Patient case: 32-year-old male with melanoma; genetic feature: NRAS (Q61H)

Clinical trial: Study to Allow Access to Pasireotide for Patients Benefiting From Pasireotide Treatment in a Novartis-sponsored Study.

Summary: The purpose of this study is to allow continued use of pasireotide in patients who are on pasireotide treatment in a Novartis-sponsored, Oncology Clinical Development & Medical Affairs (CD&MA) study and are benefiting from the treatment as judged by the investigator.

Detailed description: This is a multi-center, open label, phase IV study to provide continued supply of pasireotide to patients being treated in a current Novartis-sponsored, Oncology CD&MA study and who are benefiting from treatment with pasireotide. Eligible patients are to be consented and can then continue treatment with pasireotide in this protocol. All patients at their scheduled visits will have drug dispensing information and reported adverse events and serious adverse events collected.
  A patient will reach the end of study when pasireotide treatment is permanently discontinued and the end of treatment visit has been performed. All patients must be followed up for safety evaluations for 3 months following the last dose of pasireotide LAR treatment and for 1 month following the last dose of pasireotide s.c. treatment.
  The study is expected to remain open for approximately 10 years or until such time that enrolled patients no longer need treatment with pasireotide or are able to obtain commercial supply according to local regulations for their medical condition.

Eligibility: Inclusion Criteria:
  -  Patient is currently participating in a Novartis Oncology sponsored study receiving pasireotide (LAR and/or s.c.) and has fulfilled all required assessments in the parent study (unless the study is being terminated) and patients that are benefiting from the study drug have no other alternatives
  -  Patient is currently benefiting from the treatment with pasireotide, as determined by the investigator
  -  Patient has demonstrated compliance, as assessed by the investigator, with the parent study requirements
  -  Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures
  -  Written informed consent obtained prior to enrolling in roll-over study and receiving study medication • If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness
  Exclusion Criteria:
  -  Patient has been permanently discontinued from pasireotide study treatment in the parent study due to unacceptable toxicity, non-compliance to study procedures, withdrawal of consent or any other reason
  -  Patient has participated in a Novartis sponsored combination trial where pasireotide was dispensed in combination with another study medication and is still receiving combination therapy. (only patients receiving pasireotide monotherapy can be included)
  -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test
  -  Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 months after pasireotide s.c. last dose and 3 months after pasireotide LAR last dose Highly effective contraception methods include:
  -  Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
  -  Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
  -  Male sterilization (at least 6 months prior to screening). For female subjects on the study the vasectomized male partner should be the sole partner for that subject.
  -  Combination of any two of the following (a+b or a+c, or b+c):
  1. Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception
  2. Placement of an intrauterine device (IUD) or intrauterine system (IUS)
  3. Barrier methods of contraception: Condom or Occlusive cap diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment
  -  Sexually active males unless they use a condom during intercourse while taking drug and for 1 months after pasireotide s.c. last dose and 3 months after pasireotide LAR last dose and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid If a study patient or partner becomes pregnant or suspects being pregnant during the study or within 1 month after the final dose of pasireotide s.c. or 3 months after the final dose of pasireotide LAR, the Study Doctor needs to be informed immediately and ongoing study treatment with pasireotide has to be stopped immediately For patients taking pasireotide LAR, the future dose injections will be cancelled.

Relevance to patient case: not relevant