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PI3K Inhibitor

Pediatric participants (2-5 years of age), alpelisib (Stage 2) 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 6 years

Awards & highlights

EPIK-L1 Trial 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.Check my eligibility

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.See study design

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.

EPIK-L1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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?

EPIK-L1 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 6 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 6 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 6 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)

Secondary outcome measures

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 more

Side effects data

From 2023 Phase 3 trial • 572 Patients • NCT02437318

62%

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

Dizziness

Nasopharyngitis

Pain in extremity

Hypertension

Anaemia

Constipation

Alanine aminotransferase increased

Hypokalaemia

Dyspnoea

Myalgia

Muscle spasms

Insomnia

Lipase increased

Abdominal pain upper

Lymphoedema

Musculoskeletal pain

Erythema

Upper respiratory tract infection

Bone pain

Hot flush

Osteonecrosis of jaw

Acute kidney injury

Pleural effusion

Cellulitis

Upper gastrointestinal haemorrhage

Dehydration

Pneumonitis

Pulmonary embolism

General physical health deterioration

Hypersensitivity

Pneumonia

Hyponatraemia

Muscular weakness

Brain oedema

Renal failure

Erythema multiforme

100%

80%

60%

40%

20%

Study treatment Arm

Placebo qd + Fulvestrant

Alpelisib qd + Fulvestrant

EPIK-L1 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

~900

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor

2,860 Previous Clinical Trials

4,198,079 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available vacancies for patient participation in this clinical trial?

"Indeed, the information available on clinicaltrials.gov indicates that this particular clinical trial is currently in the process of recruiting eligible participants. The trial was initially posted on November 24th, 2023 and received its most recent update on December 27th, 2023. In total, there are plans to enroll a cohort of 230 patients across two designated sites."

Answered by AI

What is the current number of individuals eligible for enrollment in this clinical research study?

"Indeed, the information available on clinicaltrials.gov confirms that this trial is actively seeking participants. The initial posting of the trial occurred on November 24th, 2023 and it was most recently updated on December 27th, 2023. A total of two locations are enrolling a combined number of 230 patients for this study."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

2023. "Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05948943.