Pediatric Long-Term Follow-up and Rollover Study

June 1, 2026 updated by: Novartis Pharmaceuticals

An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)

A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

165

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Argentina
    • Buenos Aires
      • CABA, Buenos Aires, Argentina, C1428AQK
        • Active, not recruiting
        • Novartis Investigative Site
  • Australia
    • New South Wales
      • Darlinghurst, New South Wales, Australia, 2010
        • Active, not recruiting
        • Novartis Investigative Site
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Active, not recruiting
        • Novartis Investigative Site
  • Belgium
      • Brussels, Belgium, 1200
        • Completed
        • Novartis Investigative Site
  • Brazil
    • São Paulo
      • Barretos, São Paulo, Brazil, 14784 400
        • Active, not recruiting
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 04829-310
        • Active, not recruiting
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 08270-070
        • Completed
        • Novartis Investigative Site
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Active, not recruiting
        • Novartis Investigative Site
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Active, not recruiting
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Completed
        • Novartis Investigative Site
  • Czechia
      • Brno, Czechia, 625 00
        • Active, not recruiting
        • Novartis Investigative Site
      • Prague, Czechia, 150 06
        • Active, not recruiting
        • Novartis Investigative Site
  • Denmark
      • Copenhagen, Denmark, DK-2100
        • Active, not recruiting
        • Novartis Investigative Site
  • Finland
      • Tampere, Finland, 33521
        • Active, not recruiting
        • Novartis Investigative Site
  • France
      • Brest, France, 29609
        • Completed
        • Novartis Investigative Site
      • Marseille, France, 13885
        • Active, not recruiting
        • Novartis Investigative Site
      • Paris, France, 75231
        • Active, not recruiting
        • Novartis Investigative Site
      • Rennes, France, 35033
        • Active, not recruiting
        • Novartis Investigative Site
      • Vandœuvre-lès-Nancy, France, 54511
        • Active, not recruiting
        • Novartis Investigative Site
      • Villejuif, France, 94800
        • Active, not recruiting
        • Novartis Investigative Site
    • Brittany Region
      • Rennes, Brittany Region, France, 35203
        • Active, not recruiting
        • Novartis Investigative Site
  • Germany
      • Augsburg, Germany, 86179
        • Active, not recruiting
        • Novartis Investigative Site
      • Berlin, Germany, 13353
        • Active, not recruiting
        • Novartis Investigative Site
      • Essen, Germany, 45147
        • Active, not recruiting
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Active, not recruiting
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Germany, 50937
        • Active, not recruiting
        • Novartis Investigative Site
  • Israel
      • Petah Tikva, Israel, 4920235
        • Active, not recruiting
        • Novartis Investigative Site
  • Italy
    • FI
      • Florence, FI, Italy, 50139
        • Active, not recruiting
        • Novartis Investigative Site
    • GE
      • Genova, GE, Italy, 16147
        • Active, not recruiting
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italy, 20133
        • Active, not recruiting
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00165
        • Active, not recruiting
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italy, 10126
        • Active, not recruiting
        • Novartis Investigative Site
  • Japan
      • Osaka, Japan, 5340021
        • Active, not recruiting
        • Novartis Investigative Site
    • Tokyo
      • Setagaya-ku, Tokyo, Japan, 1578535
        • Active, not recruiting
        • Novartis Investigative Site
  • Netherlands
      • Utrecht, Netherlands, 3584 CS
        • Active, not recruiting
        • Novartis Investigative Site
  • Russia
      • Moscow, Russia, 117198
        • Active, not recruiting
        • Novartis Investigative Site
  • Spain
      • Madrid, Spain, 28009
        • Active, not recruiting
        • Novartis Investigative Site
      • Valencia, Spain, 46026
        • Active, not recruiting
        • Novartis Investigative Site
  • Sweden
      • Stockholm, Sweden, 17176
        • Completed
        • Novartis Investigative Site
  • United Kingdom
      • Liverpool, United Kingdom, L12 2AP
        • Active, not recruiting
        • Novartis Investigative Site
      • London, United Kingdom, NW1 2BU
        • Active, not recruiting
        • Novartis Investigative Site
      • London, United Kingdom, WC1N 3JH
        • Active, not recruiting
        • Novartis Investigative Site
    • Surrey
      • Sutton, Surrey, United Kingdom, SM2 5PT
        • Active, not recruiting
        • Novartis Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Active, not recruiting
        • Phoenix Children s Hospital
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Active, not recruiting
        • Childrens National Hospital
    • Florida
      • Miami, Florida, United States, 33155
        • Active, not recruiting
        • Nicklaus Childrens Hospital
    • Indiana
      • Indianapolis, Indiana, United States, 46202-2810
        • Active, not recruiting
        • Indiana Uni School of Medicine
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Active, not recruiting
        • Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Active, not recruiting
        • Dana Farber Cancer Institute
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Active, not recruiting
        • University of Minnesota
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center
        • Contact:
        • Principal Investigator:
          • Stephen Gilheeney
      • New York, New York, United States, 10065
        • Active, not recruiting
        • Memorial Sloan Kettering Cancer Ctr
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Active, not recruiting
        • Cinn Children Hosp Medical Center
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Active, not recruiting
        • St Jude Childrens Research Hospital
    • Texas
      • Houston, Texas, United States, 77030
        • Completed
        • Texas Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

