Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children
NCT06763315, Phase 2, Not yet recruiting, West China Hospital
Trial 10
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
| Field | Value | Source |
|---|---|---|
| Title | Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children | ClinicalTrials.gov |
| NCT number | NCT06763315 | ClinicalTrials.gov |
| Status | Not yet recruiting | ClinicalTrials.gov |
| Phases | Phase 2 | ClinicalTrials.gov |
| Enrollment | 50 participants, estimated | ClinicalTrials.gov |
| Lead sponsor | West China Hospital | ClinicalTrials.gov |
| Conditions | Plexiform Neurofibromas (PN) | ClinicalTrials.gov |
| Interventions | Selumetinib | ClinicalTrials.gov |
| First posted | 2025-01-08 | ClinicalTrials.gov |
| Last updated | 2025-01-08 | ClinicalTrials.gov |
Registered outcomes 9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description | Source |
|---|---|---|---|---|
| Primary | The change in tumor volume of plexiform neurofibromas | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | Treatment response was measured by volumetric magnetic resonance imaging (MRI) analysis performed at the start of treatment and after 3, 6, 9, 12, 18, and 24 cycles of treatment and independently assessed by 2 radiologists. Changes in PN size were categorized as further growth (increase of ≥20%), no change (\<20% increase and \<20% decrease), partial involution (decrease of ≥20% and \<75%), nearly complete involution (decrease of ≥75% and \<100%), or complete involution (100%). | ClinicalTrials.gov |
| Secondary | Frequency of adverse events | Once per cycle in the first 3 cycles, once every 3 cycles in the 4th to 12th cycles, and once every 6 cycles in the last 12 cycles (each cycle is 28 days). | Frequency of adverse events (e.g. gastrointestinal disorders, eye disorders, skin disorders, paronychia, abnormal left ventricular ejection fraction, abnormal myocardial marker, abnormal liver function etc.) collected regularly during follow-up by the investigators and reported by the parents. All adverse events were collected and graded according to Common Terminology Criteria for Adverse Events, version 4.0 (CTCAE v4.0). The causality of the adverse event was determined by the multidisciplinary staff and was classified as definitively not related, probably not related, possibly related, probably related, or definitively related. Any dose reductions, interruptions, or cessations enacted at the discretion of the investigators were recorded. | ClinicalTrials.gov |
| Secondary | Quality of life (QOL) in patients and their families | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | The Pediatric Quality of Life Inventory (PedsQLTM) 4.0 was used. This genetic core scale is a multidimensional child self-report and parent proxy report measure that assesses health-related quality of life (QOL) in children, adolescents, and young adults aged 2 to 25 years. The PedsQL contains 23 global core measures of QOL, including subscales assessing four domains (physical, emotional, social, and school functioning). | ClinicalTrials.gov |
| Secondary | The change of pain intensity | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | The Numerical Rating Scale-11 (NRS-11) was used. This scale is a self-report measure that assesses pain intensity on an 11-point numeric scale. It consists of a horizontal line with 0 on the right end, representing "no pain," and 10 on the left end, representing "the worst pain you can imagine." Children aged 8 to 18 years were asked to circle a number from 0 to 10 that best described their worst pain in the past week, including: 1) self-selected most important tumor pain, 2) physician-selected target tumor pain, 3) overall tumor pain, and 4) other types of pain. | ClinicalTrials.gov |
| Secondary | The change of pain interference | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | The Pain Interference Index (PII) is a six-item measure that assesses the extent to which pain has interfered with daily activities over the past week, including concentration, spending time with friends, leisure and physical activities, mood, and sleep. The scale was slightly adapted after translation into English, and a parallel parent version was developed and validated in parents of children with chronic pain. In this study, children aged 8 to 18 years completed the self-report PII, and parents of children aged 5 to 18 years completed the parent-report PII. The total PII score is the average of all six items. | ClinicalTrials.gov |
| Secondary | The change of physical functioning (mobility and upper extremity) | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | The Patient-Reported Outcome Measurement Information System (PROMIS®) Physical Function Mobility and Upper Extremity 8-item short forms were used. This short form is used to assess the ability of a person to perform a variety of physical functions over the past 7 days, ranging from motor movements (e.g., standing up from the floor) to activities including self-care skills and vigorous exercise. Children aged 8-18 years completed the Pediatric Self-Report Short Form, and parents of children aged 5-18 years completed the parallel Parent Report Form. These PROMIS Physical Functioning Short Forms demonstrated good psychometric properties, including good reliability and construct validity in adolescents with a variety of medical conditions. | ClinicalTrials.gov |
| Secondary | The change of neurocognitive abilities-attention and memory | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles(each cycle is 28 days). | The Continuous Performance Test (CPT) was used. It assesses the subject's attention, response control, and sustained attention ability by presenting continuous visual or auditory stimuli and requiring the subject to respond according to specific rules. The test content usually includes quickly identifying specific stimuli and responding while ignoring other distracting stimuli. Evaluation indicators include reaction time, accuracy of correct responses, frequency of impulsive responses, and the individual's ability to maintain attention during the task, helping to identify attention deficits or response control problems. | ClinicalTrials.gov |
| Secondary | The change of neurocognitive abilities-language ability | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | Peabody Picture Vocabulary Test (PPVT) was used. It is a standardized tool for assessing an individual's vocabulary comprehension ability. It presents a set of pictures and verbal words and asks the subject to choose the picture that best matches the meaning of the word. The test covers words of varying difficulty and assesses an individual's auditory comprehension of words. The main evaluation indicators include language comprehension level, vocabulary, and cognitive ability, which help detect language development, language disorders, and intelligence level. | ClinicalTrials.gov |
| Secondary | The change of neurocognitive abilities-executive function | Once every 3 cycles for the first 12 cycles, once every 6 cycles for the next 12 cycles (each cycle is 28 days). | The Stroop test was used. It assesses an individual's inhibitory control and selective attention. It consists of two tasks: one is to ask the subject to report the font color of a word while ignoring the meaning of the word (color word task), and the other is to simply report the font color (simple color task). The test evaluates an individual's cognitive inhibition ability in conflict situations by comparing the performance of these two tasks. The main evaluation indicators include reaction time, task accuracy, and conflict inhibition ability. | ClinicalTrials.gov |
Outcomes 0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as | Source |
|---|
Results by group 0
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units | Source |
|---|
Participant flow 0
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted | Source |
|---|
Adverse events 0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events | Source |
|---|
Adverse event totals 0
The totals the trial posted for each group.
| Total | Group | Affected | At risk | Source |
|---|
Papers 6
The papers the trial lists on ClinicalTrials.gov.
Grants 0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|
Trial comparisons 0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|
Tests 3
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Continuous Performance Test | Secondary outcome | ConductCognition |
| Stroop Color-Word Test | Secondary outcome | ConductCognition |
| Vocabulary Test | Secondary outcome | ConductCognition |
Source 5
| Field | Value | Source |
|---|---|---|
| Release | trials-2026-11-12-0.4.0 | ClinicalTrials.gov |
| Release manifest SHA-256 | 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4 | ClinicalTrials.gov |
| Registry capture | 2026-11-12 | ClinicalTrials.gov |
| Results copy, AACT | 2026-03-23 | ClinicalTrials.gov |
| NIH clinical studies file | 2026-11-13 | ClinicalTrials.gov |
Release trials-2026-11-12-0.4.0
ClinicalTrials.gov's posted results, through CTTI's AACT: ClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299 ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
ClinicalTrials.gov: none stated ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
NIH RePORTER's clinical studies and project files: Public domain (NIH RePORTER)