Trial · NCT06997588
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 19 of 19
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of participants with a confirmed objective response by BIRC | Up to Week 48 | Confirmed objective response is defined as achieving radiological response, confirmed by a subsequent assessment performed at least after 4 weeks. The achievement of radiological response requires ≥20% reduction from baseline in the sum of target lesion volumes (1 to 3 target lesions, assessed by Magnetic Resonance Imaging (MRI) by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥20% increase from nadir, and in absence of progression of non-target lesions and without new lesions. |
| Secondary | Proportion of participants with a confirmed objective response by BIRC in a protocol-defined subset of patients | Up to Week 48 | Confirmed objective response is defined as achieving radiological response, confirmed by a subsequent assessment performed at least after 4 weeks. The achievement of radiological response requires ≥20% reduction from baseline in the sum of target lesion volumes (1 to 3 target lesions, assessed by Magnetic Resonance Imaging (MRI) by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥20% increase from nadir, and in absence of progression of non-target lesions and without new lesions. |
| Secondary | Change from baseline (as assessed by BIRC) in target lesion volume | Baseline, Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | Change from baseline (as assessed by BIRC) in target lesion volume will be summarized descriptively |
| Secondary | Change from baseline (as assessed by BIRC) in MRI-measurable non-target lesion volume | Baseline, Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | Change from baseline (as assessed by BIRC) in MRI-measurable non-target lesion volume will be summarized descriptively |
| Secondary | Change from baseline (as assessed by BIRC) in all MRI-measurable (target and non-target) lesion volume | Baseline, Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | Change from baseline (as assessed by BIRC) in all MRI-measurable (target and non-target) lesion volume will be summarized descriptively |
| Secondary | Change from baseline (as assessed by BIRC) in other non-target lesion | Baseline, Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | Change from baseline (as assessed by BIRC) in other non-target lesion will be summarized descriptively |
| Secondary | Appearance of new lesions (as assessed by BIRC) | Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | Appearance of new lesions (as assessed by BIRC) will be summarized descriptively |
| Secondary | Proportion of participants with a radiological response | Week 12, Week 24, Week 48, Week 96, Week 144, Week 168, End of Treatment (last dose +< 14 day - Only for participants discontinuing on or prior to week 168) | |
| Secondary | Duration of Response (DoR) | From first documented response until progression of PROS lesions or death, assessed up to approximately 3 years | Duration of response (DOR) is defined as the time from the first documented confirmed objective response until progression of any PROS lesion by BIRC or death or rescue surgery for any PROS lesion. |
| Secondary | Alpelisib plasma concentration | Week 1 Day 1 (Post-dose 3 hour), Week 4 Day 1 (Pre-dose and Post-dose 3 hour), Week 12 Day 1 (Pre-dose and Post-dose 3 hour) | |
| Secondary | Change from Baseline in Brief Pain Inventory (BPI) | Baseline, Week 4, Week 8, Week 12, Week 16, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 168, End of Treatment (last dose +< 14 days) | Two items from the Brief Pain Inventory (BPI) will be used for adult and pediatric participants aged 12 years and older. The first item assesses the worst pain intensity in the past 24 hours, with responses on an 11-point scale from 0 (no pain) to 10 (pain as bad as you can imagine). The second item evaluates pain interference with general activity in the past 24 hours, also on an 11-point scale from 0 (does not interfere) to 10 (completely interferes). |
| Secondary | Change from Baseline in Wong-Baker Faces Scale | Baseline, Week 4, Week 8, Week 12, Week 16, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 168, End of Treatment (last dose +< 14 days) | For children aged 3-11 years, the Wong-Baker FACES® Pain Rating Scale will be used instead of the BPI worst pain intensity question. This scale features six faces showing increasing levels of pain, each with a numeric rating and a descriptor (from 0 'no hurt' to 10 'hurts worst'). Children will select the face that best represents their pain level. There is no pain intensity scale for children under 3 years. |
| Secondary | Change from Baseline in Patient Global Impression of Symptom Severity (PGI-S) | Baseline, Week 4, Week 8, Week 12, Week 16, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 168, End of Treatment (last dose +< 14 days) | A Patient Global Impression of Symptom Severity (PGI-S) item will be used to understand the overall severity of symptoms experienced and clinical meaningfulness of treatment effects experienced during this study. This item includes 5 response options: no symptoms, mild, moderate, severe, and very severe. This scale can be completed by the parent/caregiver for pediatric participants aged \<12 years. |
| Secondary | Time to Treatment Failure (TTF) | From Baseline up to approximately 3 years | Time to Treatment Failure (TTF) is defined as the time from alpelisib treatment start date until disease progression supported by radiological assessment performed by BIRC, death, rescue surgery for any PROS lesion or discontinuation of alpelisib due to any reason other than technical problems or study termination by the sponsor. Participants who complete the study, are ongoing treatment or discontinue alpelisib due to technical problems (e.g. drug supply issues, site prematurely ends study participation) or study termination by the sponsor will be censored at the date of last study treatment received. |
| Secondary | Overall Clinical Response | From Baseline up to approximately 3 years | The proportion of participants with overall clinical response reported as improvement, stable or worsening of clinical condition, as assessed by the Investigator will be summarized descriptively. |
| Secondary | Change from Baseline in symptoms and complications/comorbidities associated with PROS | From Baseline up to approximately 3 years | Changes in symptoms and complications/comorbidities (i.e., walking impairment, cardiac/pulmonary/renal function, pain, Karnofsky/Lansky performance status) will be summarized descriptively. For each complication/comorbidity, only participants who have this reported as being present at baseline will be included in the analysis. When new symptoms/complications/comorbidities appear at any time of study therapy, they will be assessed for clinical significance and reported as adverse events when applicable. |
| Secondary | Percentage of participants with healthcare visits/hospitalized due to PROS | Up to at least 3 years | Percentage of participants with healthcare visit/hospitalized due to PROS will be assessed for Group 1 and Group 2. |
| Secondary | Percentage of participants with surgeries required to manage PROS | Up to at least 3 years | Percentage of participants requiring rescue surgery due to PROS will be assessed for Group 1 and Group 2. |
| Secondary | Number of Adverse Events and Serious Adverse Events as assessed by CTCAE criteria | Up to at least 3 years | Treatment emergent Adverse Event (TEAEs) in this study were 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. |
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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