EUCTR2018-001846-32-SE
Active, not recruiting
Phase 1
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
VO Barnmedicin, Skånes University Hospital0 sites15 target enrollmentJuly 31, 2018
DrugsMekinist
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- VO Barnmedicin, Skånes University Hospital
- Enrollment
- 15
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •NF1\-related PN with severe \- or with high suspicion of threatening \-
- •manifestations
- •Informed consent given
- •Age 1:0\-17:11
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 150
- •F.1\.2 Adults (18\-64 years) no
- •F.1\.2\.1 Number of subjects for this age range
- •F.1\.3 Elderly (\>\=65 years) no
- •F.1\.3\.1 Number of subjects for this age range
Exclusion Criteria
- •NF1\-related PN does not fulfill characteristics for acceptable volumetric MRI assessments as outlined in box Criterion for volumetric assessment.
- •Lactating or pregnant or sexual active females, who do not use safe contraception. Sexual active males who do not use condom.
- •History of other malignancies than classic NF1\-related WHO grade 1 tumor (i.e. PN or optic pathway glioma).
- •Subjects with a history of NF\-1 related cerebral vascular anomaly (such as Moyamoya).
- •Subjects with NF\-1, receiving pharmaceutical therapy for optic pathway malignancy/ies.
- •Any medication for treatment of left ventricular systolic dysfunction.
- •Administration of an investigational study treatment within 30 days preceding the first dose of the study treatment in this study.
- •Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to the study treatment or excipients that contraindicate their participation.
- •Current active liver or biliary disease
- •History of hepatic sinusoid obstructive syndrome (venoocculsive disease) within the prior 3 months.
Outcomes
Primary Outcomes
Not specified
Similar Trials
Completed
Phase 2
Medical Treatment of "High-Risk" NeurofibromasNeurofibromatosis 1NCT00846430Spectrum Health Hospitals9
Active, not recruiting
Phase 2
Treatment of NF1-related Plexiform Neurofibroma With TrametinibNeurofibromatosis 1ChildNeurofibroma, PlexiformNCT03741101Region Skane15
Terminated
Phase 1
Phase I Clinical Study of the Safety of Photodynamic Therapy (PDT) Using LS11 in Children With Plexiform NeurofibromasNeurofibromaNCT00716469Children's Hospital of Philadelphia7
Recruiting
Not Applicable
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)Neurofibromatosis 1Plexiform NeurofibromasNCT02544022National Cancer Institute (NCI)476
Completed
Not Applicable
Study of Plexiform Neurofibromas in Neurofibromatosis Type 1Plexiform NeurofibromasNF1NCT00006435National Cancer Institute (NCI)24