Trial · NCT04589650
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 23
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Participants Randomized to Alpelisib With a Confirmed Objective Response by BIRC in Group 1 and Group 2 | Up to 48 weeks | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC), provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non target lesions and without new lesions. Confirmation of response requires a subsequent imaging assessment performed at least 4 weeks after the onset of response. Participants who permanently discontinued alpelisib prior to confirmation of response, and participants who received surgery as rescue therapy prior to confirmation of response are considered as non-responders. |
| Secondary | Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Week 16 | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC) at Week 16, provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non-target lesions and without new lesions. Participants who permanently discontinued alpelisib prior to Week 16, participants who received surgery as rescue therapy prior to Week 16, and participants who had a missing/non-evaluable radiological assessment at Week 16 are considered as non-responders. |
| Secondary | Proportion of Participants With a Response at Week 24 (by BIRC) in Groups 1 and 2 | Week 24 | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC) at Week 24, provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non-target lesions and without new lesions. |
| Secondary | Frequency and Severity of Adverse Events in Groups 1 and 2 up to Week 16 | Up to Week 16 | An adverse event (AE) is any untoward medical occurrence (e.g., any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a participant or clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product. This includes events reported by the participant (or, when appropriate, by a caregiver, surrogate, or the participant's legally authorized representative). Treatment emergent Adverse Event (TEAEs) in this study are events that started after the first dose of study treatment and until 30 days after the last dose of study treatment, or events present prior to the first dose of treatment which increased in severity based on preferred term within 30 days after the last study treatment. |
| Secondary | Frequency and Severity of Adverse Events in All Groups of Participants Over Time | Up to approximately 5 years | Type, frequency, seriousness, and severity of treatment-emergent adverse events per CTCAE v4.03 criteria in participants with PROS over time. |
| Secondary | Change From Baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain Intensity in Group 1 and 2 | Baseline, Week 4, Week 8, Week 12, Week 16 | For adult and pediatric patients 12 years of age and older, the BPI item that assesses worst pain intensity in the past 24 hours was used. Patients respond to the item on an 11-point response scale ranging from 0 (no pain) to 10 (pain as bad as you can imagine). Worst pain intensity was averaged weekly over a 7-day period if a patient had completed the questionnaire for at least 4 days in the 7-day period. The weekly mean was calculated based on the available assessments. Clinically important change at Week 16 was defined as a 2-point reduction for patients who had a pain intensity score ≥ 4 at baseline. For patients with a baseline score \< 4, a 1-point reduction from baseline was also considered as a clinically important change. |
| Secondary | Number of Participants With Global Impression of Symptom Severity (PGIS) Score up to Week 16 | Week 4, Week 8, Week 16 | A Patient Global Impression of Symptom Severity item was used to understand the overall severity of symptoms experienced and clinical meaningfulness of treatment effects experienced during this study. This item included 5 response options: no symptoms, mild, moderate, severe, and very severe. |
| Secondary | Percentage Change From Baseline in Target and MRI-measurable Non- Target Lesion Volume in Group 1 and Group 2 | From Baseline up to approximately 5 years | Percentage change from baseline in the sum of target lesion volume, MRI-measurable non-target lesion volume and all MRI measurable (target an non-target) lesion volume as assessed by BIRC. Target lesions are defined as anatomically reproducibly defined tissue(s) masses, which may be composed of one or several tissue types, and can be accurately measured by imaging technique MRI. Target lesion(s) (up to 3) should be identified at screening, be at least 2 cm in the longest diameter at baseline (for each selected lesion) and may be further reproducibly assessed by MRI. MRI-measurable non-target lesions are defined as all anatomic lesions other than selected as target and may be measured at radiologic assessment (at least 2 cm in the longest diameter at baseline, the volume may be further reproducibly assessed by MRI). |
| Secondary | Proportion of Participants With Changes From Baseline in Other Non-target Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years | Percentage change from baseline in other non-target lesions (by BIRC). Non Target lesions are defined as: * Anatomic lesions, limb/trunkal areas affected by PROS, organomegaly when they may be measured only by caliper/ruler (e.g., circumference of changed limb or body part) * Truly non-measurable lesions (e.g., small lesions less than 2 cm on MRI, superficial visual lesions, masses, organomegaly, PROS-related enlargement of anatomic area identified by physical exam that is not measurable by reproducible imaging technique) |
