NCT05294289

Health-related Quality of Life, Symptom Severity, and Pain Among Patients With PIK3CA-related Overgrowth Spectrum: A Mixed-methods Observational Study

Health-related Quality of Life, Symptom Severity, and Pain Among Patients Treated With Alpelisib for PIK3CA-related Overgrowth Spectrum: A Mixed-methods Observational Study

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 77 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 5 Years – 99 Years
• Healthy volunteers: Not accepted

Summary

This was a mixed-methods observational study that incorporated both qualitative interviews and longitudinal quantitative data collection through an online survey (initial, 2 months, and 4 months).

Detailed description

This study collected qualitative data through interviews with patients and caregivers and qualitative data through a web-based longitudinal survey. Both parts of the study were designed to collect information on HRQoL, symptom severity, and pain among patients with PROS who were receiving treatment with alpelisib in the US. In addition, patients who had not been treated with alpelisib also participated in the quantitative part of the study by providing data on HRQoL, symptom severity, and pain, collected from a single administration of the web-based survey.

Conditions

• PIK3CA-related Overgrowth Spectrum

Interventions

Type	Name	Description
OTHER	Alpelisib	There was no treatment allocation. Patients administered alpelisib by prescription that were involved in Novartis' alpelisib managed access program were enrolled

Timeline

• Start date: 2022-05-16
• Primary completion: 2024-06-28
• Completion: 2024-06-28
• First posted: 2022-03-24
• Last updated: 2025-06-24

Locations

1 site across 1 country: United States