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NCT04925479 · ClinicalTrials.gov registry record · Phase 1

Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

A Phase 1 study of Myeloid Leukemia, Philadelphia Positive, sponsored by Novartis Pharmaceuticals.

Recruiting
Registry status
Phase 1
Development phase
44
Enrollment target
20
Study locations

NCT04925479: Recruiting Phase 1 study of Myeloid Leukemia, Philadelphia Positive, sponsored by Novartis Pharmaceuticals.

NCT04925479 is a Phase 1 study of Myeloid Leukemia, Philadelphia Positive that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 44 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (27% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04925479, a Phase 1 study of Myeloid Leukemia, Philadelphia Positive, is actively recruiting participants, sponsored by Novartis Pharmaceuticals.

RECRUITING
Registry status
Phase 1
Development phase
44 participants
Enrollment target
20
Study locations

Study Summary

The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults.

Primary Outcome

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Interventions

  • DRUG Asciminib Pediatric formulation group
  • DRUG Asciminib Adult formulation group

Study Locations (20)

Other

  • Novartis Investigative Site - Beijing
  • Novartis Investigative Site - Shanghai
  • Novartis Investigative Site - Tianjin
  • Novartis Investigative Site - Bordeaux
  • Novartis Investigative Site - Lille
  • Novartis Investigative Site - Paris
  • Novartis Investigative Site - Poitiers
  • Novartis Investigative Site - Erlangen
  • Novartis Investigative Site - Essen
  • Novartis Investigative Site - Hamburg

Massachusetts

  • Dana Farber Cancer Institute - Boston
  • Dana Farber Cancer Institute - Boston

Indiana

  • Indiana UH Riley H for CIU - Indianapolis

Mississippi

  • University of Mississippi Medical Center - Jackson

New York

  • Columbia University Medical Center New York Presbyterian - New York

Ohio

  • Cinn Children Hosp Medical Center - Cincinnati

Pennsylvania

  • Childrens Hospital of Philadelphia - Philadelphia

Texas

  • Uni Of Texas MD Anderson Cancer Ctr - Houston

Trial Details

FieldValue
Enrollment Target 44 participants
Start Date 2021-12-27
Est. Completion 2031-11-01
Phase Phase 1
Novartis Pharmaceuticals

1,375 total trials

What NCT04925479 shows while recruiting

NCT04925479 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 44 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (27% lower).

The record links to 1 condition, with Myeloid Leukemia, Philadelphia Positive appearing as the primary indexed condition, and to 2 interventions - of which Asciminib Pediatric formulation group is the first listed.

NCT04925479 names 20 study sites across 10 states, led by Other, Massachusetts, Indiana.

Frequently Asked Questions

What is clinical trial NCT04925479 about?

NCT04925479 is a clinical study titled "Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia". The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML...

What is the current status of trial NCT04925479?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 44 participants. The study started on 2021-12-27. Estimated completion is 2031-11-01.

What conditions does trial NCT04925479 study?

This clinical trial studies the following conditions: Myeloid Leukemia, Philadelphia Positive.

What interventions are being tested in trial NCT04925479?

The interventions under investigation include: Asciminib Pediatric formulation group (DRUG), Asciminib Adult formulation group (DRUG).

Who is sponsoring clinical trial NCT04925479?

This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04925479 being conducted?

This trial has 20 study locations across Indiana, Massachusetts, Mississippi, New York, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

How this trial's enrollment target compares

Where NCT04925479's enrollment target sits among peer trials

44 1087th of 2000 higher than 901 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT04925479, the US trial registry maintained by the National Library of Medicine. NCT04925479 (small enrollment · wide site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.