All Subjects:

  • Written informed consent, according to local guidelines, signed by the subjects and/or by the parents or legal guardian prior to any study related screening procedures are performed.
  • Participation in a Novartis sponsored study such as CTMT212X2101, CDRB436G2201, CDRB436A2102, regardless of current age.
  • Parent study (or cohort of parent study) is planned to be closed.
  • Subject has demonstrated compliance, as assessed by the investigator, within the parent study protocol requirement(s).
  • Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.

For Subjects Entering the Treatment Period:

  • Subject is currently receiving treatment with dabrafenib/trametinib monotherapy or combination within a Novartis Sponsored Drug Development study. Note that subjects who were on the chemotherapy arm of the CDRB436G2201 study are eligible for treatment period of this study only after crossing over into the experimental treatment arm of the CDRB436G2201 study
  • In the opinion of the investigator is likely to benefit from continued treatment.

Key Exclusion Criteria:

All Subjects:

- Subject has participated in a combination trial where dabrafenib and/or trametinib was dispensed in combination with another study medication.

For Subjects Entering the Treatment Period:

  • Subject has permanently discontinued from study treatment in the parent protocol due to any reason.
  • Treatment with dabrafenib and/or trametinib for the subject's indication is approved for marketing and the appropriate dosage form is commercially available and reimbursed in the local country
  • Subject currently has unresolved drug related severe toxicities for which dabrafenib and/or trametinib dosing has been interrupted in the parent study. If the subject should meet criteria to resume treatment on the parent protocol then they may be eligible for treatment in this study.

Other protocol-defined inclusion/exclusion may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dabrafenib and/or trametinib

Patients in this study may receive one of the following treatments received in the parent study which are:

  • Patients who received monotherapy of either of dabrafenib or trametinib

  • Patients who received combination of dabrafenib and trametinib

    • Patients who discontinued treatment on parent study are still offered to participate in long-term follow-up
Drug: dabrafenib
dabrafenib oral, twice daily
Other Names:
  • DRB436
Drug: trametinib
trametinib oral, once daily
Other Names:
  • TMT212

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with Adverse Events and Serious Adverse Events (SAEs)
Time Frame: Baseline up to approximately 7 years
To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.
Baseline up to approximately 7 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of participants with height (measured by cm or in) changes over time
Time Frame: Baseline up to approximately 7 years
Developmental monitoring: Serial measurements of height will be collected throughout the study
Baseline up to approximately 7 years
Percentage of participants with weight (measured by kg or lb) changes over time
Time Frame: Baseline up to approximately 7 years
Developmental monitoring: Serial measurements of weight will be collected throughout the study
Baseline up to approximately 7 years
Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time
Time Frame: Baseline up to approximately 7 years
Developmental monitoring: Serial measurements of skeletal maturation will be collected throughout the study
Baseline up to approximately 7 years
Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time
Time Frame: Baseline up to approximately 7 years
Developmental monitoring: Serial measurements of sexual maturation will be collected throughout the study
Baseline up to approximately 7 years
Percentage of participants with cardiac function (measured by ECG) changes over time
Time Frame: Baseline up to approximately 7 years
Developmental monitoring: Serial measurements of cardiac function will be collected throughout the study
Baseline up to approximately 7 years
Clinical Benefit (measured by CT/MRI)
Time Frame: Baseline up to approximately 7 years
Disease specific clinical benefit, as determined by investigator using institutional standard of care. Investigator will measure tumor response based on the Response Assessment used for the subject in the parent protocol (Response Assessment in Neuro-Oncology (RANO) criteria for solid tumors, RECIST, NF1 Volumetric, Neuroblastoma, or LCH criteria).
Baseline up to approximately 7 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 4, 2019

Primary Completion (Estimated)

November 5, 2026

Study Completion (Estimated)

November 5, 2026

Study Registration Dates

First Submitted

May 30, 2019

First Submitted That Met QC Criteria

June 4, 2019

First Posted (Actual)

June 5, 2019

Study Record Updates

Last Update Posted (Actual)

June 2, 2026

Last Update Submitted That Met QC Criteria

June 1, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDRB436G2401
  • 2023-509276-42-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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