Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma (NCT02839720) | Clinical Trial Compass
CompletedPhase 2
Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma
United States11 participantsStarted 2017-08-26
Plain-language summary
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.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients be \>= 18 years old at the time of enrollment and must have a documented germline neurofibromatosis 1 (NF1) mutation in a Clinical Laboratory Improvement Act (CLIA) certified laboratory or a diagnosis of NF1 based on clinical National Institutes of Health (NIH) consensus criteria; in addition to substantial cutaneous neurofibroma burden, at least one of the criteria below have to be present
* Six or more cafe-au-lait macules (\>= 0.5 cm in prepubertal subjects or \>= 1.5 cm in post pubertal subjects)
* Freckling in axilla or groin
* Optic glioma
* Two or more Lisch nodules
* A distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
* A first-degree relative with NF1
* Histologic confirmation of tumor is not necessary in the presence of consistent clinical findings
* Patients must have substantial cutaneous neurofibroma burden causing distress to the patient by disfigurement or itching; patients must have \>= 9 measurable cutaneous neurofibromas; for the purpose of this study measurability will be defined for each of the lesions selected as target lesions as a neurofibroma with a longest diameter \>= 4 mm in the longest diameter
* Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
* Hemoglobin \>= 10 g/dL (not requiring red blood cell \[RBC\] transfusions)
* Absolute neutrophil count \>= 1,500/mcL
* Platelets \>= 100,000/mcL (not requiring platelet transfusions)
* Total…
What they're measuring
1
Median Best Response of Cutaneous Neurofibromas in Participants With at Least One Restaging Evaluation
Timeframe: Up to 24 cycles of treatment (1 cycle = 28 days)