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Pediatric Long-Term Follow-up and Rollover Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03975829
Recruitment Status : Recruiting
First Posted : June 5, 2019
Last Update Posted : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
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 : December 23, 2022
Estimated Study Completion Date : December 31, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: DRB436

Drug: trametinib
trametinib oral, once daily
Other Name: TMT212


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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).


Eligibility Criteria
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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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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         
France
Novartis Investigative Site Recruiting
Marseille Cedex 5, France, 13385
Novartis Investigative Site Recruiting
Villejuif Cedex, France, 94805
Spain
Novartis Investigative Site Recruiting
Madrid, Spain, 28009
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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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: January 18, 2020
Last Verified: January 2020
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
v600-mutation
neuroblastoma
Trametinib
pediatrics
Langerhans Cell Histiocytosis
 low grade glioma
plexiform neurofibromas
high grade glioma
dabrafenib
NF-1
Additional relevant MeSH terms:
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Glioblastoma
Glioma
Astrocytoma
Oligodendroglioma
Neurofibromatoses
Neurofibromatosis 1
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