This study has been transitioned to CTIS with ID 2023-509276-42-00 check the CTIS register for the current data. Primary:To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.Secondary:To assess the long-term…
ID
Source
Brief title
Condition
- Miscellaneous and site unspecified neoplasms malignant and unspecified
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Frequency and severity of (serious) adverse events.
Secondary outcome
Efficacy parameters according to the local standards, at least every 6 months.
Background summary
This is a global single-arm, open-label, multi-center study to collect data on
the long-term effects of dabrafenib, trametinib or the combination in pediatric
subjects who have been treated on Novartis sponsored trials. No formal
hypothesis will be tested. Additionally, this study will provide continued
access to study medication(s) for subjects who have previously participated in
dabrafenib and/or trametinib treatment studies (parent studies):
CDRB436G2201 (The only study that was performed in the Netherland): Phase II
open-label global study to evaluate the effect of dabrafenib in combination
with trametinib in children and adolescent patients with BRAF V600-mutation
positive Low Grade Glioma (LGG) or relapsed or refractory High Grade Glioma
(HGG).
CDRB436A2102: Phase I/IIa, 2-part, multi-center, single-arm, open-label study
to determine the safety, tolerability and pharmacokinetics of oral dabrafenib
in children and adolescent patients with advanced BRAF V600-mutation positive
solid tumors.
CTMT212X2101: Pharmacodynamics and clinical activity of the MEK inhibitor
trametinib in children and adolescents patients with cancer or plexiform
neurofibromas and trametinib in combination with dabrafenib in children and
adolescents with cancers harboring V600 mutation.
The participants were aged between 1 and 17 years of age at the time of
enrollment in the parent study.
Dabrafenib (trade name Tafinlar) is a BRAF-inhibitor. It is a targeted cancer
inhibiting therapy. Trametinib (trade name Mekinist) is a MEK-inhibitor; a
targeted cancer inhibiting therapy as well. The combination of both drugs has
been granted marketing authorization in the EU for the treatment of adults with
certain melanomas and metastatic non- small cell lung cancer. Treatment of
children and adolescents has not received marketing approval yet.
This study is part of the development program of dabrafenib and trametinib for
children and adolescents up to and including 17 years of age.
Study objective
This study has been transitioned to CTIS with ID 2023-509276-42-00 check the CTIS register for the current data.
Primary:
To assess the long-term safety of treatment with dabrafenib, trametinib or the
combination.
Secondary:
To assess the long-term effect of treatment with dabrafenib, trametinib or the
combination on general health, growth and development.
To assess efficacy as determined by institutional standard of care procedures.
Study design
This is a global single-arm, open-label, multi-center study to collect data on
the long-term effects of dabrafenib, trametinib or the combination in pediatric
subjects who have been treated on Novartis sponsored trials. No formal
hypothesis will be tested. Additionally, this study will provide continued
access to study medication(s) for subjects who have previously participated in
dabrafenib and/or trametinib treatment studies (parent studies).
Intervention
Treatment with the combination of dabrafenib and trametinib, as in the parent
study CDRB436G2201, the only parent study that was performed in NL). Subjects
are to use the study treatment based on the parent protocol. The starting dose
should be the same dosage the subject was receiving at the last visit or at the
completion of the parent study.
Study burden and risks
Risk: Adverse events of the study medication.
Burden:
During the treatment period:
• Contact every 3 months. Yearly: month 3 and 9: by phone or in person; month 6
and 12: in the hospital.
• Physical examination month 6, 12. Dermatological examination month 12. Tanner
score month 12.
• Blood tests (fasting, approx. 15 mL) month 6, 12.
• Pregnancy test (if relevant) every month (if necessary at home, result to be
communicated to investigator by phone).
• Urine test month 12.
• Best corrected visual acuity month 6, 12.
• EKG and echocardiography (or MUGA-scan) month 6, 12.
• X-ray wrist or tibia (for bone age) month 12.
During follow-up period when study treatment has been discontinued and disease
has not progressed:
• Hospital visit every 12 weeks.
• Physical examination, including dermatological examination and Tanner score.
• Blood tests.
• EKG.
• Bone age.
Optional during or after the study:
• Use of data and remaining body material for other research.
During follow-up period when study treatment has been discontinued and disease
has progressed:
• Every 6 to 12 months phone call.
Haaksbergweg 16
Amsterdam 1101 BX
NL
Haaksbergweg 16
Amsterdam 1101 BX
NL
Listed location countries
Age
Inclusion criteria
Subjects eligible for inclusion in this study must meet all of the following
criteria:
All subjects
1. Written informed consent, according to local guidelines, signed by the
patients and / or by the parents or legal guardian prior to any study related
screening procedures are performed.
2. Participation in a Novartis sponsored study such as TMT212X2101,
DRB436G2201, DRB436A2102, regardless of current age
3. Parent study (or cohort of parent study) is planned to be closed
4. Patient has demonstrated treatment compliance, as assessed by the
Investigator, within the parent study protocol requirement(s).
5. Willingness and ability to comply with scheduled visits, treatment plans and
any other study procedures.
For Subjects Entering the Treatment Period
6. Patient is currently receiving treatment with dabrafenib/trametinib
monotherapy or combination within a Novartis Sponsored Drug Development study.
7. In the opinion of the Investigator, the subject is likely to benefit from
continued treatment.
8. Does not require treatment with prohibited concomitant medications.
Exclusion criteria
1. Patient has participated in a combination trial where dabrafenib and/or
trametinib was dispensed in combination with another study medication.
(Exception: Patients who were on the chemotherapy arm of the CDRB436G2201 study
are eligible for this study after crossing over into the experimental treatment
arm of the CDRB436G2201 study or have discontinued the study treatment and are
now in follow-up)
2. Patient has permanently discontinued from study treatment in the parent
protocol due to any reason.
3. Treatment with dabrafenib and/or trametinib for the patient*s indication is
approved for marketing and the appropriate dosage form is commercially
available and reimbursed in the local country
4. Patient currently has unresolved drug related severe toxicities for which
dabrafenib and/or trametinib dosing has been interrupted in the parent study.
If the patient should meet criteria to resume treatment on the parent protocol
then they may be eligible for enrolment in this study.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-509276-42-00 |
| EudraCT | EUCTR2018-004459-19-NL |
| ClinicalTrials.gov | NCT03975829 |
| CCMO | NL81911.041.22 |