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PI3K Inhibitor
Alpelisib for Lymphatic Malformations (EPIK-L1 Trial)
Phase 2 & 3
Recruiting
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participant has evidence of a somatic mutation(s) in the PIK3CA gene
Participant has a physician confirmed and documented diagnosis of a LyM at the time of informed consent
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 8 years
Summary
This trial aims to see if alpelisib can help improve symptoms and reduce the size of lymphatic malformations in people with a specific gene mutation.
Who is the study for?
This trial is for pediatric and adult patients with lymphatic malformations (LyM) that are linked to a PIK3CA gene mutation. Participants must be willing to follow the study's schedule, have a confirmed diagnosis of LyM, not be candidates for or unwilling to undergo local treatments like sclerotherapy until after 24 weeks, and have at least one measurable LyM lesion.
What is being tested?
The trial is testing the effectiveness of Alpelisib compared to a placebo in treating lymphatic malformations. The focus is on observing changes in radiological response and symptom severity between the two groups.
What are the potential side effects?
While specific side effects for this trial aren't provided here, Alpelisib can generally cause issues like high blood sugar levels, skin rash, diarrhea, fatigue, nausea and mouth sores among others.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My cancer has a PIK3CA gene mutation.
Select...
My doctor has confirmed I have lymphoma.
Select...
I have a measurable lymphoma lesion confirmed by a specialist.
Select...
It seems like the criterion is incomplete. Could you provide more context or details so that I can accurately summarize it for you?
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to approximately 8 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 8 years
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Secondary study objectives
Alpelisib plasma concentrations (Stage 1 and 2)
Change from baseline in LyM lesions in adult and pediatric participants (Stage 1 and 2)
Change from baseline in LyM lesions in adult and pediatric participants at Week 24 (Stage 1 and 2)
+15 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%
Dry mouth
10%
Urinary tract infection
10%
Blood creatinine increased
10%
Gamma-glutamyltransferase increased
10%
Aspartate aminotransferase increased
10%
Cough
8%
Nasopharyngitis
8%
Pain in extremity
8%
Dizziness
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%
Pneumonitis
1%
Dehydration
1%
Pulmonary embolism
1%
Upper gastrointestinal haemorrhage
1%
General physical health deterioration
1%
Cellulitis
1%
Pleural effusion
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
Trial Design
9Treatment groups
Experimental Treatment
Placebo Group
Group I: Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1)Experimental Treatment1 Intervention
Pediatric participants 6-17 years of age who will receive dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Group II: Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1)Experimental Treatment1 Intervention
Pediatric participants 6-17 years of age who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Group III: Pediatric participants (6-17 years of age), alpelisib (Stage 2)Experimental Treatment1 Intervention
Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2)
Group IV: Pediatric participants (2-5 years of age), alpelisib (Stage 2)Experimental Treatment1 Intervention
Pediatric participants of 2-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier
Group V: Adult participants, alpelisib dose 2 (Stage 1)Experimental Treatment1 Intervention
Adult participants (≥18 years of age) who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Group VI: Adult participants, alpelisib dose 1 (Stage 1)Experimental Treatment1 Intervention
Adult participants (≥18 years of age) who will receive dose 1 of alpelisib an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Group VII: Adult participants, alpelisib (Stage 2)Experimental Treatment1 Intervention
Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2)
Group VIII: Adult participants, placebo (Stage 2)Placebo Group1 Intervention
Adult participants (≥18 years of age) who will receive matching placebo
Group IX: Pediatric participants (6-17 years of age), placebo (Stage 2)Placebo Group1 Intervention
Pediatric participants (6-17 years of age) who will receive matching placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alpelisib
2018
Completed Phase 3
~1340
Find a Location
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,921 Previous Clinical Trials
4,254,082 Total Patients Enrolled