18 and older, any sex, with Cutaneous Neurofibroma or Neurofibromatosis Type 1. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Median Best Response of Cutaneous Neurofibromas in Participants With at Least One Restaging EvaluationPrimary· Up to 24 cycles of treatment (1 cycle = 28 days)
Average percent change in volume of target cutaneous neurofibromas from baseline. Cutaneous neurofibromas measured with calipers and volumes calculated by multiplying length, width and height of each target neurofibroma. At each response evaluation (baseline and then after every 4 cycles), the sum of the on-treatment volumes for the target cutaneous neurofibromas were subtracted from the pre-treatment volumes of the same tumors to arrive at an overall percentage change in target cutaneous neurofibroma size for each participant at each restaging evaluation. The average percent change that was m
Group
Value
95% CI
Treatment (Selumetinib)
-28.5
-84.9 – 24.8
Change in the Number of Cutaneous NeurofibromasSecondary· Baseline to up to 1 year
At the time of each response evaluation (baseline and then after every 4 cycles), the number of cutaneous neurofibromas (cNFs) that are greater than 4mm as measured with a caliper were counted in the picture frames. We report here the change in overall number of cutaneous neurofibromas counted after 12 cycles of treatment (Number of cNFs counted after 12 cycles - Number of cNFs counted at baseline).
Group
Value
95% CI
Treatment (Selumetinib)
-7
-30 – 4
Changes in Skin Related MorbiditySecondary· Baseline to up to 1 year
Assessed by Skindex. Global Impression of Change Scale and Numeric Rating Scale evaluations were not used in this outcome measure due to limited data set. Skindex is a dermatology survey whose scores are normalized to a 0-100 scale with higher scores indicating worse quality of life. We report here the change in overall Skindex score after 12 cycles of treatment (Score at 12 cycles - Score at Baseline).
Group
Value
95% CI
Treatment (Selumetinib Sulfate)
-1.72
-15.52 – 12.07
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline through study completion, a maximum of 36 cycles (approximately 3 years).
Reporting threshold: 1%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Treatment (Selumetinib)
Serious: 5/11 (45%)
Deaths: 0/11
Serious adverse events (6 terms)
Reaction
System
Treatment (Selumetinib)
Eye disorders - Other, specify
Eye disorders
—
Immune system disorders - Other, specify
Immune system disorders
—
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Tumor hemorrhage
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Hypertension
Vascular disorders
—
Hypertension
Vascular disorders
—
Other adverse events (41 terms — click to expand)
Reaction
System
Treatment (Selumetinib)
Rash acneiform
Skin and subcutaneous tissue disorders
—
Dry skin
Skin and subcutaneous tissue disorders
—
Rash acneiform
Skin and subcutaneous tissue disorders
—
Diarrhea
Gastrointestinal disorders
—
Edema limbs
General disorders
—
CPK increased
Investigations
—
Constipation
Gastrointestinal disorders
—
Nausea
Gastrointestinal disorders
—
Fatigue
General disorders
—
Alanine aminotransferase increased
Infections and infestations
—
Aspartate aminotransferase increased
Infections and infestations
—
Lipase increased
Investigations
—
Skin and subcutaneous tissue disorders - Other, specify (Onchomycosis)
This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04142398 — Incidence of HPV Infection and HPV-Associated Disease in Screening Indian Men Who Have Sex With HIV-Positive Men
· withdrawn
NCT04142385 — Incidence of HIV Infection in Screening Indian Men Who Have Sex With Men
· withdrawn
NCT05558904 — An Investigational Scan (Me-4FDG PET/CT) for the Detection of Sodium-Glucose Transport for Early Diagnosis of Lung Cance
· Phase 1, PHASE2
· recruiting
NCT05564650 — Navitoclax in Relapsed or Refractory High-Risk Myelodysplastic Syndrome
· Phase 1
· active not recruiting
NCT05396859 — Entrectinib in Combination With ASTX727 for the Treatment of Relapsed/Refractory TP53 Mutated Acute Myeloid Leukemia
· Phase 1
· active not recruiting
Other recruiting trials for Cutaneous Neurofibroma
Currently open trials in the same condition.
NCT06159166 — Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
· Phase 1, PHASE2
· recruiting
NCT05581511 — Natural History Study of Cutaneous Neurofibromas in People With NF1
· active not recruiting
Other National Cancer Institute (NCI) trials
Trials by the same sponsor.
NCT07147231 — Testing the Effectiveness of the Anti-cancer Drug Pidnarulex (CX-5461), in Combination With Another Anti-cancer Drug Cem
· Phase 1, PHASE2
· recruiting
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07281417 — Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasa
· Phase 2
· recruiting
NCT07012044 — A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After
· Phase 1
· not yet recruiting
NCT07437950 — Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A Myel
· Phase 2
· not yet recruiting
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 National Cancer Institute (NCI)
Last refreshed: 6 December 2023
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/NCT02839720.