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

Conditions

• Recurrent Langerhans Cell Histiocytosis
• Refractory Langerhans Cell Histiocytosis

Eligibility Criteria

Sex

ALL

Age

180 Days - 22 Years

Healthy Volunteers

Not accepted

Interventions

• Biospecimen Collection — PROCEDURE
  Undergo collection of blood and urine samples
• Bone Marrow Aspiration — PROCEDURE
  Undergo bone marrow aspiration
• Bone Marrow Biopsy — PROCEDURE
  Undergo bone marrow biopsy
• Computed Tomography — PROCEDURE
  Undergo CT
• Echocardiography Test — PROCEDURE
  Undergo ECHO
• FDG-Positron Emission Tomography and Computed Tomography Scan — PROCEDURE
  Undergo FDG-PET imaging
• Lumbar Puncture — PROCEDURE
  Undergo lumbar puncture
• Multigated Acquisition Scan — PROCEDURE
  Undergo MUGA
• Tovorafenib — DRUG
  Given PO

Study Details

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.

Key Dates

Start date

Mar 28, 2024

Status verified

Jan 2026

Primary completion

Sep 30, 2028

Completion

Sep 30, 2028

Study Design

Enrollment

48 participants (estimated)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Treatment (tovorafenib)
  Patients receive tovorafenib PO QW on days 1, 8, 15, and 22 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MUGA or ECHO scans, and FDG-PET or CT throughout the trial, and collection of blood and urine samples on study. Patients with suspicion of bone marrow and/or central nervous system involvement will also undergo bone marrow biopsy and aspiration and lumbar puncture on study and during follow up.

Primary Outcome Measure

Frequency of dose limiting toxicity (dose finding phase) [ Time Frame: Up to 28 days ]

Locations (104)

Find similar trials in Birmingham, AL

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