This trial will help determine how well a new granule formulation of a drug works in children with NF1 related symptomatic, inoperable PN, as well as how safe it is.
- Neurofibromatosis Type 1
2 Primary · 19 Secondary · Reporting Duration: Pre-dose and 1, 2, 3, 4, 6, 8, 10-12, and 18-24 hours post-dose after selumetinib single dose on the first day of treatment (Cycle 1 Day 1). Pre-dose and 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on week 5 (Cycle 2, Day 1, each cycle is 28 days).
1 Treatment Group
Selumetinib single arm
1 of 1
44 Total Participants · 1 Treatment Group
Primary Treatment: Selumetinib granule formulation · No Placebo Group · Phase 1 & 2
Who is running the clinical trial?
Age 1 - 6 · All Participants · 6 Total Inclusion CriteriaMark “Yes” if the following statements are true for you:
Frequently Asked Questions
Are there different research facilities conducting this investigation in town?
"At the moment, there are six sites conducting this study. They can be found in Houston, Phoenix and Philadelphia as well as three other locations. If you participate, choosing a site near to your location will help reduce travel burdens." - Anonymous Online Contributor
How many people are being treated with this new medication?
"This is an active clinical trial that was first posted on 1/21/2022 and is looking for 38 patients at 6 different sites." - Anonymous Online Contributor
Are new participants being welcomed into this research program?
"Indeed, the data available on clinicaltrials.gov illustrates that this trial is recruiting patients as we speak. The trial was first posted on 1/21/2022 and has since been updated on 10/24/2022. Presently, the study is looking for 38 individuals who are willing to participate at 6 different sites." - Anonymous Online Contributor
Are any patients older than 25 years old being accepted into this trial?
"According to the entrance requirements for this research, children as young as 1 year old are eligible to participate with a maximum age of 6 years." - Anonymous Online Contributor
Who is most likely to respond well to this treatment option?
"This particular clinical trial is seeking 38 individuals that have watson syndrome and are aged 1 year to 6 years old. It's important to note that candidates must meet the following requirements: Be either male or female, be between the ages of 1-7 years old when their legal guardian signs the informed consent, have a diagnosis of NF1 with symptomatic inoperable PN, have at least one measurable PN , have a Lansky performance score of ≥ 70 (unless they're wheelchair bound or need mechanical breathing support) , have a BSA ≥ 0.4 and ≤ 1.09 m2 . Lastly, the participant or their" - Anonymous Online Contributor