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PI3K Inhibitor
Alpelisib for Overgrowth Spectrum (EPIK-P2 Trial)
Phase 2
Recruiting
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients with diagnosis of PROS with symptomatic and /or progressive overgrowth and at least one measurable PROS-related lesion confirmed by blinded independent review committee (BIRC) assessment
Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 5 years
Awards & highlights
EPIK-P2 Trial Summary
This trial is testing a new drug to see if it's effective and safe for kids and adults with a growth disorder.
Who is the study for?
This trial is for pediatric and adult patients with PIK3CA-related Overgrowth Spectrum (PROS) who have at least one measurable lesion over 2 cm confirmed by MRI. Participants must be able to provide a tissue sample, have stable blood sugar levels, and not have had previous treatment with alpelisib or similar drugs. Those with isolated macrodactyly, skin nevus/nevi, macroencephaly without other lesions, recent radiation or surgery in the area of interest are excluded.Check my eligibility
What is being tested?
The study tests the effectiveness and safety of Alpelisib compared to a placebo in treating PROS. Initially, there's a 16-week blind phase where neither doctors nor participants know who gets Alpelisib or placebo. After that period, all may receive Alpelisib during extension periods while monitoring how their bodies absorb the drug.See study design
What are the potential side effects?
While specific side effects for this trial aren't listed here, common ones from similar medications include nausea, diarrhea, increased blood sugar levels which could affect diabetes control; rash; fatigue; and potential liver enzyme changes.
EPIK-P2 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have PROS with worsening symptoms and a confirmed measurable lesion.
Select...
My tests show a PIK3CA gene mutation.
Select...
I am mostly able to care for myself but may need help.
Select...
My blood sugar and HbA1c levels are within the required range.
EPIK-P2 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to approximately 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Proportion of participants randomized to alpelisib with a confirmed objective response by BIRC in Group 1 and Group 2
Secondary outcome measures
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
Change from baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain intensity in Group 1 and 2
Change from baseline to Week 16 in Patient Global Impression of Symptom Severity (PGIS) in Group 1 and 2
+19 moreSide effects data
From 2023 Phase 3 trial • 572 Patients • NCT0243731862%
Hyperglycaemia
57%
Diarrhoea
45%
Nausea
35%
Rash
35%
Decreased appetite
26%
Vomiting
26%
Weight decreased
24%
Fatigue
24%
Stomatitis
20%
Asthenia
20%
Alopecia
18%
Mucosal inflammation
18%
Pruritus
17%
Dysgeusia
17%
Headache
15%
Dry skin
14%
Oedema peripheral
14%
Pyrexia
14%
Back pain
13%
Rash maculo-papular
11%
Dyspepsia
11%
Abdominal pain
11%
Arthralgia
10%
Blood creatinine increased
10%
Urinary tract infection
10%
Gamma-glutamyltransferase increased
10%
Dry mouth
10%
Aspartate aminotransferase increased
10%
Cough
8%
Dizziness
8%
Nasopharyngitis
8%
Pain in extremity
8%
Hypertension
8%
Anaemia
8%
Constipation
8%
Alanine aminotransferase increased
8%
Hypokalaemia
8%
Dyspnoea
7%
Myalgia
7%
Muscle spasms
7%
Insomnia
6%
Lipase increased
6%
Abdominal pain upper
5%
Lymphoedema
5%
Musculoskeletal pain
5%
Erythema
4%
Upper respiratory tract infection
4%
Bone pain
3%
Hot flush
2%
Osteonecrosis of jaw
2%
Acute kidney injury
1%
Pleural effusion
1%
Cellulitis
1%
Upper gastrointestinal haemorrhage
1%
Dehydration
1%
Pneumonitis
1%
Pulmonary embolism
1%
General physical health deterioration
1%
Hypersensitivity
1%
Pneumonia
1%
Hyponatraemia
1%
Muscular weakness
1%
Brain oedema
1%
Renal failure
1%
Erythema multiforme
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo qd + Fulvestrant
Alpelisib qd + Fulvestrant
EPIK-P2 Trial Design
7Treatment groups
Experimental Treatment
Placebo Group
Group I: Pediatric cohort (group 5: 6-17 years old)-Alpelisib FCTExperimental Treatment1 Intervention
Pediatric participants (6 to 17 year old) will receive 125 mg alpelisib film-coated (FCT) once daily, in an open-label setting.
Group II: Pediatric cohort (group 4: 2 to 5 years old)- Alpelisib FCTExperimental Treatment1 Intervention
Pediatric participants (2 to 5 years old) will receive 50 mg of alpelisib film-coated tablets (FCT) once daily in an open-label setting.
Group III: Pediatric cohort (group 3: 2 to 5 years old)- Alpelisib granulesExperimental Treatment1 Intervention
Pediatric participants (2 to 5 years old) will receive alpelisib with the alpelisib granules formulation at dose determined based on the primary analysis for efficacy, safety and PK of alpelisib in Groups 1 and 2 in addition to the data from Group 4 and 5 as available. An extrapolation approach will be used for dose selection for this group.
