Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumet… (NCT05101148) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
United States, Poland, Russia24 participantsStarted 2021-07-21
Plain-language summary
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed under fed and fasted conditions; and potentially, to confirm an appropriate dosing recommendation of selumetinib with a low fat meal that maintains efficacy with acceptable safety. These results may support labelling statements with regard to posology and food.
Who can participate
Age range
12 Years – 17 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:
* Male and female participants aged ≥ 12 to \< 18 years at the time of signing the informed consent.
* All study participants must be diagnosed with (i) NF1 per NIH Consensus Development Conference Statement and (ii) inoperable PN. In addition to PN, participants must have at least 1 other diagnostic criterion for NF1 as defined in protocol.
* Participants must require treatment for NF1 and inoperable PN due to actual symptoms or because of the potential to develop significant clinical complications, as judged by the Investigator, as defined in the protocol.
* Participants who have had prior treatment with any MEKi (including selumetinib) may be considered for inclusion in this study.
* Participants must have a BSA ≥ 1.3 and ≤ 2.5 m2
Exclusion Criteria:
* Evidence or suspicion of optic glioma, malignant glioma, MPNST, or other cancer requiring treatment with chemotherapy or radiation therapy
* Prior malignancy requiring active treatment (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the participant had been disease free for ≥ 2 years or which would not have limited survival to \< 2 years).
* A life-threatening illness, medical condition, organ system dysfunction of laboratory finding which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of selumetinib, or put the study outcomes at undue risk.
* Participants …
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.
1This is a Phase 1 study focused on how food affects how selumetinib is absorbed and its stomach-related side effects in adolescents with NF1 — does that mean there's still a lot unknown about the right way to take this drug with meals, and how might that uncertainty affect my child's treatment experience?
2The trial is actively enrolling but no longer recruiting new participants — is there any chance my child could still be considered, or are there similar studies or expanded access options we should be looking into instead?
3Since one of the main things being measured is gastrointestinal side effects like nausea, vomiting, and stool changes, how significant have these kinds of stomach issues been for adolescents taking selumetinib so far, and how are they typically managed?
4Given that this study is specifically tracking whether taking selumetinib with or without food changes how the drug behaves in the body, would the findings from this trial potentially change how my child would be instructed to take selumetinib if they were on it outside of a study?
5Before considering a food-effect study like this one, should my child first be evaluated for whether selumetinib as a treatment — regardless of the study — is the right path for their specific plexiform neurofibromas, and what would that evaluation involve?
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
Selumetinib area under the plasma concentration-time curve from zero to 12 hours post-dose (AUC0-12)
Timeframe: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
2
Gastrointestinal Adverse Events graded by CTCAE Ver 5.0 (Grade 1 to 5)
Timeframe: from screening until 30 days after last dose
3
Assessing change of Gastrointestinal toxicity diary: Modified Bristol Stool Form Scale for Children (mBSFS-C)
Timeframe: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
4
Assessing change of Gastrointestinal toxicity diary: Nausea and Vomiting Symptom Rating Scale (adapted from the Children's Cancer and Leukaemia Group)
Timeframe: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
5
Number of patients who take each gastrointestinal medication
Timeframe: From screening until 30 days after last dose
6
Proportion of patients who take each gastrointestinal medication