Clinical trial NCT03975829: Pediatric Long-Term Follow-up and Rollover Study
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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
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 Glioneuronal Tumor
Central Neurocytoma
Extraventricular Neurocytoma
Cerebellar Liponeurocytoma
Neurofibromatosis Type 1
Keywords: v600-mutation
neuroblastoma
Trametinib
pediatrics
Langerhans Cell Histiocytosis
low grade glioma
plexiform neurofibromas
high grade glioma
dabrafenib
NF-1
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2019-06-05
Enrollment: 164
Sex: ALL
Minimum age (years): 1
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility 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.

Primary outcomes:
measure: Number of participants with Adverse Events and Serious Adverse Events (SAEs)
timeFrame: Baseline up to approximately 7 years
Secondary outcomes:
measure: Percentage of participants with height (measured by cm or in) changes over time
timeFrame: Baseline up to approximately 7 years
measure: Percentage of participants with weight (measured by kg or lb) changes over time
timeFrame: Baseline up to approximately 7 years
measure: Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time
timeFrame: Baseline up to approximately 7 years
measure: Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time
timeFrame: Baseline up to approximately 7 years
measure: Percentage of participants with cardiac function (measured by ECG) changes over time
timeFrame: Baseline up to approximately 7 years
measure: Clinical Benefit (measured by CT/MRI)
timeFrame: Baseline up to approximately 7 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children s Hospital
state: Arizona
city: Washington
country: United States
facility: Childrens National Hospital
state: District of Columbia
city: Miami
country: United States
facility: Nicklaus Childrens Hospital
state: Florida
city: Indianapolis
country: United States
facility: Indiana Uni School of Medicine
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cincinnati
country: United States
facility: Cinn Children Hosp Medical Center
state: Ohio
city: Memphis
country: United States
facility: St Jude Childrens Research Hospital
state: Tennessee
city: Houston
country: United States
facility: Texas Childrens Hospital
state: Texas
city: Caba
country: Argentina
facility: Novartis Investigative Site
state: Buenos Aires
city: Darlinghurst
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Parkville
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Barretos
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Vancouver
country: Canada
facility: Novartis Investigative Site
state: British Columbia
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Brno Bohunice
country: Czechia
facility: Novartis Investigative Site
city: Praha 5
country: Czechia
facility: Novartis Investigative Site
city: Copenhagen
country: Denmark
facility: Novartis Investigative Site
city: Tampere
country: Finland
facility: Novartis Investigative Site
city: Rennes
country: France
facility: Novartis Investigative Site
state: Bretagne
city: Brest
country: France
facility: Novartis Investigative Site
city: Marseille
country: France
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Rennes
country: France
facility: Novartis Investigative Site
city: Vandoeuvre Les Nancy
country: France
facility: Novartis Investigative Site
city: Villejuif
country: France
facility: Novartis Investigative Site
city: Augsburg
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Hamburg
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
city: Petach Tikva
country: Israel
facility: Novartis Investigative Site
city: Firenze
country: Italy
facility: Novartis Investigative Site
state: FI
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Torino
country: Italy
facility: Novartis Investigative Site
state: TO
city: Setagaya-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Osaka
country: Japan
facility: Novartis Investigative Site
city: Utrecht
country: Netherlands
facility: Novartis Investigative Site
state: CS
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Valencia
country: Spain
facility: Novartis Investigative Site
city: Stockholm
country: Sweden
facility: Novartis Investigative Site
city: Sutton
country: United Kingdom
facility: Novartis Investigative Site
state: Surrey
city: Liverpool
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: