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 | 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 |
| 2 | 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 |
| 3 | 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 | ||
| 4 | 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 |
| 5 | 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 | ||
| 6 | 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 |
| 7 | 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 |