Pediatric Long-Term Follow-up and Rollover Study
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ClinicalTrials.gov Identifier: NCT03975829 |
Recruitment Status :
Recruiting
First Posted : June 5, 2019
Last Update Posted : April 21, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
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 | Drug: dabrafenib Drug: trametinib | Phase 4 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 250 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib) |
Actual Study Start Date : | November 4, 2019 |
Estimated Primary Completion Date : | May 29, 2026 |
Estimated Study Completion Date : | May 29, 2026 |

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:
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Drug: dabrafenib
dabrafenib oral, twice daily
Other Name: DRB436 Drug: trametinib trametinib oral, once daily
Other Name: TMT212 |
- 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.
- 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
- 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
- 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
- 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
- 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
- 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).

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 1 Year and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03975829
Contact: Novartis Pharmaceuticals | 1-888-669-6682 | novartis.email@novartis.com | |
Contact: Novartis Pharmaceuticals | +41613241111 |
United States, Arizona | |
Phoenix Childrens Hospital | Recruiting |
Phoenix, Arizona, United States, 85016 | |
Contact 602-546-0895 | |
Principal Investigator: Lindsey Hoffman | |
United States, District of Columbia | |
Children s National Hospital CQTI571A2306 | Recruiting |
Washington, District of Columbia, United States, 20010 | |
Contact 202-476-6083 | |
Principal Investigator: Lindsay Kilburn | |
United States, Maryland | |
Johns Hopkins University IDS Pharmacy | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: Tammy Scott scottta@jhmi.edu | |
Principal Investigator: Kenneth J Cohen | |
United States, Massachusetts | |
Dana Farber Cancer Institute | Recruiting |
Boston, Massachusetts, United States, 02215 | |
Contact: Diana Cukali 617-632-4907 Diana_cukali@dfci.harvard.edu | |
Principal Investigator: Karen Wright | |
United States, Minnesota | |
University of Minnesota | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact 651-220-6000 | |
Principal Investigator: Christopher Moertel | |
United States, New York | |
Memorial Sloan Kettering Cancer Center | Recruiting |
New York, New York, United States, 10065 | |
Contact: Jessica Sollitto 212-639-3112 dunkel@MSKCC.ORG | |
Principal Investigator: Stephen Gilheeney | |
United States, Ohio | |
Cincinnati Children s Hospital Medical Center | Recruiting |
Cincinnati, Ohio, United States, 45229-3039 | |
Contact 800-344-2462 | |
Principal Investigator: Brian Weiss | |
United States, Tennessee | |
St Jude Children's Research Hospital | Recruiting |
Memphis, Tennessee, United States, 38105 | |
Contact: Melissa Johnson 901-521-9005 Melissa.Johnson2@stjude.org | |
Principal Investigator: Santhosh Upadhyaya | |
Canada, Ontario | |
Novartis Investigative Site | Recruiting |
Toronto, Ontario, Canada, M5G 1X8 | |
France | |
Novartis Investigative Site | Recruiting |
Rennes, Bretagne, France, 35203 | |
Novartis Investigative Site | Recruiting |
Marseille Cedex 5, France, 13385 | |
Novartis Investigative Site | Recruiting |
Paris, France, 75231 | |
Novartis Investigative Site | Recruiting |
Vandoeuvre Les Nancy, France, 54511 | |
Novartis Investigative Site | Recruiting |
Villejuif Cedex, France, 94800 | |
Spain | |
Novartis Investigative Site | Recruiting |
Madrid, Spain, 28009 | |
United Kingdom | |
Novartis Investigative Site | Recruiting |
Sutton, Surrey, United Kingdom, SM2 5PT | |
Novartis Investigative Site | Recruiting |
London, United Kingdom, WC1N 3JH |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03975829 |
Other Study ID Numbers: |
CDRB436G2401 2018-004459-19 ( EudraCT Number ) |
First Posted: | June 5, 2019 Key Record Dates |
Last Update Posted: | April 21, 2022 |
Last Verified: | April 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
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. |
URL: | https://www.clinicalstudydatarequest.com |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
v600-mutation neuroblastoma Trametinib pediatrics Langerhans Cell Histiocytosis |
low grade glioma plexiform neurofibromas high grade glioma dabrafenib NF-1 |
Glioblastoma Glioma Astrocytoma Neurofibromatoses Neurofibromatosis 1 Oligodendroglioma Ganglioglioma Neurocytoma Ganglioneuroma Neoplasms Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms, Glandular and Epithelial |
Neoplasms, Nerve Tissue Neurofibroma Nerve Sheath Neoplasms Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Peripheral Nervous System Diseases Neuromuscular Diseases Nervous System Neoplasms Brain Neoplasms Central Nervous System Neoplasms Neoplasms by Site |