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Selumetinib granule formulation for Neurofibromatosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
National Center for Child Health and Development, Setagaya-ku, JapanNeurofibromatosisSelumetinib granule formulation - Drug
Eligibility
1 - 6
All Sexes
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Study 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.

Eligible Conditions
  • Neurofibromatosis Type 1

Treatment Effectiveness

Effectiveness Progress

1 of 3

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1
AC-OLE-01-VA
1
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1
NMS-03305293

Study Objectives

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).

Week 97
Objective Response Rate
Day 7
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
Serum albumin, bovine
Selumetinib and N-desmethyl selumetinib BSA normalised AUC0-6 after multiple dose administration
Selumetinib and N-desmethyl selumetinib BSA normalised AUClast after multiple dose administration
Serum albumin, bovine
Selumetinib and N-desmethyl selumetinib CL/F after multiple dose administration
Selumetinib and N-desmethyl selumetinib Cmax after multiple dose administration
Selumetinib and N-desmethyl selumetinib Vss/F after multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUC0-12 after multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUC0-6 after multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUClast after multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised Cmax after multiple dose administration
Selumetinib and N-desmethyl selumetinib parent to metabolite ratio for AUC0-12 after multiple dose administration
Selumetinib and N-desmethyl selumetinib parent to metabolite ratio for AUC0-6 after multiple dose administration
Selumetinib and N-desmethyl selumetinib parent to metabolite ratio for Cmax after multiple dose administration
Selumetinib and N-desmethyl selumetinib tlast after multiple dose administration
Selumetinib and N-desmethyl selumetinib tmax after multiple dose administration
Week 25
Palatability using the parent-reported observer palatability questionnaire
Day 28
Selumetinib and N-desmethyl selumetinib AUC0-6 derived after single and multiple dose administration
Day 28
N-desmethyl selumetinib AUC0 12 derived after single dose administration
Selumetinib AUC0-12 derived after single dose administration
Day 28
Parent-to-metabolite ratio for AUC after single and multiple dose administration.
Day 28
Selumetinib and N-desmethyl selumetinib AUClast derived after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib Cmax derived after single and multiple dose administration
Day 28
Parent to metabolite ratio for Cmax after single and multiple dose administration.
Selumetinib and N-desmethyl selumetinib Rac AUC derived after multiple dose administration
Selumetinib and N-desmethyl selumetinib Rac Cmax derived after multiple dose administration
Selumetinib and N-desmethyl selumetinib tlast derived after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib tmax derived after single and multiple dose administration
Day 28
Selumetinib AUC0-24 derived after single dose administration
Selumetinib CL/F derived after single dose administration
Selumetinib Vz/F derived after single dose administration
Selumetinib t1/2 derived after single dose administration
Day 1
Serum albumin, bovine
Selumetinib and N-desmethyl selumetinib dose normalised AUC0-24 after single dose administration
Week 5
Selumetinib and N-desmethyl selumetinib BSA normalised AUC0-12 after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib BSA normalised AUC0-6 after single and multiple dose administration
Serum albumin, bovine
Selumetinib and N-desmethyl selumetinib BSA normalised Cmax after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUC0-12 after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUC0-6 after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised AUClast after single and multiple dose administration
Selumetinib and N-desmethyl selumetinib dose normalised Cmax after single and multiple dose administration
Day 28
Selumetinib and N-desmethyl selumetinib AUC0-12 derived after multiple dose administration
Day 28
Selumetinib CL/F derived after multiple dose administration
Selumetinib Vss/F derived after multiple dose administration
Day 30
Adverse Events graded by CTCAE Ver 5.0

Trial Safety

Safety Progress

1 of 3

Similar Trials

1
AC-OLE-01-VA
1
NMS-03597812
1
NMS-03305293

Trial Design

1 Treatment Group

Selumetinib single arm
1 of 1

Experimental Treatment

44 Total Participants · 1 Treatment Group

Primary Treatment: Selumetinib granule formulation · No Placebo Group · Phase 1 & 2

Selumetinib single armExperimental Group · 2 Interventions: Selumetinib granule formulation, Selumetinib capsule formulation · Intervention Types: Drug, Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 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).

Who is running the clinical trial?

AstraZenecaLead Sponsor
4,018 Previous Clinical Trials
240,376,870 Total Patients Enrolled
Merck Sharp & Dohme LLCIndustry Sponsor
3,708 Previous Clinical Trials
4,967,276 Total Patients Enrolled
Study physician Study physician, MDStudy DirectorAstraZeneca
1 Previous Clinical Trials
24 Total Patients Enrolled

Eligibility Criteria

Age 1 - 6 · All Participants · 6 Total Inclusion Criteria

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References

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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