A Phase III, Multicentre, International Study With a Parallel, Randomised, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

JRCT ID: jRCT2041210036

Registered date:24/06/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Neurofibromatosis 1, Plexiform Neurofibroma (PN)
• Date of first enrollment: 01/07/2022
• Target sample size: 15
• Countries of recruitment: Australia,Japan,Brasil,Japan,Canada,Japan,China,Japan,France,Japan,Germany,Japan,Italy,Japan,Poland,Japan,Russia,Japan,Spain,Japan,USA,Japan,UK,Japan
• Study type: Interventional

Intervention(s)

Arm A: selumetinib 25 mg/m2 orally bid
Arm B: placebo orally bid

Outcome(s)

Primary Outcome

Confirmed Objective Response Rate (ORR) for Arm A [ Time Frame: Approximately 3 years ]
ORR will be defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Adults 18 years or more at enrollment with diagnosis of NF1 with symptomatic, inoperable PN

- At least one inoperable target PN measurable by volumetric MRI analysis

- Chronic target PN pain score documented for minimum period during screening period

- Stable chronic PN pain medication use at enrollment

- Adequate organ and marrow function

Exclude criteria

- Confirmed or suspected malignant glioma or MPNST (optic glioma not requiring chemotherapy or radiation therapy are exempt from this exclusion)

- History of malignancy except for malignancy treated with curative intent with no known active disease 5 years or more before the first dose of study intervention and of low potential risk for recurrence

- Clinically significant cardiovascular disease, including inherited coronary disease, acute coronary syndrome within 6 months prior to enrollment, uncontrolled angina, symptomatic heart failure, cardiomyopathy, severe valvular heart disease, abnormal LVEF and uncontrolled hypertension

- Ophthalmological findings/conditions including intraocular pressure > 21 mmHg, RPED/CSR or RVO

- Prior exposure to MEK inhibitors

Related Information

• Primary Sponsor: Kobayashi Hiroshi
• Secondary ID(s): NCT04924608