Group IV: Pediatric cohort (group 2: 6 to 17 years old) -AlpelisibExperimental Treatment1 Intervention
During double-blind randomized study period (from baseline up to Week 16, pediatric participants (6 to 17 years old) will be randomized to receive alpelisib (50 mg, oral, once daily). After Week 16, participants will continue their active treatment at the same dose level.
Group V: Adult cohort (group 1)- AlpelisibExperimental Treatment1 Intervention
During double-blind randomized study period (from baseline up to Week 16), adult participants will be randomized to receive alpelisib (125 mg, oral, once daily). After Week 16, participants will continue their active treatment at the same dose level.
Group VI: Adult cohort (group 1)- PlaceboPlacebo Group2 Interventions
During double-blind randomized study period (from baseline up to Week 16), adult participants will be randomized to receive placebo (125 mg, oral, once daily). After Week 16, participants will be switched to active treatment with alpelisib at the placebo dose level received at the end of the placebo period.
Group VII: Pediatric cohort (group 2: 6 to 17 years old)-PlaceboPlacebo Group2 Interventions
During double-blind randomized study period (from baseline up to Week 16), pediatric participants (6 to 17 years old) will be randomized to receive Placebo (50mg, oral, once daily). After Week 16, participants will be switched to active treatment with alpelisib at the placebo dose level received at the end of the placebo period.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alpelisib
2018
Completed Phase 3
~900
Find a Location
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,857 Previous Clinical Trials
4,197,618 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not had major surgery within the last 3 months.I have only large fingers/toes, skin moles, or an enlarged head without other PROS-related issues.I have PROS with worsening symptoms and a confirmed measurable lesion.I have not been treated with alpelisib or similar drugs for more than 2 weeks.My tests show a PIK3CA gene mutation.I have a tumor larger than 2 cm causing symptoms or affecting my daily life, confirmed by MRI.I can provide a tissue sample for testing, either from previous or new biopsies.I have uncontrolled seizures or epilepsy and cannot switch my medication.I've had a serious blood clot or needed treatment for blood vessel issues recently.If in Germany, I will send my tissue sample to a specific lab or get a new biopsy if needed and possible.I have not had radiation on potential target lesions for PROS in the last year, except for those that got worse after treatment.My health has worsened recently, with new or increasing symptoms like swelling or pain.I have been diagnosed with type I diabetes or my type II diabetes is not under control.I have had acute pancreatitis within the last year or have chronic pancreatitis.I am mostly able to care for myself but may need help.My blood sugar and HbA1c levels are within the required range.I have lung issues not caused by PROS, confirmed by tests or suspected on MRI.
Research Study Groups:
This trial has the following groups:- Group 1: Adult cohort (group 1)- Placebo
- Group 2: Pediatric cohort (group 2: 6 to 17 years old) -Alpelisib
- Group 3: Pediatric cohort (group 3: 2 to 5 years old)- Alpelisib granules
- Group 4: Pediatric cohort (group 4: 2 to 5 years old)- Alpelisib FCT
- Group 5: Pediatric cohort (group 5: 6-17 years old)-Alpelisib FCT
- Group 6: Adult cohort (group 1)- Alpelisib
- Group 7: Pediatric cohort (group 2: 6 to 17 years old)-Placebo
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What potential risks should be considered when administering Alpelisib to individuals?
"There is evidence of Alpelisib's safety, but no proof yet that it can sustainably treat any conditions. Thus, it has been rated 2 out of 3 on our team's internal scale."
Answered by AI
Are there numerous Canadian locations actively participating in this clinical investigation?
"At present, 12 clinical sites are actively enrolling patients for this trial. Locations include Toronto, Seattle and Los Angeles along with 9 other sites nearby. To minimize travel requirements if you choose to participate in the study it is important to select a clinic that is close by."
Answered by AI
Are there any additional investigations into Alpelisib's efficacy?
"Alpelisib was first investigated in 2013 at two esteemed medical centres, Massachusetts General Hospital and Dana-Farber Cancer Institute. Since then there has been an impressive 18352 completed studies of this medication. Today, 32 live trials are underway with many located in Toronto, Ontario."
Answered by AI
What is the overall enrollment count for this clinical investigation?
"Affirmative. The details listed on clinicaltrials.gov showcase that the medical trial is actively enrolling participants and opened for recruitment on April 19th, 2021 with a most recent revision occurring September 1st, 2022. This study requires 174 patients to be recruited from 12 different locations across the country."
Answered by AI
Does this experiment require any additional participants?
"The clinical trial described on the website of ClinicalTrials.gov is searching for suitable participants, having been initially posted on April 19th 2021 and recently edited in September 1st 2022."
Answered by AI
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