Pediatric Long-Term Follow-up and Rollover Study

An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)

Sponsors

Lead Sponsor: Novartis Pharmaceuticals

Source Novartis
Brief Summary

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

Overall Status Recruiting
Start Date November 4, 2019
Completion Date May 29, 2026
Primary Completion Date May 29, 2026
Phase Phase 4
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of participants with Adverse Events and Serious Adverse Events (SAEs) Baseline up to approximately 7 years
Secondary Outcome
Measure Time Frame
Percentage of participants with height (measured by cm or in) changes over time Baseline up to approximately 7 years
Percentage of participants with weight (measured by kg or lb) changes over time Baseline up to approximately 7 years
Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time Baseline up to approximately 7 years
Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time Baseline up to approximately 7 years
Percentage of participants with cardiac function (measured by ECG) changes over time Baseline up to approximately 7 years
Clinical Benefit (measured by CT/MRI) Baseline up to approximately 7 years
Enrollment 250
Condition
Intervention

Intervention Type: Drug

Intervention Name: dabrafenib

Description: dabrafenib oral, twice daily

Arm Group Label: Dabrafenib and/or trametinib

Other Name: DRB436

Intervention Type: Drug

Intervention Name: trametinib

Description: trametinib oral, once daily

Arm Group Label: Dabrafenib and/or trametinib

Other Name: TMT212

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.

Gender: All

Minimum Age: 1 Year

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Novartis Pharmaceuticals Study Director Novartis Pharmaceuticals
Overall Contact

Last Name: Novartis Pharmaceuticals

Phone: 1-888-669-6682

Email: [email protected]

Location
Facility: Status: Contact: Investigator:
Phoenix Childrens Hospital | Phoenix, Arizona, 85016, United States Recruiting 602-546-0895 Lindsey Hoffman Principal Investigator
Johns Hopkins University IDS Pharmacy | Baltimore, Maryland, 21287, United States Recruiting Tammy Scott [email protected] Kenneth J Cohen Principal Investigator
Dana Farber Cancer Institute | Boston, Massachusetts, 02215, United States Recruiting Diana Cukali 617-632-4907 [email protected] Karen Wright Principal Investigator
University of Minnesota | Minneapolis, Minnesota, 55455, United States Recruiting 651-220-6000 Christopher Moertel Principal Investigator
Memorial Sloan Kettering Cancer Center | New York, New York, 10065, United States Recruiting Jessica Sollitto 212-639-3112 [email protected] Stephen Gilheeney Principal Investigator
Cincinnati Children s Hospital Medical Center | Cincinnati, Ohio, 45229-3039, United States Recruiting 800-344-2462 Brian Weiss Principal Investigator
St Jude Children's Research Hospital | Memphis, Tennessee, 38105, United States Recruiting Melissa Johnson 901-521-9005 [email protected] Santhosh Upadhyaya Principal Investigator
Novartis Investigative Site | Toronto, Ontario, M5G 1X8, Canada Recruiting
Novartis Investigative Site | Rennes, Bretagne, 35203, France Recruiting
Novartis Investigative Site | Marseille Cedex 5, 13385, France Recruiting
Novartis Investigative Site | Paris, 75231, France Recruiting
Novartis Investigative Site | Vandoeuvre Les Nancy, 54511, France Recruiting
Novartis Investigative Site | Villejuif Cedex, 94800, France Recruiting
Novartis Investigative Site | Madrid, 28009, Spain Recruiting
Novartis Investigative Site | Sutton, Surrey, SM2 5PT, United Kingdom Recruiting
Novartis Investigative Site | London, WC1N 3JH, United Kingdom Recruiting
Location Countries

Canada

France

Spain

United Kingdom

United States

Verification Date

March 2021

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Dabrafenib and/or trametinib

Type: Experimental

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

Patient Data Yes
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov

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