Compassionate Use Program BRAF Mutation-positive Patients in the Adjuvant Treatment of Melanoma After Surgical Resection


Study First Submitted Date 2020-09-03
Study First Posted Date 2020-09-10
Last Update Posted Date 2023-05-03
Verification Month Year September 2022
Verification Date 2022-09-30
Last Update Posted Date 2023-05-03

Detailed Descriptions

Sequence: 20713293
Description The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication. This program will provide access to patients until: All participating countries have received marketing authorization and product is commercially available and accessible to all participating patient(s) or Alternative treatment options are available and/or In case of changes in the safety profile or a lack of overall efficacy of the product.

Browse Interventions

Sequence: 96013376 Sequence: 96013377 Sequence: 96013378 Sequence: 96013379 Sequence: 96013380 Sequence: 96013381
Mesh Term Trametinib Mesh Term Dabrafenib Mesh Term Antineoplastic Agents Mesh Term Protein Kinase Inhibitors Mesh Term Enzyme Inhibitors Mesh Term Molecular Mechanisms of Pharmacological Action
Downcase Mesh Term trametinib Downcase Mesh Term dabrafenib Downcase Mesh Term antineoplastic agents Downcase Mesh Term protein kinase inhibitors Downcase Mesh Term enzyme inhibitors Downcase Mesh Term molecular mechanisms of pharmacological action
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 52148321 Sequence: 52148322
Name Melanoma Name Adjuvant
Downcase Name melanoma Downcase Name adjuvant

Id Information

Sequence: 40142003
Id Source org_study_id
Id Value CDRB436F2001CM


Sequence: 52464152 Sequence: 52464153 Sequence: 52464154
Intervention Type Drug Intervention Type Drug Intervention Type Drug
Name Dabrafenib and Trametinib Name Dabrafenib Name Trametinib
Description The starting dose of the combination treatment will be administered as follows: Dabrafenib, 150 mg, twice daily (BID); Trametinib, 2.0 mg, once daily (QD) Description If administration of dabrafenib is interrupted or permanently discontinued, administration of trametinib may continue. Description If administration of trametinib is interrupted or permanently discontinued, administration of dabrafenib may be continued.


Sequence: 79835361 Sequence: 79835362 Sequence: 79835363 Sequence: 79835364 Sequence: 79835365 Sequence: 79835366 Sequence: 79835367 Sequence: 79835368 Sequence: 79835369 Sequence: 79835370 Sequence: 79835371 Sequence: 79835372 Sequence: 79835373 Sequence: 79835374 Sequence: 79835375 Sequence: 79835376
Name Melanoma Name Adjuvant Name BRAF V600 Name Malignant skin cancer Name Skin cancer Name Stage III melanoma Name Cutaneous Name Resection Name Surgical resection Name Mutation Name Compassionate Use Name Dabrafenib Name Trametinib Name DRB436 Name TMT212 Name Cohort
Downcase Name melanoma Downcase Name adjuvant Downcase Name braf v600 Downcase Name malignant skin cancer Downcase Name skin cancer Downcase Name stage iii melanoma Downcase Name cutaneous Downcase Name resection Downcase Name surgical resection Downcase Name mutation Downcase Name compassionate use Downcase Name dabrafenib Downcase Name trametinib Downcase Name drb436 Downcase Name tmt212 Downcase Name cohort

Browse Conditions

Sequence: 193401370 Sequence: 193401371 Sequence: 193401372 Sequence: 193401373 Sequence: 193401374 Sequence: 193401375 Sequence: 193401376 Sequence: 193401377
Mesh Term Melanoma Mesh Term Neuroendocrine Tumors Mesh Term Neuroectodermal Tumors Mesh Term Neoplasms, Germ Cell and Embryonal Mesh Term Neoplasms by Histologic Type Mesh Term Neoplasms Mesh Term Neoplasms, Nerve Tissue Mesh Term Nevi and Melanomas
Downcase Mesh Term melanoma Downcase Mesh Term neuroendocrine tumors Downcase Mesh Term neuroectodermal tumors Downcase Mesh Term neoplasms, germ cell and embryonal Downcase Mesh Term neoplasms by histologic type Downcase Mesh Term neoplasms Downcase Mesh Term neoplasms, nerve tissue Downcase Mesh Term nevi and melanomas
Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48298731
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Novartis Pharmaceuticals

Central Contacts

Sequence: 12003301 Sequence: 12003302
Contact Type primary Contact Type backup
Name Novartis Pharmaceuticals Name Novartis Pharmaceuticals
Phone +41613241111
Role Contact Role Contact


