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Kinase Inhibitor

Selumetinib Granules for Neurofibromatosis (SPRINKLE Trial)

Q: 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.

Q: 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.

Q: 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.

Q: 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.

Q: 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 <a href="https://www.withpower.com/clinical-trials/nf1">NF1</a> 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

Phase 1 & 2

Recruiting

Research Sponsored by AstraZeneca

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male and female participants aged ≥ 1 to < 7 years of age at the time their legally authorised representative (parent or guardian) signs the informed consent

Participants must have a BSA ≥ 0.4 and ≤ 1.09 m2 at study entry (date of ICF signature)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 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).

Awards & highlights

SPRINKLE Trial Summary

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.

Who is the study for?

This trial is for children aged 1 to less than 7 with Neurofibromatosis Type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). They must have a body surface area within specified limits and at least one measurable PN. Children who've had incomplete surgery for PN are eligible if the remaining PN can be measured. Those with certain renal, liver, or hematological issues, previous MEKi treatment complications, or inability to undergo MRI are excluded.Check my eligibility

What is being tested?

The study tests Selumetinib granule formulation versus capsule formulation in young children with NF1-related PNs. It aims to determine appropriate dosing and assess how the drug moves through and affects the body (pharmacokinetics), its safety profile, as well as preliminary effectiveness of reducing symptoms related to these tumors.See study design

What are the potential side effects?

While specific side effects for this age group aren't listed here, Selumetinib may generally cause skin rash, diarrhea, fatigue, nausea/vomiting among others. Side effects could vary based on individual reactions and dosage received during the trial.

SPRINKLE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am a parent or guardian of a child aged 1 to less than 7 years.

Select...

My body surface area is between 0.4 and 1.09 square meters.

Select...

I can do most activities, but may need help, or I use a wheelchair/breathing support but can still do some activities.

Select...

I have a tumor that is at least 3 cm large, visible on scans, and not fully removed by surgery.

Select...

I have NF1 with a tumor that can't be removed by surgery.

SPRINKLE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 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).

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~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).

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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). for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Adverse Events graded by CTCAE Ver 5.0

Selumetinib AUC0-12 derived after single dose administration

Secondary outcome measures

N-desmethyl selumetinib AUC0 12 derived after single dose administration

Objective Response Rate

Palatability using the parent-reported observer palatability questionnaire

+16 more

Other outcome measures

Selumetinib and N-desmethyl selumetinib AUC0-12 after multiple dose administration

Selumetinib and N-desmethyl selumetinib AUC0-6 after multiple dose administration

Selumetinib and N-desmethyl selumetinib AUClast after multiple dose administration

+26 more

SPRINKLE Trial Design

1Treatment groups

Experimental Treatment

Group I: Selumetinib single armExperimental Treatment2 Interventions

This study consists of a screening period (up to 28 days), a treatment period (25 cycles) and a long term safety follow-up for participants until they are 5 years old or commence an alternative systemic NF1-PN treatment, whichever is the earlier. Participants may continue treatment with selumetinib throughout the long term safety follow-up as long as they are considered to be receiving clinical benefit in the opinion of their Investigator. A safety follow up assessment will be performed 30 days after the last dose of study intervention for all study participants.

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Merck Sharp & Dohme LLCIndustry Sponsor

3,892 Previous Clinical Trials

5,060,627 Total Patients Enrolled

AstraZenecaLead Sponsor

4,271 Previous Clinical Trials

288,613,316 Total Patients Enrolled

Study physician Study physician, MDStudy DirectorAstraZeneca

1 Previous Clinical Trials

24 Total Patients Enrolled

Media Library

Selumetinib (Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05309668 — Phase 1 & 2

Neurofibromatosis Research Study Groups: Selumetinib single arm

Neurofibromatosis Clinical Trial 2023: Selumetinib Highlights & Side Effects. Trial Name: NCT05309668 — Phase 1 & 2

Selumetinib (Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05309668 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

2022. "Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05309668.

All > Nf 1 > Neurofibromatosis