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出境医 / 临床实验 / Pediatric Long-Term Follow-up and Rollover Study
Pediatric Long-Term Follow-up and Rollover Study
Study Description
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
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 250 participants |
| Allocation: | N/A |
| 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 : | May 29, 2026 |
| Estimated Study Completion Date : | May 29, 2026 |
Arms and Interventions
| 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:
|
Drug: dabrafenib
dabrafenib oral, twice daily
Other Name: DRB436
Drug: trametinib trametinib oral, once daily
Other Name: TMT212
|
Outcome Measures
Primary Outcome Measures :
- 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 :
- 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
- 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
- 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
- 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
- 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
- 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
| 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
Contacts
| Contact: Novartis Pharmaceuticals | 1-888-669-6682 | novartis.email@novartis.com | |
| Contact: Novartis Pharmaceuticals | +41613241111 |
Locations
| United States, Arizona | |
| Phoenix Childrens Hospital | Recruiting |
| Phoenix, Arizona, United States, 85016 | |
| Contact 602-546-0895 | |
| Principal Investigator: Lindsey Hoffman | |
| United States, District of Columbia | |
| Children s National Hospital CQTI571A2306 | Recruiting |
| Washington, District of Columbia, United States, 20010 | |
| Contact 202-476-6083 | |
| Principal Investigator: Lindsay Kilburn | |
| United States, Maryland | |
| Johns Hopkins University IDS Pharmacy | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Tammy Scott scottta@jhmi.edu | |
| Principal Investigator: Kenneth J Cohen | |
| United States, Massachusetts | |
| Dana Farber Cancer Institute | Recruiting |
| Boston, Massachusetts, United States, 02215 | |
| Contact: Diana Cukali 617-632-4907 Diana_cukali@dfci.harvard.edu | |
| Principal Investigator: Karen Wright | |
| United States, Minnesota | |
| University of Minnesota | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| Contact 651-220-6000 | |
| Principal Investigator: Christopher Moertel | |
| United States, New York | |
| Memorial Sloan Kettering Cancer Center | Recruiting |
| New York, New York, United States, 10065 | |
| Contact: Jessica Sollitto 212-639-3112 dunkel@MSKCC.ORG | |
| Principal Investigator: Stephen Gilheeney | |
| United States, Ohio | |
| Cincinnati Children s Hospital Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45229-3039 | |
| Contact 800-344-2462 | |
| Principal Investigator: Brian Weiss | |
| 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 | |
| Canada, Ontario | |
| Novartis Investigative Site | Recruiting |
| Toronto, Ontario, Canada, M5G 1X8 | |
| France | |
| Novartis Investigative Site | Recruiting |
| Rennes, Bretagne, France, 35203 | |
| Novartis Investigative Site | Recruiting |
| Marseille Cedex 5, France, 13385 | |
| Novartis Investigative Site | Recruiting |
| Paris, France, 75231 | |
| Novartis Investigative Site | Recruiting |
| Vandoeuvre Les Nancy, France, 54511 | |
| Novartis Investigative Site | Recruiting |
| Villejuif Cedex, France, 94800 | |
| Spain | |
| Novartis Investigative Site | Recruiting |
| Madrid, Spain, 28009 | |
| United Kingdom | |
| Novartis Investigative Site | Recruiting |
| Sutton, Surrey, United Kingdom, SM2 5PT | |
| Novartis Investigative Site | Recruiting |
| London, United Kingdom, WC1N 3JH | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date ICMJE | May 30, 2019 | ||||||||
| First Posted Date ICMJE | June 5, 2019 | ||||||||
| Last Update Posted Date | April 19, 2021 | ||||||||
| Actual Study Start Date ICMJE | November 4, 2019 | ||||||||
| Estimated Primary Completion Date | May 29, 2026 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
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.
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Pediatric Long-Term Follow-up and Rollover Study | ||||||||
| Official Title ICMJE | An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib) | ||||||||
| Brief Summary | A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib. | ||||||||
| Detailed Description | Not Provided | ||||||||
| Study Type ICMJE | Interventional | ||||||||
| Study Phase ICMJE | Phase 4 | ||||||||
| Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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| Condition ICMJE |
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| Intervention ICMJE |
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| Study Arms ICMJE | Experimental: Dabrafenib and/or trametinib
Patients in this study may receive one of the following treatments received in the parent study which are:
Interventions:
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE |
250 | ||||||||
| Original Estimated Enrollment ICMJE | Same as current | ||||||||
| Estimated Study Completion Date ICMJE | May 29, 2026 | ||||||||
| Estimated Primary Completion Date | May 29, 2026 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE |
Key Inclusion Criteria: All Subjects:
For Subjects Entering the Treatment Period:
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:
Other protocol-defined inclusion/exclusion may apply. |
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| Sex/Gender ICMJE |
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| Ages ICMJE | 1 Year and older (Child, Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | Canada, France, Spain, United Kingdom, United States | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT03975829 | ||||||||
| Other Study ID Numbers ICMJE | CDRB436G2401 2018-004459-19 ( EudraCT Number ) |
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| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | Novartis ( Novartis Pharmaceuticals ) | ||||||||
| Study Sponsor ICMJE | Novartis Pharmaceuticals | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| PRS Account | Novartis | ||||||||
| Verification Date | April 2021 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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