Vorasidenib Study in Pediatric Participants With Grade 2 Astrocytoma or Oligodendroglioma With an IDH1 or IDH2 Mutation

Conditions

• Grade 2 Astrocytoma or Oligodendroglioma With an IDH1 or IDH2 Mutation

Eligibility Criteria

Sex

ALL

Age

12 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Vorasidenib — DRUG
  40mg taken orally daily for participants weighing ≥ 40 kg OR 20mg taken orally daily for participants weighing ≥ 25 kg to \< 40 kg

Study Details

The objective of this study is to evaluate the safety, tolerability, efficacy profile, and effect on growth and development of vorasidenib in pediatric participants aged 12 to \< 18 years old with grade 2 glioma with an IDH1 or IDH2 mutation. The study includes a screening period, a treatment period consisting of continuous 28-day cycles of treatment, a safety follow-up period and a long-term follow-up period. The long-term follow-up period will assess participants for growth, development, and long-term safety impacts for approximately 5 years after the start of treatment or until Tanner Stage V is reached (whichever is later). Participants may undergo blood tests, heart tests (electrocardiogram (ECG)), imaging (MRI, X-ray), vital sign checks, and physical exams.

Key Dates

First listed

Dec 16, 2025

Start date

Jul 23, 2026

Status verified

Aug 2026

Primary completion

May 2, 2033

Completion

May 2, 2033

Study Design

Enrollment

10 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Open Label Vorasidenib

Primary Outcome Measure

Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events (AEs) leading to discontinuation, or Adverse Events (AEs) leading to death [ Time Frame: From start of treatment through 28 days after last dose for AEs (Safety follow-up), study-related SAEs will be reported through long-term follow-up (approximately 5 years per participant for a total study duration of approximately 7 years) ]

Central Contacts

• Institut de Recherches Internationales Servier (I.R.I.S.)
  +33 1 55 72 60 00
  [email protected]