial | The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial | Neurofibromatosis 1 | Product Name: SIMVASTATIN Product Code: SIMVASTATIN INN or Proposed INN: SIMVASTATIN | Erasmus MC - Department of Pediatrics | NULL | Not Recruiting | Female: yes Male: yes | 106 | Netherlands | |||
| 112 | NCT00973739 (ClinicalTrials.gov) | September 2009 | 3/9/2009 | Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors | Phase II Study of Lapatinib in Children and Adults With Neurofibromatosis Type 2(NF2) and NF2-related Tumors | Neurofibromatosis 2;Vestibular Schwannoma | Drug: Lapatinib | New York University School of Medicine | GlaxoSmithKline | Completed | 4 Years | 80 Years | All | 21 | Phase 2 | United States |
| 113 | NCT00352599 (ClinicalTrials.gov) | September 2009 | 13/7/2006 | Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) | Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) | Neurofibromatosis 1 | Drug: Lovastatin;Drug: placebo pill | University of California, Los Angeles | NULL | Completed | 10 Years | 50 Years | Both | 44 | Phase 1 | United States |
| 114 | NCT00911248 (ClinicalTrials.gov) | July 31, 2009 | 28/5/2009 | PTC299 for Treatment of Neurofibromatosis Type 2 | A Phase 2 Study to Assess the Efficacy, Safety, and Pharmacodynamic Activity of PTC299 in Patients With Neurofibromatosis Type 2 | Neurofibromatosis 2 | Drug: PTC299 | PTC Therapeutics | United States Department of Defense | Terminated | 18 Years | N/A | All | 11 | Phase 2 | United States |
| 115 | NCT00853580 (ClinicalTrials.gov) | July 2009 | 23/2/2009 | A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1 | A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1 | Neurofibromatosis Type 1 | Drug: Lovastatin ™;Device: placebo | University of Alabama at Birmingham | Boston Children's Hospital;Children's Hospital of Philadelphia;Children's National Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of Medicine;Sydney Children's Hospitals Network;University of Texas Southwestern Medical Center | Completed | 8 Years | 15 Years | All | 146 | Phase 2 | United States;Australia |
| 116 | NCT00863122 (ClinicalTrials.gov) | June 2009 | 13/3/2009 | Concentration and Activity of Lapatinib in Vestibular Schwannomas | Exploration and Estimation of Intratumoral Concentration and Activity of Lapatinib in Vivo in Vestibular Schwannomas | Vestibular Schwannoma;NF2;Neurofibromatosis 2;Acoustic Neuroma;Auditory Tumor | Drug: lapatinib | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | The Children's Tumor Foundation;GlaxoSmithKline;New York University;Ohio State University;House Research Institute;Washington University School of Medicine;Weill Medical College of Cornell University;Massachusetts General Hospital | Completed | 18 Years | N/A | All | 26 | Early Phase 1 | United States |
| 117 | NCT00865644 (ClinicalTrials.gov) | March 2009 | 17/3/2009 | Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1 | Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1 | Neurofibromatosis Type 1;Cutaneous Neurofibromas | Drug: Imiquimod 5% Cream | Massachusetts General Hospital | NULL | Completed | 18 Years | N/A | All | 11 | Phase 1 | United States |
| 118 | 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 | Active, not recruiting | 2 Years | 30 Years | Both | 20 | Phase 2 | United States |
| 119 | NCT00727233 (ClinicalTrials.gov) | July 8, 2008 | 31/7/2008 | Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas | Phase I Trial of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib (BAY 43-9006, Nexavar) in Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas | Neurofibromatosis Type I;Plexiform Neurofibroma | Drug: Nexavar (BAY 43-9006) (Sorafenib);Drug: Toxicity, Pharmacokinetics;Drug: Pharmacodynamics;Drug: Radiographic Evaluation;Drug: QOL assessment, Neuropsychological;Drug: Bony Toxicity | National Cancer Institute (NCI) | NULL | Completed | 3 Years | 18 Years | All | 9 | Phase 1 | United States |
| 120 | NCT00634270 (ClinicalTrials.gov) | April 2008 | 20/2/2008 | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1 | Drug: Sirolimus | University of Alabama at Birmingham | Boston Children’s Hospital;Children's Hospital of Philadelphia;Children's Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of Medicine | Completed | 3 Years | 75 Years | All | 58 | Phase 2 | United States |
| 121 | NCT00652990 (ClinicalTrials.gov) | March 2008 | 3/4/2008 | Sirolimus to Treat Plexiform Neurofibromas in Patients With Neurofibromatosis Type I | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1;Plexiform Neurofibromas;Paraspinal Plexiform Neurofibromas | Drug: Sirolimus | University of Alabama at Birmingham | National Cancer Institute (NCI) | Active, not recruiting | 3 Years | N/A | Both | 18 | Phase 2 | United States |
| 122 | NCT00657202 (ClinicalTrials.gov) | March 2008 | 27/3/2008 | Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1 | Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1 | Neurofibromatosis Type 1;Cutaneous Neurofibromas | Drug: Ranibizumab | Massachusetts General Hospital | NULL | Completed | 18 Years | N/A | Both | 11 | Phase 0 | United States |
| 123 | NCT00589784 (ClinicalTrials.gov) | October 2007 | 26/12/2007 | Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma | Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma | CNS Cancer;Meningioma;Intracranial Hemangiopericytoma;Hemangioblastoma;Neurofibromatosis | Drug: Sunitinib | Memorial Sloan Kettering Cancer Center | Dana-Farber Cancer Institute;University of Virginia;University of Pittsburgh;Pfizer | Completed | 18 Years | N/A | All | 50 | Phase 2 | United States |