Sequence: 30752678
Gender All
Minimum Age 1 Year
Maximum Age 100 Years
Healthy Volunteers No
Criteria Inclusion Criteria: Has or is willing to give consent to the Treating Physician in accordance with the local regulatory requirements. Has the following diagnosis: Completely resected (R0) histologically confirmed high-risk (stage III) cutaneous melanoma with confirmed BRAF V600E/K activating mutation. Patients presenting with initial resectable lymph node recurrence after a diagnosis of Stage I or II melanoma are eligible. Patients with an unknown primary melanoma are not eligible. All clinical trials that the patient might qualify for have been ruled out. Is receiving care at a clinical site with a Treating Physician who has experience with administering investigational agents for patients with this condition, or the patient is willing and/or able to travel to a site and receive treatment under the guidance of physician with this experience. Is able to retain oral medication and swallow tablets/capsules (appropriate exceptions allowed for patients who are unable to swallow tablets/capsules – this is subject to availability of alternative (liquid) oral formulations). Is not eligible for participation in any of the IMP’s ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (e.g., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 3 (or equivalent) and is in stable clinical condition. NOTE: patient in rapidly deteriorating clinical condition prior to start of therapy should not be considered for this program. Does not require treatment with prohibited concomitant medications (please refer to the IB or approved label/SmPC). Women of childbearing potential must have had a negative serum β-human chorionic gonadotropin (HCG) pregnancy test within 7 days prior to starting dabrafenib and trametinib treatment. Subjects with a positive pregnancy test result must be excluded from the program. Subjects with a negative pregnancy test result must agree to use an effective contraception method as described below throughout the treatment period and for a total of 4 months following the last dose of treatment. Contraceptive Methods for Females of Childbearing Potential: An intrauterine device with a documented failure rate of less than 1% per year Vasectomized partner who is sterile prior to the female patient’s entry into the Compassionate Use program, and this male is the sole sexual partner for that female. Complete abstinence from sexual intercourse for 14 days prior to first dose of treatment, through the dosing period, and for at least 4 months after the last dose of treatment. Abstinence is only acceptable when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods, etc.) and withdrawal are not acceptable methods of contraception. Double-barrier contraception: condom and occlusive cap (diaphragm or cervical/vault caps) with vaginal spermicidal agent (foam/gel/cream/suppository). Note: Hormonal-based methods (e.g., oral contraceptives) are not permitted as contraception due to potential drug-drug interactions with dabrafenib. Females Not of Childbearing Potential Non-childbearing potential (i.e., physiologically incapable of becoming pregnant) is defined as any female who has had a documented hysterectomy, bilateral oophorectomy (ovariectomy), bilateral tubal ligation or tubal occlusion, or is post-menopausal. A practical definition accepts menopause after 1 year without menses with an appropriate clinical profile; e.g., age appropriate, >45 years in the absence of hormone replacement therapy (HRT). In questionable cases, the patient must have a follicle stimulating hormone (FSH) value >40 mIU/mL and an estradiol value <40 pg/mL (<140 pmol/L). Female patients determined not to be post-menopausal must use adequate contraception, as defined immediately above for females of childbearing potential. Female subjects who are lactating must discontinue nursing prior to the first dose of program treatment and must refrain from nursing throughout the treatment period and for 4 months following the last dose of program treatment. If a subject becomes pregnant during the treatment period of the program, the treatments should be stopped immediately. Written patient informed consent must be obtained by the Treating Physician prior to start of treatment in accordance with the applicable local regulatory requirements. Exclusion Criteria: Patients eligible for this Treatment Plan must not meet any of the following criteria: History of hypersensitivity to any drugs or metabolites of similar chemical classes as the product. Uveal or mucosal melanoma. Evidence of metastatic disease. Female who is pregnant or nursing (patient must discontinue nursing in order to enroll in the program). NOTE: Safety and efficacy in pregnant or nursing women has not been investigated. Inclusion of pregnant or nursing woman may be considered in individually upon review by the Novartis Country Pharma Organization Medical Advisor/Director. Patients who have any lab abnormalities or AE/SAEs greater than Grade 3 (CTCAE v5.0) Concurrent treatment with other systemic anti-cancer therapies is not allowed, with the exception of surgery (other exceptions might be allowed and are subject to individual evaluation). Patients who are currently being treated with another systemic anti-cancer therapy (e.g., chemotherapy, immune, biologic, or targeted therapy) must discontinue use prior to initiation of treatment with trametinib and dabrafenib. NOTE: Radiation skin injury has been reported with concurrent use of dabrafenib and radiation. Presence of any malignancy with confirmed activating RAS mutation. NOTE: Prospective RAS testing is not required. However, if the results of previous RAS testing are known, they must be used in assessing eligibility. Has a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to trametinib or dabrafenib, or excipients or to dimethyl sulfoxide (DMSO). Any medical conditions or physical examination or clinical laboratory findings which, would put the patient at high risk for an adverse outcome. Current evidence / risk of retinal vein occlusion (RVO) or central serous retinopathy. Current evidence of cardiovascular risk including any of the following: LVEF<LLN A QT interval corrected for heart rate using the Bazett’s formula ≥ 480 msec A clinically significant uncontrolled arrhythmias Acute coronary syndrome (including myocardial infarction and unstable angina) Congestive heart failure ≥ Class II as defined by New York Heart Association Not able to understand and to comply with treatment instructions and requirements.
Adult True
Child True
Older Adult True

Calculated Values

Sequence: 254180896
Registered In Calendar Year 2020
Were Results Reported False
Has Single Facility False
Minimum Age Num 1
Maximum Age Num 100
Minimum Age Unit Year
Maximum Age Unit Years

Responsible Parties

Sequence: 28865220
Responsible Party Type Sponsor