Alpelisib for Overgrowth Spectrum
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effectiveness of a drug called alpelisib for individuals with PIK3CA-related overgrowth spectrum (PROS). PROS is a genetic disorder causing abnormal and excessive growth in certain body areas, leading to physical symptoms and functional limitations. Researchers aim to determine if taking alpelisib daily can help manage these symptoms. Suitable candidates for this trial are those with symptomatic and progressive overgrowth due to PROS, including at least one measurable lesion affecting daily life. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.
Is there any evidence suggesting that alpelisib is likely to be safe for humans?
Research shows that alpelisib has been tested for safety in people with PIK3CA-related overgrowth spectrum (PROS). Previous studies found that alpelisib is generally well-tolerated, though some side effects have been observed. Common side effects include high blood sugar, nausea, and diarrhea, which standard care can usually manage.
The FDA has already approved alpelisib for treating breast cancer, indicating a known safety record in humans. While this doesn't guarantee safety for PROS, it offers some reassurance.
In summary, although alpelisib can cause side effects, it has been tested before and is generally well-tolerated, especially with proper monitoring and care.12345Why do researchers think this study treatment might be promising for overgrowth?
Most treatments for overgrowth spectrum conditions focus on managing symptoms through surgeries and supportive care. But Alpelisib works differently, targeting a specific genetic pathway known as the PI3K pathway, which is often overactive in these disorders. Researchers are excited about Alpelisib because it directly addresses the root cause of the overgrowth by inhibiting this pathway, potentially offering more precise and effective control of the condition. This targeted approach could mean fewer complications and better outcomes for both adults and children dealing with overgrowth spectrum conditions.
What evidence suggests that alpelisib might be an effective treatment for PROS?
Research has shown that alpelisib may help treat PIK3CA-related overgrowth spectrum (PROS). In one study, 37.5% of patients experienced a noticeable decrease in the size of their overgrowths. Another study found that 27% of patients had a positive response after 24 weeks. Additionally, real-world evidence suggests that alpelisib can reduce the size of problematic growths and improve symptoms in children. Although not everyone benefits, the data indicates that alpelisib can be effective for some people with PROS. Participants in this trial will be divided into two groups: Group 1 for adults and Group 2 for children and adolescents, to evaluate the effects of alpelisib across different age ranges.678910
Who Is on the Research Team?
Novartis Pharmaceuticals
Principal Investigator
Novartis Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for pediatric and adult patients aged ≥2 years with PIK3CA-related Overgrowth Spectrum (PROS). Participants must have a documented PIK3CA gene mutation, at least one measurable lesion related to PROS, and adequate organ function. They should not have isolated macrodactyly, macrocephaly or epidermal nevus without other syndromic features.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Core Treatment
Participants receive daily oral alpelisib treatment; adults start with 250 mg, children with 50 mg or 125 mg based on age
Extension 1
Participants continue treatment under the same rules as the core period until Week 168
Extension 2
Participants continue treatment until the last participant completes the Extension 2 period; post-trial access may be provided
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Alpelisib
Trial Overview
The study tests the effectiveness of Alpelisib, an oral medication taken daily by participants with PROS. It aims to demonstrate how well it works and evaluate its safety and tolerability in affected individuals.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Children and adolescents 2 to \<18 years of age.
Adult participants ≥18 years of age.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD
Citations
Alpelisib for PIK3CA-related Overgrowth Spectrum (PROS)
The confirmed response rate at Week 24 (+/− 4 weeks) by BICR was 27%. Additional efficacy results are described in Table 2 (14) and Figure 1 (13). Table 2:.
2.
ashpublications.org
ashpublications.org/blood/article/144/Supplement%201/5512/527723/Epik-P2-A-Phase-2-Study-of-Alpelisib-ALP-inEpik-P2: A Phase 2 Study of Alpelisib (ALP) in Pediatric and ...
The primary objective was not met; confirmed objective response with ALP (any time) was achieved in 16.7% (9/54; 97.5% CI: 7.0%-31.1%) and 23.2% ...
NCT04285723 | Retrospective Chart Review Study of ...
Improvement in PIK3CA Related Overgrowth Spectrum (PROS) signs and symptoms was defined based on Common Toxicity Criteria (CTC) grade reduction or resolution of ...
Novartis announces findings from a real-world study of ...
Results from EPIK-P1 showed alpelisib effectively reduced volume of clinically significant PROS-related lesions and improved signs and symptoms in pediatric ...
Alpelisib (ALP), a breast cancer therapy, for <i>PIK3CA</i>
In the primary analysis of the primary endpoint, 12/32 pts (37.5%; 95% CI, 21.1-56.3%) with complete cases responded (≥20% reduction in the sum ...
6.
vijoice-hcp.com
vijoice-hcp.com/products/vijoice/pik3ca-related-overgrowth-spectrum/safety-profile/full-safety-profileFull Safety Profile | VIJOICE® (alpelisib) tablets | HCP
View the safety profile and adverse reactions of VIJOICE in patients with PIK3CA-Related Overgrowth Spectrum (PROS). See full prescribing and safety info.
Alpelisib-related adverse events: The FDA ...
Alpelisib was approved for treatment of breast cancer. We assessed the safety signals associated with alpelisib by data mining the FDA ...
A real-world disproportionality analysis of FDA adverse ...
Our research scrutinizes the FDA database to assess alpelisib 's safety. We retrospectively analyzed data from April 2019 to June 2023 using ...
NCT04980833 | Study Assessing Long-term Safety and ...
Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study ...
10.
ashpublications.org
ashpublications.org/blood/article/146/Supplement%201/8227/553828/Efficacy-safety-and-pharmacokinetics-of-alpelisibEfficacy, safety, and pharmacokinetics of alpelisib (ALP) in ...
Efficacy, safety, and pharmacokinetics of alpelisib (ALP) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS): Epik- ...
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