| Secondary | Proportion of Participants With New Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years | The proportion of patients with new lesions (as assessed by BIRC) will be assessed throughout the study. |
| Secondary | Pharmacokinetics (PK) of Alpelisib in Group 1 and Group 2: Maximum Concentration (Cmax) | Week 17 Day 1 (Pre-dose, 1h post dose, 3h post dose, 5h post dose, 8h post dose , 24h post dose/ Pre-dose of Day 2), Week 20 Day 1 (Pre-dose, 3h post dose) and after Week 28, on Day 1 (Pre-dose and 3h post dose) 4 weeks after the first dose escalation | Maximum concentration of alpelisib following drug administration will be assessed for Group 1 and Group 2. After Week 28, blood samples will be collected only for participants who had dose escalation at next scheduled visit 4 weeks after the first dose escalation. |
| Secondary | Pharmacokinetics (PK) of Alpelisib in Group 1 and Group 2: Trough Concentration (Ctrough) | Week 17 Day 1 (Pre-dose and 24 h post dose/ Pre-dose of Day 2), Week 20 Day 1 (Pre-dose) and after Week 28, on Day 1 (Pre-dose) 4 weeks after the first dose escalation | The trough observed concentration of alpelisib will be assessed for Group 1 and Group 2. After Week 28, blood samples will be collected only for participants who had dose escalation at next scheduled visit 4 weeks after the first dose escalation. |
| Secondary | Change From Baseline in Patient-reported Pain Assessed by Brief Pain Inventory (BPI) Worst Pain Intensity Item or Wong-Baker Faces Scale (Age Appropriate) in Pediatric and Adult Populations | From Baseline up to approximately 5 years | Change in scores from Brief Pain Inventory (BPI) items, or Wong-Baker Faces Scale (age appropriate). The BPI item that assesses worst pain intensity in the past 24 hours will be used to assess pain intensity for adult participants (≥18 years old) and pediatric participants (≥12 years old). Participants respond to the item on an 11-point numerical rating scale ranging from 0 (no pain) to 10 (pain as bad as you can imagine). For children under 12, the Wong-Baker Faces Scale will be used in place of the BPI worst pain intensity item. This scale is a single-item that includes drawings of 6 faces that are associated with both a numeric rating and a descriptor (ranging from 0/no hurt - 10/hurts worst) |
| Secondary | Changes From Baseline in Patient-reported Health-related Quality of Life Assessed by PROMIS-profile (Patient Reported Outcome Measurement Information System) in Pediatric and Adult Populations | From Baseline up to approximately 5 years | Change in scores from the PROMIS-profile (Patient Reported Outcome Measurement Information System). The PROMIS Profiles are a group of PROMIS short forms measuring different domains of health-related quality of life (physical function, fatigue, ability to participate in social/peer relationships, pain interference, pain severity, anxiety, depression and sleep disturbance). All items include 5 response options, except for the pain intensity item, which has 11 response options. |
| Secondary | Changes From Baseline in Patient-reported Overall Impression of Symptoms Assessed by Patient Global Impression of Symptom Severity (PGIS) in Pediatric and Adult Populations | From Baseline up to approximately 5 years | Change in PGIS item. The PGIS is a single item to assess the participant's perception in the severity of their symptoms using a 5-point verbal rating scale, from "No symptoms" to "Very Severe". |
| Secondary | Duration of Response (DOR) in Participants Who Received Alpelisib in Group 1 and Group 2 | From first documented response until progression of PROS lesions or death, assessed up to approximately 5 years | Duration of response (DOR) is defined as the time from first documented response until progression of PROS lesions by BIRC or death. |
| Secondary | Time to Treatment Failure in Participants Who Received Alpelisib in Group 1 and Group 2 | From Baseline up to approximately 5 years | Time from randomization/alpelisib treatment start date until the discontinuation of study treatment due to lack of efficacy (including unsatisfactory therapeutic effect, disease progression) or safety reasons (including adverse events, death). Participants who complete the study or discontinue study treatment for other reasons (e.g. discontinuation due to Participant/Guardian decision, technical problems) will be censored at the date of last study treatment received. |
| Secondary | Overall Clinical Response Rate as Assessed by Investigator in Participants Who Received Alpelisib in Group 1 and Group 2 | Week 16, 24, 40, 48, 72, 96 and thereafter every 48 weeks | Proportion of participants with overall clinical response reported as improvement, stable or worsening of clinical condition, as assessed by the investigator |
| Secondary | Proportion of Participants With Response During the Extension Period in Group 1 and Group 2 | Week 40, 48, 72, 96, 144, 192, 240 and 264. | Response (yes/no) at scheduled protocol visit. Response is defined by achieving at least 20% reduction from baseline in the sum of target lesion volumes (1 to 3 lesions, assessed by MRI by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥ 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. |
| Secondary | Changes in Symptoms and Complications/Comorbidities up to Week 16 on Treatment With Alpelisib as Compared to Placebo in Group 1 and Group 2 | Baseline up to Week 16 | Change in PROS-related symptoms and complications/comorbidities associated with PROS up to Week 16 among participants with symptoms and complications/comorbidities present at baseline |
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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