Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
← catalyst calendarSponsored by AstraZeneca (industry) · AZN — their whole pipeline →. With Merck Sharp & Dohme LLC.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
145actual
Sites
34
Countries
Australia, Brazil, Canada +10
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2021-11-19 | actual | When the study began enrolling |
| Primary completion | 2024-08-05 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2029-02-15 | estimated | The whole study's end, after follow-up |
| First posted | 2021-06-14 | actual | When this record first appeared on the registry |
| Results posted | 2025-10-20 | actual | When the sponsor posted results to the registry |
| Record updated | 2026-03-23 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Neurofibromatosis 1
- Plexiform Neurofibroma (PN)
Interventions
- Drug: Selumetinib — also filed as AZD6244
- Other: Placebo
Primary outcome
what the primary-completion date above is the date OFConfirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN.
measured From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days)
Posted documents
hosted by the registry — we link, never re-serveStudy Protocolposted 2023-11-03 · 6.1 MB · Prot_000.pdf
Statistical Analysis Planposted 2024-08-30 · 1.9 MB · SAP_001.pdf
Publications
- PMID 40473450 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
Permalink · AZN's whole pipeline · Catalyst calendar · Every registered study · What changed