Trial · NCT04924608
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 29
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. | From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days) | Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD. |
| Secondary | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Primary Analysis | Baseline and end of cycle 12 of study intervention | The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening perio, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo in participants with a PAINS-pNF chronic target PN pain score of ≥ 3 at baseline is presented. |
| Secondary | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis | Baseline and end of cycle 12 of study intervention | The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening period, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo participants is presented. |
| Secondary | (Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12 | Baseline and end of Cycle 12 of study intervention | PlexiQoL (Plexiform Neurofibroma Quality of Life scale) is a patient-derived QoL measure specific to adults with NF1-associated PNs. It assesses the impact of PNs on patients' ability to fulfil their human needs. The measure consists of 18 dichotomous items with 0 =Not True and 1 = True. PlexiQoL total scores were calculated by the sum of all items to a maximum of 18, with lower scores indicating better quality of life. The change from baseline to the end of each cycle in PlexiQoL total score was derived as the PlexiQoL total score at the cycle 12 minus baseline PlexiQoL total score and presented. |
| Secondary | Confirmed Partial and Complete Response Rate (ORR) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of study (final DCO) as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD. |
| Secondary | Duration of Response by Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | The duration of response is defined as the time from the date of first documented response (which is subsequently confirmed) until date of documented progression by ICR per REiNS criteria or death due to any cause. Duration of response is derived using while on-treatment MRI volumetric assessments. The percentage of participants with event are provided. |
| Secondary | Progression Free Survival (PFS) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Progression free survival (PFS) is the time from randomisation date until the date of progression by ICR per REiNS criteria or death due to any cause, or last evaluable MRI assessment for subjects that do not progress. For subjects that progress after two or more consecutive missed MRI assessments, the subject is censored at the time of the latest evaluable MRI assessment prior to the missed visits. The percentage of participants with event are provided. |
| Secondary | Time to Progression (TTP) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Time to progression (TTP) is the time from randomisation date until the date of first documented objective disease progression by ICR per REiNS criteria, or last evaluable MRI assessment for subjects that do not progress. For subjects that progress after two or more consecutive missed MRI assessments, the subject is censored at the time of the latest evaluable MRI assessment prior to the missed visits. The percentage of participants with event are provided. |
| Secondary | Time to Response (TTR) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Time to response (TTR) is the time from randomisation date until the date of first documented objective partial and complete response (which is subsequently confirmed, cCR or cPR) as determined by ICR per REiNS criteria. Only participant who have achieved a cCR or a cPR are evaluated for TTR. |
| Secondary | Best Percentage Change From Baseline (PCHG) in Target PN During Randomized Period | From randomisation date until end of cycle 12 of study intervention | The best change in volume is the largest decrease from baseline or the smallest increase from baseline in the absence of a reduction and includes all on-treatment MRI volumetric assessments by the end of Cycle 12 Day 28 visit and prior to the earliest of death in the absence of progression, any evidence of progression, or the last evaluable REiNS assessment if the patient has not died or progressed. |
| Secondary | Proportion of Patients With Chronic Target PN Pain Palliation During the Randomised Period | Baseline and end of cycle 12 of study intervention | Target PN Pain palliation occurs if an improvement of \>= 2 in PAINS-pNF chronic target PN pain intensity score and no increase (\>=1 in chronic PN pain medication score) in chronic PN pain medication are observed. The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening period, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. Chronic PN pain medication was collected through eDiary. |
| Secondary | Time to First Chronic Target PN Pain Palliation (TTPP) in Months During the Randomised Period | From randomisation date to end of cycle 12 of study intervention | Time to chronic target PN pain palliation (TTPP) is defined as time to first chronic target PN pain palliation. The date of the last day of the cycle will be used for calculating the time to event. TTPP is defined as the duration of time in months from randomisation to the date of the last day of the first cycle where chronic target PN pain palliation is achieved during the randomization period (by the end of cycle 12). The percentage of participants with event are provided. And for comparison of treatment group, the hazard ratio and 95% confidence interval based on COX proportional hazard regression model, and p-value based on unstratified log-rank test are provided. |
| Secondary | Percentage of Responders of Decrease in Pain Medication Use From Baseline as Reported Using the eDiary at Cycle 12 | Baseline and end of cycle 12 of study intervention | The eDiary PN pain medications reported daily by the patient will be assigned by medical review to a drug class and analgesic ladder score (0=No analgesia use, 1=Non-opioids use, 2=Neuropathic/other non-typical pain medications use, 3=Weak opioids use, 4=Strong opioids use). In case a combination of one or more drug is reported in a daily diary entry, the strongest drug class and its related daily dose will be selected during the medical adjudication process. The responders of decrease in pain medication use as defined the analgesic ladder score has decreased by \>=1 point. |
| Secondary | The Difference of the Means in the Change From Baseline in PII-pNF Pain Interference Total Score at Cycle 12 Between Selumetinib and Placebo | Baseline and end of cycle 12 of study intervention | The PII-pNF assesses the extent to which PN pain interferes with daily functioning. All items ask the patient to consider the pain related to their PN in the past 7 days. Participants are asked how much their PN pain has made it hard for them to engage in physical activities (e.g., challenging physical activities and self-care), affected their physiological processes (e.g., sleep and energy) or impacted their social-emotional functioning (e.g., enjoyment of activities and mood). Items are rated on a 7-point Likert scale (from 0 = not at all to 6 = completely), and the total score is the mean of the completed items. The mean change from baseline in PII-pNF total score during the randomised period will be calculated using the participant's PII-pNF score at cycle 12 minus the baseline score. |
| Secondary | The Difference of the Means in the Change From Baseline in PROMIS Physical Function Scores at Cycle 12 Between Selumetinib and Placebo | Baseline and end of cycle 12 study intervention | The Adult Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function items measure self-reported capability rather than the actual performance of physical activities. 3 Items from the PROMIS item bank that map to physical function concepts relevant to patients with symptomatic PN: (1). Does your health now limit you in bending, kneeling, or stooping? (2) How much difficulty do you have doing your daily physical activities, because of your health? (3) To what extent are you able to carry out your everyday physical activities such as walking, climbing stairs, carrying groceries, or moving a chair? Items are rated on a 5-point rating scale (1-5, the higher the better). A total score was derived by summing up the responses of the three items (Range 3-15). Changes from baseline (CHG) to the end of each cycle in each item and total scores were derived. |
| Secondary | The Difference of the Means in Change From Baseline in the Skin Sensations Domain From the PedsQL at Cycle 12 Between Selumetinib and Placebo | Baseline and end of cycle 12 study intervention | The adult version of the Paediatric Quality of Life Inventory (PedsQL) NF1 Module (NF1 module acute version 3.0 - adult report) can be used to understand the multidimensional nature of NF1 on the HRQoL patients with this disorder and may assist in medical decision making. Skin Sensations domain (3 items) of PedsQL is used. Items are rated on a 5-point Likert scale from 0 ("Never") to 4 ("Almost always") (Higher value represent worse outcome). Items are reverse-scored and linearly transformed to a 0 to 100 scale as follows: 0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0 (For reversed score, the higher value represent better outcome). Domain scores are calculated by the sum of all the items over the number of items answered if more than 50% of the items. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo is presented. |
| Secondary | The Difference of Means in the Change From Baseline in EQ-5D Index Score at Cycle 12 Between Selumetinib and Placebo | Baseline and the end of cycle 12 of study intervention | The EuroQoL five dimensions, five level health state utility index (EQ-5D-5L) is a generic questionnaire that provides a simple descriptive profile of health and a single index value for health status for economic appraisal (EuroQoL 2019). The EQ-5D-5L questionnaire comprises 6 questions that cover 5 dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) over 5 levels of increasing severity of problems ("no" "slight" "moderate" "severe" and "extreme"). Participants indicate their current health state by selecting the most appropriate level in each of the 5 dimensions. The EQ-5D profile is converted into a weighted health state utility value, termed the EQ-5D index that represents the comparative value of health states (the index score generally ranges from negative to 1.0, the higher the score, the better). The difference of the means in the change from baseline of index score at cycle 12 between selumetinib and placebo is presented. |
| Secondary | The Difference of Means in Change From Baseline in EQ-VAS Score at Cycle 12 Between Selumetinib and Placebo | Baseline and the end of cycle 12 of study intervention | The EuroQoL five dimensions, five level health state utility index (EQ-5D-5L) developed by the EuroQoL Group, is a generic questionnaire that provides a simple descriptive profile of health and a single index value for health status for economic appraisal (EuroQoL 2019). The EQ-5D-5L questionnaire comprises 6 questions that cover 5 dimensions of health and also assess their health today using the EQ-VAS, which ranges from 0 (worst imaginable health) to 100 (best imaginable health). The difference of the means in the change from baseline of EQ-VAS score at cycle 12 between selumetinib and placebo is presented. |
| Secondary | Plasma Concentrations (ng/mL) of Selumetinib - Multiple Dose Cycle 1, Day 8 | Pre-dose and 0.5, 1.5, 3, 8, 8 hours after selumetinib single dose on the 8th day of study treatment (Cycle 1 Day 8). | The geometric mean and geometric coefficient of variance (CV) % of the plasma concentrations (ng/mL) of selumetinib for multiple dose of cycle 1 day 8 are presented. |
| Secondary | Plasma Concentrations (ng/mL) of N-desmethyl Selumetinib - Multiple Dose Cycle 1, Day 8 | Pre-dose and 0.5, 1.5, 3, 6, 8 hours after selumetinib single dose on the 8th day of study treatment (Cycle 1 Day 8). | The geometric mean and geometric coefficient of variance (CV) % of the plasma concentrations (ng/mL) of N-desmethyl selumetinib for multiple dose of cycle 1 day 8 are presented. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.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.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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