An open label, multi-center roll-over study to assess longterm effect in pediatric patients treated with Tafinlar (dabrafenib) and/or Mekinist (trametinib).

M
Michael Ferguson, MD

Primary Investigator

Administratively Closed
1 year - 100 years
All
Phase 4
250 participants needed
1 Location

Brief description of study

What is the purpose of this study?
This is an open-label, multi-center, global long-term follow-up and roll-over study designed to facilitate collection of information on the long-term outcomes of pediatric subjects who have been treated on Novartis sponsored trials with dabrafenib, trametinib or the combination. Additionally, this study will provide continued access of medication to subjects who have previously participated in a dabrafenib and/or trametinib treatment study (parent study) and who have shown or are still showing continued investigator-determined clinical benefit to treatment.

Dabrafenib and trametinib are investigational, meaning they may not have been approved by the Food and Drug Administration (FDA), European Medicines Agency (EMA) or other regulatory agency in your region for people with certain medical condition. The medicines dabrafenib taken with trametinib are approved by the FDA, EMA and other regulatory agencies for doctors to prescribe to adult patients with specific types of cancer (such as a type of melanoma and lung cancer).

Only eligible patients that have been referred by a physician or self-referred to and seen by section of Pediatric Hematology/Oncology staff will be invited to participate in the clinical trial. Potential subjects will be contacted in the hospital or at a clinic visit in Pediatric Hematology/Oncology.

THIS STUDY IS ENROLLING BY INVITATION ONLY - Consistent with most oncology trials, patients are not actively “recruited,” but are screened by their physician for appropriate clinical trial(s) at the time of their routine clinic visit. Occasionally, a patient may be a self-referral or physician referral, but are still screened for appropriate clinical trials at the time of their routine clinic visit. PI and staff may send copies of relevant consent forms to these patients to look over prior to actually consenting or enrolling them. This may take place at the patient's visit at which the consent is presented or the patient's next visit to the outpatient hematology/oncology clinic.

Interested in participating? For more information about this research study or other cancer-related clinical trials at IU Simon Comprehensive Cancer Center, please contact:
IU Clinical Trials Office 
Phone: (317) 278-5632

Detailed description of study

What will happen during the study?
  • If the patient is currently on treatment in the parent protocol and would benefit from continued treatment (per investigator determination), this study will offer continued access to study treatment(s). 
  • Study treatment(s) or study medication(s) refers to dabrafenib monotherapy, trametinib monotherapy or combination therapy of dabrafenib taken with trametinib. 
  • Patients will use the same study treatment(s) based on the parent protocol and the starting dose should be the same dosage and frequency the patient was receiving at the last visit or at the completion of the parent study.
  • If a patient has discontinued study treatment and is still in follow-up on the parent protocol, he/she will be eligible for continued follow-up on this study but will not be eligible to receive study treatment(s) in this study.
  • At the study visits, the following procedures should happen: physical examination, dermatological examination, best corrected visual acuity (vision test), ophthalmic examination, tanner staging, and the collection of blood and urine samples.  
  • Those study participants on study treatment(s) may also have bone age (x-ray), ECHO/MUGA and ECG.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneurona Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
  • Age: 1 year - 100 years
  • Gender: All

Inclusion Criteria:
All Subjects:
  • Written informed consent, according to local guidelines, signed by the subjects and/orby the parents or legal guardian prior to any study related screening procedures ared.
  • 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 theudy protocol requirement(s).
  • Willingness and ability to comply with scheduled visits, treatment plans and any otherudy procedures.
For Subjects Entering the Treatment Period:
  • Subject is currently receiving treatment with dabrafenib/trametinib monotherapy orbination within a Novartis Sponsored Drug Development study. Note that subjects whowere on the chemotherapy arm of the CDRB436G2201 study are eligible for treatmentd of this study only after crossing over into the experimental treatment arm ofhe 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 wasdispensed in combination with another study medication.
For Subjects Entering the Treatment Period:
  • Subject has permanently discontinued from study treatment in the parent protocol duey reason.
  • Treatment with dabrafenib and/or trametinib for the subject's indication is approvedketing and the appropriate dosage form is commercially available and reimbursedhe local country
  • Subject currently has unresolved drug related severe toxicities for which dabrafenibd/or trametinib dosing has been interrupted in the parent study. If the subjecthould meet criteria to resume treatment on the parent protocol then they may begible for treatment in this study.
Other protocol-defined inclusion/exclusion may apply.

Updated on 01 Aug 2024. Study ID: CDRB436G2401, PHO-NOVARTIS-CDRB436G2401, 17768

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