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OpenTrials
Active, Not Recruiting

NCT Number: NCT03975829

Pediatric Long-Term Follow-up and Rollover Study

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Diffuse Astrocytoma Anaplastic Astrocytoma Anaplastic Ganglioglioma Anaplastic Oligodendroglioma Anaplastic Pleomorphic Xanthoastrocytoma Angiocentric Glioma Astrocytoma Brain Diseases Brain Neoplasms Central Nervous System Diseases Central Nervous System Neoplasms Central Neurocytoma Cerebellar Liponeurocytoma Chordoid Glioma of Third Ventricle Congenital, Hereditary, and Neonatal Diseases and Abnormalities Desmoplastic Infantile Astrocytoma and Ganglioglioma Dysplastic Gangliocytoma of Cerebrellum Extraventricular Neurocytoma Gangliocytoma Ganglioglioma Ganglioneuroma Genetic Diseases, Inborn Giant Cell Astrocytoma Glioblastoma Glioma Hemic and Lymphatic Diseases Heredodegenerative Disorders, Nervous System Histiocytosis Histiocytosis, Langerhans-Cell Lung Diseases Lung Diseases, Interstitial Lymphatic Diseases Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Nervous System Neoplasms Neuroblastoma Neurocutaneous Syndromes Neurocytoma Neurodegenerative Diseases Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral Neurofibroma Neurofibroma, Plexiform Neurofibromatoses Neurofibromatosis 1 Neurofibromatosis Type 1 Neuromuscular Diseases Oligodendroglioma Oligodendroglioma, Childhood Papillary Glioneuronal Tumor Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms Pilocytic Astrocytoma Pleomorphic Xanthoastrocytoma Respiratory Tract Diseases Rosette-forming Glioneuronal Tumor

Age range

1 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Novartis Investigative Site, CABA, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Dabrafenib

Drug

dabrafenib oral, twice daily

Other names: DRB436

Trametinib

Drug

trametinib oral, once daily

Other names: TMT212

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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).

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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)

Important dates

Study start
2019
Primary completion
2026
Study completion
2026
First posted
Jun 5, 2019
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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