Pediatric Long-Term Follow-up and Rollover Study

Glioblastoma Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03975829
• Other study ID #: CDRB436G2401
• Secondary ID: 2018-004459-19
• Status: Recruiting
• Phase: Phase 4
• Start date: November 4, 2019
• Est. completion date: May 29, 2026

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: 1-888-669-6682
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: May 29, 2026
• Est. primary completion date: May 29, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Drug:
• dabrafenib
dabrafenib oral, twice daily
• trametinib
trametinib oral, once daily

Locations

Country	Name	City	State
Argentina	Novartis Investigative Site	Caba	Buenos Aires
Australia	Novartis Investigative Site	Darlinghurst	New South Wales
Australia	Novartis Investigative Site	Parkville	Victoria
Belgium	Novartis Investigative Site	Bruxelles
Brazil	Novartis Investigative Site	Barretos	SP
Brazil	Novartis Investigative Site	Sao Paulo	SP
Brazil	Novartis Investigative Site	Sao Paulo	SP
Canada	Novartis Investigative Site	Montreal	Quebec
Canada	Novartis Investigative Site	Toronto	Ontario
Canada	Novartis Investigative Site	Vancouver	British Columbia
Czechia	Novartis Investigative Site	Brno
Czechia	Novartis Investigative Site	Praha 5
Denmark	Novartis Investigative Site	Copenhagen
Finland	Novartis Investigative Site	Tampere
France	Novartis Investigative Site	Brest Cedex	Finistere
France	Novartis Investigative Site	Marseille Cedex 05
France	Novartis Investigative Site	Paris
France	Novartis Investigative Site	Rennes	Bretagne
France	Novartis Investigative Site	Vandoeuvre Les Nancy
France	Novartis Investigative Site	Villejuif
Germany	Novartis Investigative Site	Augsburg
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Essen
Germany	Novartis Investigative Site	Hamburg
Germany	Novartis Investigative Site	Koeln
Israel	Novartis Investigative Site	Petach-Tikva
Italy	Novartis Investigative Site	Firenze	FI
Italy	Novartis Investigative Site	Genova	GE
Italy	Novartis Investigative Site	Milano	MI
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Torino	TO
Japan	Novartis Investigative Site	Osaka
Japan	Novartis Investigative Site	Setagaya-ku	Tokyo
Netherlands	Novartis Investigative Site	Utrecht	CS
Russian Federation	Novartis Investigative Site	Moscow
Spain	Novartis Investigative Site	Madrid
Spain	Novartis Investigative Site	Valencia
Sweden	Novartis Investigative Site	Stockholm
United Kingdom	Novartis Investigative Site	Liverpool
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	Sutton	Surrey
United States	Johns Hopkins University IDS Pharmacy	Baltimore	Maryland
United States	Dana Farber Cancer Institute .	Boston	Massachusetts
United States	Cinn Children Hosp Medical Center	Cincinnati	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Indiana Uni School of Medicine .	Indianapolis	Indiana
United States	St Jude Childrens Research Hospital .	Memphis	Tennessee
United States	Nicklaus Childrens Hospital	Miami	Florida
United States	University of Minnesota .	Minneapolis	Minnesota
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Phoenix Children's Hospital .	Phoenix	Arizona
United States	Childrens National Hospital CQTI571A2306	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Belgium,  Brazil,  Canada,  Czechia,  Denmark,  Finland,  France,  Germany,  Israel,  Italy,  Japan,  Netherlands,  Russian Federation,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with Adverse Events and Serious Adverse Events (SAEs)	To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.	Baseline up to approximately 7 years
Secondary	Percentage of participants with height (measured by cm or in) changes over time	Developmental monitoring: Serial measurements of height will be collected throughout the study	Baseline up to approximately 7 years
Secondary	Percentage of participants with weight (measured by kg or lb) changes over time	Developmental monitoring: Serial measurements of weight will be collected throughout the study	Baseline up to approximately 7 years
Secondary	Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time	Developmental monitoring: Serial measurements of skeletal maturation will be collected throughout the study	Baseline up to approximately 7 years
Secondary	Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time	Developmental monitoring: Serial measurements of sexual maturation will be collected throughout the study	Baseline up to approximately 7 years
Secondary	Percentage of participants with cardiac function (measured by ECG) changes over time	Developmental monitoring: Serial measurements of cardiac function will be collected throughout the study	Baseline up to approximately 7 years
Secondary	Clinical Benefit (measured by CT/MRI)	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