| 124 | NCT00901849 (ClinicalTrials.gov) | May 2007 | 13/5/2009 | Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1) | Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1) | Low-grade Gliomas | Drug: Tarceva and Rapamycin | Roger Packer | Children's Research Institute | Completed | N/A | 21 Years | Both | 21 | Phase 1 | United States |
| 125 | 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 |
| 126 | NCT01673009 (ClinicalTrials.gov) | May 2006 | 22/8/2012 | Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas | Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas | Neurofibromatosis | Drug: Gleevec | Indiana University | NULL | Completed | 3 Years | 65 Years | All | 36 | Phase 2 | United States |
| 127 | NCT00326872 (ClinicalTrials.gov) | May 2006 | 16/5/2006 | AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine | A Phase II Study of AZD2171 in Adult Patients With Neurofibromatosis Type 1 and Extensive Plexiform and Paraspinal Neurofibromas | Neurofibromatosis Type 1;Plexiform Neurofibroma;Spinal Cord Neurofibroma | Drug: Cediranib Maleate | National Cancer Institute (NCI) | NULL | Terminated | 18 Years | N/A | All | 26 | Phase 2 | United States |
| 128 | NCT00304083 (ClinicalTrials.gov) | December 2005 | 15/3/2006 | Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors | Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated High Grade Malignant Peripheral Nerve Sheath Tumors | Neurofibromatosis Type 1;Sarcoma | Biological: filgrastim;Drug: doxorubicin hydrochloride;Drug: etoposide;Drug: ifosfamide;Procedure: conventional surgery;Radiation: radiation therapy | Sarcoma Alliance for Research through Collaboration | National Cancer Institute (NCI) | Completed | N/A | N/A | All | 48 | Phase 2 | United States;Canada |
| 129 | NCT00076102 (ClinicalTrials.gov) | July 21, 2004 | 13/1/2004 | Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas | Phase II Trial of Pirfenidone in Children, Adolescents, and Young Adults With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas | Neurofibromatosis 1;Neurofibroma, Plexiform | Drug: Pirfenidone | National Cancer Institute (NCI) | NULL | Completed | 3 Years | 21 Years | All | 36 | Phase 2 | United States |
| 130 | NCT00169611 (ClinicalTrials.gov) | January 2004 | 9/9/2005 | NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate | Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo | Neurofibromatosis Type 1 | Drug: methylphenidate | Hospices Civils de Lyon | NULL | Completed | 7 Years | 12 Years | Both | 80 | Phase 4 | France |
| 131 | NCT00053937 (ClinicalTrials.gov) | December 2002 | 5/2/2003 | Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas | Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas | Neurofibromatosis Type 1;Precancerous Condition | Drug: pirfenidone | National Cancer Institute (NCI) | NULL | Completed | 3 Years | 21 Years | Both | Phase 1 | United States | |
| 132 | NCT00060008 (ClinicalTrials.gov) | April 2002 | 6/5/2003 | Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma | Novel Imaging Modalities For Plexiform Neurofibromas | Neurofibromatosis Type 1;Precancerous Condition | Radiation: fludeoxyglucose F 18;Radiation: gadopentetate dimeglumine | Children's Hospital of Philadelphia | NULL | Terminated | N/A | 25 Years | All | 18 | N/A | United States |
| 133 | NCT00021541 (ClinicalTrials.gov) | July 17, 2001 | 19/6/2006 | R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas | A Phase II Randomized, Cross-Over, Double-Blinded, Placebo-Controlled Trial of the Farnesyltransferase Inhibitor R115777 in Pediatric Patients With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas | Neurofibroma, Plexiform;Neurofibromatosis Type I | Drug: tipifarnib;Other: placebo | National Cancer Institute (NCI) | NULL | Completed | 3 Years | 25 Years | All | 62 | Phase 2 | United States;Germany |
| 134 | NCT00030264 (ClinicalTrials.gov) | February 2001 | 14/2/2002 | Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas | Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study | Neurofibromatosis Type 1;Precancerous Condition | Drug: Methotrexate;Drug: Vinblastine | Children's Hospital of Philadelphia | NULL | Completed | N/A | 25 Years | All | 23 | Phase 2 | United States |
| 135 | NCT00754780 (ClinicalTrials.gov) | September 2000 | 16/9/2008 | Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1 | Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I | Neurofibromatosis | Drug: Pirfenidone | Mayo Clinic | NULL | Completed | 18 Years | 70 Years | Both | 24 | Phase 2 | NULL |
| 136 | EUCTR2016-001563-36-Outside-EU/EEA (EUCTR) | 12/05/2016 | Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1) | A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1) | Plexiform Neurofibroma Associated With Neurofibromatosis Type 1;Therapeutic area: Diseases [C] - Cancer [C04] | Trade Name: Votubia Product Name: Everolimus Product Code: RAD001 | Novartis Pharmaceuticals | NULL | NA | Female: yes Male: yes | 26 | Phase 4 | Israel | |||
| 137 | EUCTR2011-002228-42-FR (EUCTR) | 29/08/2011 | N/A | N/A - AFINF2 | MedDRA version: 14.0;Level: PT;Classification code 10029268;Term: Neurofibromatosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Cancer [C04] | Trade Name: Afinitor Product Name: Afinitor INN or Proposed INN: Evérolimus Other descriptive name: RAD001 | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | France |