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NCT04590235
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
Phase 1 trial testing Selumetinib in Neurofibromatosis 1 in 32 participants. Participants enrolled and being followed up; not accepting new ones.
16 August 2022
Quick facts
| Lead sponsor | AstraZeneca |
|---|---|
| Phase | Phase 1 |
| Status | Active, enrolled |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 32 |
| Start date | 16 December 2020 |
| Primary completion | 16 August 2022 |
| Estimated completion | 31 August 2026 |
| Sites | 2 locations across China |
Drugs / interventions tested
- Selumetinib — full drug profile →
Conditions studied
- Neurofibromatosis 1 — all drugs for Neurofibromatosis 1 →
- Neurofibroma Plexiform — all drugs for Neurofibroma Plexiform →
Sponsor
AstraZeneca — full company profile →
Who can join
Adults 3 to 99, any sex, with Neurofibromatosis 1 or Neurofibroma Plexiform. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN).
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Treatment decisions and the use of MEK inhibitors for children with neurofibromatosis type 1-related plexiform neurofibromas.
Armstrong AE, Belzberg AJ, Crawford JR, Hirbe AC, et al · · 2023 · cited 46× · PMID 37328781 · DOI 10.1186/s12885-023-10996-y -
Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review.
Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, et al · · 2021 · cited 35× · PMID 34388689 · DOI 10.1016/j.esmoop.2021.100223 -
Real-world outcomes for selumetinib in pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas in Japan: A 1-year interim analysis.
Nishida Y, Kitajima A, Ishii T. · · 2026 · PMID 41847168 · DOI 10.1093/noajnl/vdag042
Verify or expand the search:
- PubMed search for NCT04590235
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Selumetinib
Trials testing the same drug.
- NCT06735820 — Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST · Phase 1, PHASE2 · not yet recruiting
- NCT06188741 — Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 · Phase 2 · recruiting
- NCT06763315 — Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children · Phase 2 · not yet recruiting
- NCT06621082 — The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule · Phase 2 · not yet recruiting
- NCT06620354 — Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule · Phase 2 · not yet recruiting
Other recruiting trials for Neurofibromatosis 1
Currently open trials in the same condition.
- NCT07233408 — Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy · NA · recruiting
- NCT06188741 — Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 · Phase 2 · recruiting
- NCT06222203 — Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST · recruiting
- NCT06360406 — Real-World Treatment Study of Koselugo (Selumetinib) · recruiting
- NCT05361811 — Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and F · NA · recruiting
Other AstraZeneca trials
Trials by the same sponsor.
- NCT06998095 — Tezepelumab (Tezspire) Regulatory Postmarketing Surveillance in Korea · not yet recruiting
- NCT07431775 — Saphnelo Use in Females of Child-bearing Potential · not yet recruiting
- NCT07516184 — Explore the Diagnostic Value of Bronchodilation Test With Portable Oscillometry in Asthma Diagnosis · NA · not yet recruiting
- NCT07279935 — Osimertinib Combined With Chemotherapy in Patients Who Had Distant Recurrence After Adjuvant Osimertinib for EGFRm Resec · Phase 4 · not yet recruiting
- NCT07279948 — A Single-arm Observational Study to Characterize the Demographic, Clinical Features and Outcomes of a Brazilian Cohort o · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04590235 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by AstraZeneca
- Last refreshed: 30 January 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04590235.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing