EU Clinical Trial Information System (CTIS)

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.

I searched for some studies on AstraZeneca Clinical Trials, and found these studies that may also be of interest to you.
Here is the link to my search results

Official Title

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.
All request will be evaluated as per the AZ disclosure commitment:
https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

D134BC00001 (KOMET) Primary Analysis CSR Synopsis-30Jul25_Redacted-PDF-A.pdf

A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.

Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas

This is a randomized, double-blind, placebo-controlled, 2 arm multicentre, global Phase III study to assess the efficacy and safety of selumetinib compared with placebo in adult participants with NF1 who have symptomatic, inoperable PN.

CTIS Number

EudraCT Number

NCT ID

Patient Level Data

Therapeutic Area

Selumetinib oral capsules (10 mg and 25  mg)

Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Research Site

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 (KOMET)

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

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 characteristics were collected at screening period.

Baseline PAINS-pNF chronic target PN pain intensity score

Study Specific Characteristic

Baseline characteristics were collected at screening period

Age Continuous

Height

Weight

Baseline Body Surface Area. Baseline characteristics were collected at screening period

Geographical region

Sex: Female, Male

Race

Baseline characteristic were collected at screening period

Ethnicity (NIH/OMB)

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.

Full analysis set - All patients who are randomized to study intervention

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.

LS Mean and Standard Error

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.

Pain full analysis set - subjects randomised to study intervention with a baseline PAINS-pNF chronic target PN pain intensity score >= 3.

(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

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 participants is presented.

supplementary analysis is not controlled for multiplicity.

Full analysis set - subjects randomised to study intervention

(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

LS mean and Standard Error

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.

(Second key secondary endpoint) The difference of the means in the change from baseline in PlexiQoL total score at Cycle 12

Overall Study

Global Clinical Lead

Difference in mean change from baseline in chronic target PN pain intensity score between Arm A and Arm B, obtained using an NRS-11 scale to assess pain intensity of a target plexiform neurofibroma

DoR will be defined as the time from the date of first documented response (which is subsequently confirmed) until progression by ICR per REiNS criteria or death due to any cause

PFS will be defined as the time from first selumetinib dose until date of disease progression by ICR per REiNS criteria or death due to any cause

TTP is defined as the time from the date of first selumetinib dose until date of disease progression by ICR per REiNS criteria

TTR is defined as the time from date of first selumetinib dose until the date of objective response by ICR per REiNS criteria

Difference in best percentage change from baseline in target PN volume by ICR per REiNS criteria

Difference in change from baseline between Arm A and Arm B

Arms	Assigned Interventions
Experimental: Arm A Selumetinib	Drug: Selumetinib Selumetinib oral capsules (10 mg and 25 mg) Other Name: AZD6244
Placebo Comparator: Arm B Placebo	Other: Placebo Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas - KOMET

Official Title

Medical condition

Phase

Healthy volunteers

Study drug

Sex

Actual Enrollment

Study type

Age

Date

Study design

Verification:

Sponsors

Collaborators

Inclusion Criteria

Exclusion Criteria

Documents available

Redacted Protocol

Redacted Statistical Analysis Plan

Redacted CSR synopsis

Trial Results Summaries

Contacts

Contact

Investigators

Principal Investigator