Treatment of symptomatic plexiform neurofibromas, a benign tumour associated with the disorder Neurofibromatosis type 1, in children with the drug trametinib

Phase 1

• Conditions: F1-related plexiform neurofibroma; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-001846-32-SE
• Lead Sponsor: VO Barnmedicin, Skånes University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-07-31
• Last Update Posted: 13 November 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 15

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.
•History of heparin-induced thrombocytopenia.
•History of interstitial lung disease or pneumonitis.
•History of or current evidence of retinal vein occlusion (RVO).
•A history of known Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV) infection. Subjects with laboratory evidence of cleared HBV and HCV infection may be enrolled.
•Presence of condition that will interfere significantly with the absorption of drugs.
•Evidence of cardiovascular risk, LVEF below LLN, Qtc >480 millisecond, clinical significant uncontrolled arrhythmia, congestive heart failure, acute coronary syndrome or history thereof.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified