MedTrace Logo

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

NCT05828069 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2026-05-22

No results posted yet for this study

Summary

This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.

Conditions

  • Recurrent Langerhans Cell Histiocytosis
  • Refractory Langerhans Cell Histiocytosis

Interventions

PROCEDURE

Biospecimen Collection

Undergo collection of blood and urine samples

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow aspiration

PROCEDURE

Bone Marrow Biopsy

Undergo bone marrow biopsy

PROCEDURE

Computed Tomography

Undergo CT

PROCEDURE

Echocardiography Test

Undergo ECHO

PROCEDURE

FDG-Positron Emission Tomography and Computed Tomography Scan

Undergo FDG-PET imaging

PROCEDURE

Lumbar Puncture

Undergo lumbar puncture

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

DRUG

Tovorafenib

Given PO

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • Carl E Allen · Children's Oncology Group

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
180 Days
Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-28
Primary Completion
2028-09-30
Completion
2028-09-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05828069 on ClinicalTrials.gov