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Kinase Inhibitor

DAY101 for Langerhans Cell Histiocytosis

Q: Does this medical experiment allow individuals aged 18 or older to participate?

To be accepted into this trial, applicants must fall between 180 days and 22 years of age. For those under 18 or over 65 there are 27 other clinical trials that may match their needs.

Q: Has tovorafenib been given the stamp of approval by the FDA?

The safety of tovorafenib was estimated as a 2 on our scale, due to the lack of clinical evidence supporting its efficacy but existing data suggesting it is safe.

Q: Am I a viable candidate to be included in this experiment?

This medical trial is recruiting approximately 29 individuals with langerhans cell histiocytosis that are between 180 days and 22 years of age.

Q: Is there still an opportunity to partake in this research endeavor?

Affirmative, the clinical trial registry shows that this research is still open to participants. It was initially posted on April 7th 2023, then modified recently on April 26th of the same year. 29 participants need to be recruited from 2 distinct centres.

Q: What is the current recruitment size for this trial?

Affirmative. Evidence hosted on clinicaltrials.gov claims that this medical experiment, which was first published in April 7th 2023, is presently recruiting individuals. In total there are 29 patients to be enrolled between two distinct medical clinics.

Q: What objectives are the researchers hoping to accomplish through this clinical trial?

As the primary objective of this trial, monitored over two 28-day cycles, is to measure frequency of dose limiting toxicity (DLT). Secondary objectives include estimating progression free survival rate (PFS) using Kaplan–Meier method from study enrolment and evaluating overall survival rate (OS) with Log-rank test after enrolling last patient. Additionally, duration of response rate will be determined by modified RECIST/PERCIST criteria used in previous pediatric LCH trials and adult histiocytosis trials involving MAPK inhibitors.

Phase 2

Recruiting

Led By Michelle L Hermiston

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients aged 180 days to < 22 years at time of study enrollment

Patients must not have received myelosuppressive chemotherapy within 14 days of entry onto this study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 1 and 2 years

Awards & highlights

Study Summary

This trial tests if a medication called DAY101 can help treat a type of disease that forms tumors in certain organs in children & young adults. DAY101 blocks enzymes needed for cell growth, which may stop the growth of cancer cells.

Who is the study for?

This trial is for patients aged 6 months to under 22 years with progressive, relapsed, or refractory Langerhans cell histiocytosis who've had prior chemotherapy. They must have a certain level of organ function and performance status, no severe CNS toxicity, controlled infections or seizures, and not be pregnant or breastfeeding. Patients can't join if they've had recent surgeries, uncontrolled diseases, specific drug allergies or previous MAPK inhibitor therapy.Check my eligibility

What is being tested?

The trial tests the safety and effectiveness of tovorafenib (DAY101) in young patients with Langerhans cell histiocytosis that's worsening or not responding to treatment. It aims to find the best dose while monitoring how well it stops cancer cells by blocking enzymes needed for their growth.See study design

What are the potential side effects?

Potential side effects include reactions related to immune system activation such as inflammation in various organs but specifics are not provided; however, participants will be closely monitored for any adverse reactions during the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 6 months and 21 years old.

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I haven't had chemotherapy that lowers my blood cell counts in the last 14 days.

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I can take medicine by mouth.

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My brain-related side effects are mild.

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My cancer has worsened or returned after chemotherapy.

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I am mostly able to care for myself and carry out daily activities.

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I can take care of myself but might not be able to do heavy physical work.

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My LCH has come back or spread, and it can be measured.

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My kidney function tests are within the required range for my age and gender.

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My cancer has a known harmful gene mutation.

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I don't have shortness of breath at rest, can exercise, and my oxygen levels are above 94%.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 1 and 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 1 and 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Frequency of dose limiting toxicity (DLT) (dose finding phase)

Overall response rate (ORR) (phase II)

Secondary outcome measures

Duration of response rate

Event free survival rate (EFS)

Overall survival rate (OS)

+1 more

Other outcome measures

Percent peripheral blood mononuclear cells (PBMC) with mutated allele

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (tovorafenib)Experimental Treatment9 Interventions

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 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Biospecimen Collection

2004

Completed Phase 2

~1700

Bone Marrow Aspiration

2011

Completed Phase 2

~1740

Bone Marrow Biopsy

2021

Completed Phase 2

~10

Computed Tomography

2017

Completed Phase 2

~2720

Echocardiography

2013

Completed Phase 4

~11670

Lumbar Puncture

2016

Completed Phase 3

~510

0 / 11Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor

13,666 Previous Clinical Trials

40,926,000 Total Patients Enrolled

Michelle L HermistonPrincipal InvestigatorChildren's Oncology Group

Media Library

Tovorafenib (Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05828069 — Phase 2

Langerhans Cell Histiocytosis Research Study Groups: Treatment (tovorafenib)

Langerhans Cell Histiocytosis Clinical Trial 2023: Tovorafenib Highlights & Side Effects. Trial Name: NCT05828069 — Phase 2

Tovorafenib (Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05828069 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this medical experiment allow individuals aged 18 or older to participate?

"To be accepted into this trial, applicants must fall between 180 days and 22 years of age. For those under 18 or over 65 there are 27 other clinical trials that may match their needs."

Answered by AI

Has tovorafenib been given the stamp of approval by the FDA?

"The safety of tovorafenib was estimated as a 2 on our scale, due to the lack of clinical evidence supporting its efficacy but existing data suggesting it is safe."

Answered by AI

Am I a viable candidate to be included in this experiment?

"This medical trial is recruiting approximately 29 individuals with langerhans cell histiocytosis that are between 180 days and 22 years of age."

Answered by AI

Is there still an opportunity to partake in this research endeavor?

"Affirmative, the clinical trial registry shows that this research is still open to participants. It was initially posted on April 7th 2023, then modified recently on April 26th of the same year. 29 participants need to be recruited from 2 distinct centres."

Answered by AI

What is the current recruitment size for this trial?

"Affirmative. Evidence hosted on clinicaltrials.gov claims that this medical experiment, which was first published in April 7th 2023, is presently recruiting individuals. In total there are 29 patients to be enrolled between two distinct medical clinics."

Answered by AI

What objectives are the researchers hoping to accomplish through this clinical trial?

"As the primary objective of this trial, monitored over two 28-day cycles, is to measure frequency of dose limiting toxicity (DLT). Secondary objectives include estimating progression free survival rate (PFS) using Kaplan–Meier method from study enrolment and evaluating overall survival rate (OS) with Log-rank test after enrolling last patient. Additionally, duration of response rate will be determined by modified RECIST/PERCIST criteria used in previous pediatric LCH trials and adult histiocytosis trials involving MAPK inhibitors."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05828069.

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