A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF… (NCT04590235) | Clinical Trial Compass
Active — Not RecruitingPhase 1
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
China32 participantsStarted 2020-12-16
Plain-language summary
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).
Who can participate
Age range
3 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Paediatric cohort: Chinese subjects ≥3 years and \<18 years of age
* Adult cohort: Chinese subjects ≥18 years of age at the time of study enrollment
* Subjects must be diagnosed with (i) NF1 as per NIH Consensus Development Conference Statement and（ii) PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. (iii) inoperable PN
* Subjects must have at least one measurable typical or nodular PN
* Absolute neutrophil count ≥1.5×10\^9/L, haemoglobin ≥9g/dL, and platelet count ≥100×10\^9/L. Subject must be without growth factor support and platelet transfusion support 7 days before the screening assessment.
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2×upper limit of normal (ULN), total bilirubin ≤1.5×ULN except in the case of subjects with documented Gilbert's disease (≤2.5×ULN).
Exclusion Criteria:
* Evidence of malignant peripheral nerve sheath tumour.
* Clinically significant cardiovascular disease
* Prior malignancy (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject had been disease free for ≥2 years or which would not have limited survival to \<2 years) or other cancer requiring treatment with chemotherapy or radiation therapy.
* Subjects with the following ophthalmological findings/conditions:
Current or past history of retinal pigment epithelial detachment/central serou…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1 trial focused on how selumetinib behaves in the body — things like drug levels in the blood and how long it stays in the system — what does that mean for what we'd actually learn about whether it helps shrink the tumors, and how does that compare to what's already known from earlier selumetinib studies?
2The trial is no longer recruiting but is still active, so it sounds like participants are already enrolled and being followed — is there any chance we could still discuss this trial as an option, or should we be focusing on other available pathways to access selumetinib?
3The study is specifically designed for Chinese pediatric and adult patients — does my or my child's background, age, and medical history fit the profile of who this trial was designed to study, and does that affect whether the safety and dosing findings would be relevant to us?
4Since adverse events are one of the primary things being measured, what kinds of side effects have been reported with selumetinib in earlier studies, and how would my doctor monitor for and manage those if we were considering this drug?
5Given that this is a Phase 1 study still gathering basic safety and drug-behavior data, would my doctor recommend first exploring whether selumetinib is accessible through an approved indication or expanded access program rather than waiting for results from a study that's already closed to new participants?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse events
Timeframe: For paediatric cohort: from signing the informed consent form until up to 3 years after last subject dosed; For adult cohort: from signing the informed consent form until up to 2 years+30 days after last subject dosed.
2
Area under the concentration-time curve from zero to the last measurable concentration (AUC0-t) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
Timeframe: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
3
Maximum plasma concentration (Cmax) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
Timeframe: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
4
Terminal half-life (t1/2) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
Timeframe: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.