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Pediatric Long-Term Follow-up and Rollover Study

Primary Purpose

Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma

Status
Recruiting
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
dabrafenib
trametinib
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diffuse Astrocytoma focused on measuring v600-mutation, neuroblastoma, Trametinib, pediatrics, Langerhans Cell Histiocytosis, low grade glioma, plexiform neurofibromas, high grade glioma, dabrafenib, NF-1

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Phoenix Children's Hospital .Recruiting
  • Childrens National Hospital CQTI571A2306Recruiting
  • Nicklaus Childrens HospitalRecruiting
  • Indiana University School of Medicine .Recruiting
  • Johns Hopkins University IDS Pharmacy
  • Dana Farber Cancer Institute .Recruiting
  • University of Minnesota .Recruiting
  • Memorial Sloan Kettering Cancer CenterRecruiting
  • Cinn Children Hosp Medical Center
  • St Jude Children's Research Hospital .Recruiting
  • Texas Children's HospitalRecruiting
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative SiteRecruiting
  • Novartis Investigative SiteRecruiting
  • Novartis Investigative SiteRecruiting
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative SiteRecruiting
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dabrafenib and/or trametinib

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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
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
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
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
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
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).

Full Information

First Posted
May 30, 2019
Last Updated
October 2, 2023
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT03975829
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 4, 2019 (Actual)
Primary Completion Date
May 29, 2026 (Anticipated)
Study Completion Date
July 20, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
Keywords
v600-mutation, neuroblastoma, Trametinib, pediatrics, Langerhans Cell Histiocytosis, low grade glioma, plexiform neurofibromas, high grade glioma, dabrafenib, NF-1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
250 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dabrafenib and/or trametinib
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
dabrafenib
Other Intervention Name(s)
DRB436
Intervention Description
dabrafenib oral, twice daily
Intervention Type
Drug
Intervention Name(s)
trametinib
Other Intervention Name(s)
TMT212
Intervention Description
trametinib oral, once daily
Primary Outcome Measure Information:
Title
Number of participants with Adverse Events and Serious Adverse Events (SAEs)
Description
To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.
Time Frame
Baseline up to approximately 7 years
Secondary Outcome Measure Information:
Title
Percentage of participants with height (measured by cm or in) changes over time
Description
Developmental monitoring: Serial measurements of height will be collected throughout the study
Time Frame
Baseline up to approximately 7 years
Title
Percentage of participants with weight (measured by kg or lb) changes over time
Description
Developmental monitoring: Serial measurements of weight will be collected throughout the study
Time Frame
Baseline up to approximately 7 years
Title
Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time
Description
Developmental monitoring: Serial measurements of skeletal maturation will be collected throughout the study
Time Frame
Baseline up to approximately 7 years
Title
Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time
Description
Developmental monitoring: Serial measurements of sexual maturation will be collected throughout the study
Time Frame
Baseline up to approximately 7 years
Title
Percentage of participants with cardiac function (measured by ECG) changes over time
Description
Developmental monitoring: Serial measurements of cardiac function will be collected throughout the study
Time Frame
Baseline up to approximately 7 years
Title
Clinical Benefit (measured by CT/MRI)
Description
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).
Time Frame
Baseline up to approximately 7 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Phone
1-888-669-6682
Email
novartis.email@novartis.com
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Phone
+41613241111
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Children's Hospital .
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Felicia Frank
Phone
602-546-0895
Email
ffrank@phoenixchildrens.com
First Name & Middle Initial & Last Name & Degree
Lindsey Hoffman
Facility Name
Childrens National Hospital CQTI571A2306
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie Fatil
Phone
202-476-6083
Email
mfatil2@childrensnational.org
First Name & Middle Initial & Last Name & Degree
Lindsay Kilburn
Facility Name
Nicklaus Childrens Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara Fernandez
Phone
786-624-4908
Email
barbara.fernandez@nicklaushealth.org
First Name & Middle Initial & Last Name & Degree
Ziad Khatib
Facility Name
Indiana University School of Medicine .
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202-2810
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michael Ferguson
Facility Name
Johns Hopkins University IDS Pharmacy
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Dana Farber Cancer Institute .
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Seo Won Kim
Phone
617-632-4907
Email
SeoWon_Kim@DFCI.HARVARD.EDU
First Name & Middle Initial & Last Name & Degree
Karen Wright
Facility Name
University of Minnesota .
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
651-220-6000
First Name & Middle Initial & Last Name & Degree
Christopher Moertel
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jessica Sollitto
Phone
212-639-3112
Email
dunkel@MSKCC.ORG
First Name & Middle Initial & Last Name & Degree
Stephen Gilheeney
Facility Name
Cinn Children Hosp Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
St Jude Children's Research Hospital .
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Melissa Johnson
Phone
901-521-9005
Email
Melissa.Johnson2@stjude.org
First Name & Middle Initial & Last Name & Degree
Amar Gajjar
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Najeeba Ali
Phone
832-822-1630
Email
nmali@texaschildrens.org
First Name & Middle Initial & Last Name & Degree
Patricia A Baxter
Facility Name
Novartis Investigative Site
City
Caba
State/Province
Buenos Aires
ZIP/Postal Code
C1428AQK
Country
Argentina
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Darlinghurst
State/Province
New South Wales
ZIP/Postal Code
2010
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Bruxelles
ZIP/Postal Code
1200
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Barretos
State/Province
SP
ZIP/Postal Code
14784 400
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
04829-310
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
08270-070
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Brno
ZIP/Postal Code
613 00
Country
Czechia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Praha 5
ZIP/Postal Code
150 06
Country
Czechia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Copenhagen
ZIP/Postal Code
DK-2100
Country
Denmark
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Tampere
ZIP/Postal Code
33521
Country
Finland
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Rennes
State/Province
Bretagne
ZIP/Postal Code
35203
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Brest Cedex
State/Province
Finistere
ZIP/Postal Code
29609
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Marseille Cedex 05
ZIP/Postal Code
13885
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Paris
ZIP/Postal Code
75231
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Vandoeuvre Les Nancy
ZIP/Postal Code
54511
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Villejuif
ZIP/Postal Code
94800
Country
France
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Augsburg
ZIP/Postal Code
86179
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Essen
ZIP/Postal Code
45147
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Koeln
ZIP/Postal Code
50937
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Petach-Tikva
ZIP/Postal Code
49202
Country
Israel
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Firenze
State/Province
FI
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Genova
State/Province
GE
ZIP/Postal Code
16147
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20133
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Roma
State/Province
RM
ZIP/Postal Code
00165
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Torino
State/Province
TO
ZIP/Postal Code
10126
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Setagaya-ku
State/Province
Tokyo
ZIP/Postal Code
157-8535
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Utrecht
State/Province
CS
ZIP/Postal Code
3584
Country
Netherlands
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Moscow
ZIP/Postal Code
117198
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Stockholm
ZIP/Postal Code
17176
Country
Sweden
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Sutton
State/Province
Surrey
ZIP/Postal Code
SM2 5PT
Country
United Kingdom
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Individual Site Status
Active, not recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.clinicalstudydatarequest.com

Learn more about this trial

Pediatric Long-Term Follow-up and Rollover Study

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