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

Conditions

• Lymphatic Malformations

Eligibility Criteria

Sex

ALL

Age

0 Years - 100 Years

Healthy Volunteers

Not accepted

Interventions

• Alpelisib — DRUG
  In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib. In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib
• Placebo — DRUG
  In Stage 2, participants will receive matching placebo for 24 weeks of the study

Study Details

The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.

Key Dates

Start date

Nov 24, 2023

Status verified

Jun 2026

Primary completion

May 31, 2028

Completion

May 2, 2033

Study Design

Enrollment

232 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Adult participants, alpelisib dose 1 (Stage 1)
  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)
• Experimental: Adult participants, alpelisib dose 2 (Stage 1)
  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).
• Experimental: Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1)
  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)
• Experimental: Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1)
  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).
• Experimental: Adult participants, alpelisib (Stage 2)
  Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2)
• Placebo Comparator: Adult participants, placebo (Stage 2)
  Adult participants (≥18 years of age) who will receive matching placebo
• Experimental: Pediatric participants (6-17 years of age), alpelisib (Stage 2)
  Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2)
• Placebo Comparator: Pediatric participants (6-17 years of age), placebo (Stage 2)
  Pediatric participants (6-17 years of age) who will receive matching placebo
• Experimental: Pediatric participants (0-5 years of age), alpelisib (Stage 2)
  Pediatric participants of 0-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier

Primary Outcome Measure

Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants) [ Time Frame: Baseline, Week 24 ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111
  [email protected]

Locations (16)

Find similar trials in Oakland, CA

Related Studies

• A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation
  PHASE2 · Recruiting · Relay Therapeutics, Inc. · Phoenix, Arizona