Long-term study of dabrafenib and trametinib in children and adolescents with cancers that have V600 mutation

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What is this study about?

This study focuses on children and adolescents with cancers that have a specific genetic change called V600 mutation. The study will evaluate the long-term effects of two medications: dabrafenib (also known as DRB436) and trametinib (also known as Mekinist). These medications can be given either separately or in combination. The purpose is to assess the long-term safety of these treatments in young patients.

The medications are available in different forms to make them suitable for children – including tablets that can be dissolved in water, hard capsules, and oral solution powder. The study will monitor how these treatments affect the participants’ growth, development, and overall health over time. The medications are taken by mouth daily, and patients will continue receiving the same treatment they were getting in their previous studies.

Throughout the study, doctors will track any health changes and measure important aspects of development such as height, weight, bone growth, and heart function. The study will continue following patients for several years to understand how the treatments affect young patients in the long term.

1 Initial assessment

Your medical condition will be evaluated to confirm the presence of a V600 mutation in your cancer.

The doctor will review your previous participation in related clinical trials and current treatment status with dabrafenib and/or trametinib.

Your ability to follow the study schedule and treatment plan will be assessed.

2 Treatment continuation

You will continue receiving the same medication you were taking in your previous study:

Dabrafenib (DRB436) taken by mouth as capsules or dispersible tablets

Trametinib (Mekinist) taken by mouth as film-coated tablets or oral solution

The specific dosage will remain the same as in your previous treatment plan

3 Regular monitoring visits

Your health will be monitored through regular check-ups that include:

Recording any side effects or health changes

Laboratory tests to check your blood values

Measurement of vital signs

Tracking of height, weight, and physical development

Assessment of heart function

Evaluation of your cancer response to treatment

4 Long-term follow-up

The study will continue until May 2026

Regular assessments will track your growth and development over time

Your overall health status will be monitored throughout the study period

Who Can Join the Study?

  • Patient (or parent/legal guardian) must provide written informed consent before any screening procedures
  • Must have previously participated or currently participating in one of these Novartis studies:
    • CTMT212X2101
    • CDRB436G2201
    • CDRB436A2102
  • Must be currently receiving treatment with dabrafenib and/or trametinib (medicines used to treat certain types of cancer)
  • Must have shown good compliance with treatment and study requirements in the previous study
  • Must be willing and able to:
    • Attend scheduled clinic visits
    • Follow treatment plans
    • Complete other study procedures
  • Doctor must believe that continuing treatment will be beneficial for the patient
  • Must not be taking any medications that are not allowed in the study
  • Can be male or female
  • Must be a child or adolescent with cancer that has a specific genetic change called V600 mutation

Who Cannot Join the Study?

  • History of severe allergic reactions to dabrafenib or trametinib (medications used in the study)
  • Participation in another clinical trial within the last 30 days
  • Presence of active infections requiring treatment
  • Significant heart problems or uncontrolled high blood pressure
  • Severe liver problems (liver function tests more than 3 times above normal)
  • Severe kidney problems (kidney function less than 30% of normal)
  • Pregnancy or breastfeeding
  • Unable to swallow pills
  • Mental conditions that prevent understanding of study requirements
  • Known drug or alcohol abuse
  • Cancer that has spread to the brain, unless previously treated and stable
  • Taking medications that may interfere with the study drugs
  • Any medical condition that the doctor believes makes participation unsafe

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Institut Gustave Roussy Villejuif France
Hospital Universitario Y Politecnico La Fe Valencia Spain
Centre Hospitalier Regional Et Universitaire De Brest Brest France

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Hospital General Universitario Gregorio Maranon Madrid Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Karolinska University Hospital Solna Sweden
Prinses Maxima Centrum voor Kinderoncologie B.V. Utrecht The Netherlands
Centre Hospitalier Universitaire De Rennes Rennes France
Rigshospitalet Copenhagen Denmark
Pirkanmaan hyvinvointialue Tampere Finland
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Cryaioysq Uowxykygiozaoc Scqtsgesr Woluwe-Saint-Lambert Belgium
Uqzrlbinzv Mpcbbls Ceiitb Hvzqotxpfeueuutqt Hamburg Germany
Ubuodqeivphrejooojyux Ezuni Aym Essen Germany
Utvcyfdjtsvxobjonmhfk Aaoxsyqz Augsburg Germany
Uvaxqqiplu Hghofomy Czitoer Cologne Germany
Ftmeqrtl nxwpifokz Mfgxi a Hgkebdo Prague Czechia
Aqgywqbxrl Pmzrdwqv Horhwwfg Di Mrgbbjkzs Marseille France
Ampncxf Oxeaqddsgfm Uaqaoxpluucmu Clbjqqnlubnm Dwayu Seeguj E Dbjsh Sasazvy Dm Ttghxm Turin Italy
Cpcc Dm Nblan Vandoeuvre Les Nancy France
Iurnlcih Cfijg Paris France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
04.11.2019
Czechia Czechia
Not recruiting
04.11.2019
Denmark Denmark
Not recruiting
04.11.2019
Finland Finland
Not recruiting
04.11.2019
France France
Not recruiting
04.11.2019
Germany Germany
Not recruiting
04.11.2019
Italy Italy
Not recruiting
04.11.2019
Spain Spain
Not recruiting
04.11.2019
Sweden Sweden
Not recruiting
04.11.2019
The Netherlands The Netherlands
Not recruiting
04.11.2019

Trial locations

Tafinlar (dabrafenib) is a medication used to treat certain types of cancer. It works by blocking a specific protein that can cause cancer cells to grow. This medication is being studied for long-term use in children who have certain types of cancer.

Mekinist (trametinib) is another cancer medication that works by targeting a different protein involved in cancer cell growth. Like Tafinlar, it is being studied for long-term treatment in pediatric patients with cancer.

The trial also studies the combination of Tafinlar and Mekinist together, as these medications can work in partnership to fight cancer cells through different mechanisms. This combination approach may provide better results than using either medication alone.

Investigated diseases:

Cancer with V600 mutation – A specific type of cancer characterized by a mutation in the BRAF gene at position V600. This genetic alteration causes cells to grow and divide uncontrollably. The mutation can occur in various types of cancer, particularly affecting children and adolescents. The condition involves changes in normal cell growth patterns, leading to the formation of tumors. The disease progresses when mutated cells continue to multiply and potentially spread to other parts of the body.

Rare Cancer – A form of cancer that affects a limited number of people compared to more common cancer types. This condition occurs in a small percentage of the population, making it less frequently encountered in medical practice. The disease develops when specific cell types in the body begin to grow abnormally. These cancers can appear in various parts of the body and may have unique growth patterns.

Trial ID:
2023-509276-42-00
Protocol code:
CDRB436G2401
NCT ID:
NCT03975829
Trial Phase:
Therapeutic confirmatory (Phase III)

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