Official Title
Managed Access Program (MAP) to Provide Access to Alpelisib for Patients With HR-positive, HER2-negative Advanced Breast Cancer With Mutated Phosphoinositide 3-kinase Who Progressed on or After AI Treatment
Brief Summary

The purpose of this Cohort Treatment Plan is to allow access to alpelisib for patients diagnosed with HR-positive, HER2-negative advanced breast cancer with mutated phosphoinositide 3-kinase who progressed on or after AI treatment. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Intermediate-size Population
HR+, HER2-, Advanced Breast Cancer

Drug: alpelisib
Alpelisib is provided as 50 mg and 200 mg film coated tablets as individual patient supply, packaged in bottles. Alpelisib will be dosed on a flat scale of mg/day and not be adjusted to body weight or body surface area. Alpelisib will be administered at a starting dose of 300 mg orally once daily on a continuous dosing schedule and can be adjusted for toxicity per the recommendations in this treatment plan.

Eligibility Criteria

Inclusion Criteria:

Patients eligible for inclusion in this Treatment Plan have to meet all of the following

Written patient informed consent must be obtained prior to start of treatment. 1. Patient is an adult male or female (≥ 18 years of age) with advanced (locoregionally recurrent not amenable to curative therapy or metastatic) hormone receptor-positive, HER2-negative breast cancer 2. Patient has documented evidence of a mutation in the PIK3CA gene as determined in tumor tissue or plasma by a local laboratory. 3. If female, then the patient is postmenopausal. Postmenopausal status is defined either by: - Prior bilateral oophorectomy - Age ≥60 - Age 470 msec at screening (mean of triplicate ECGs).

10. Participant has any other concurrent severe and/or uncontrolled medical condition that would, in the physician's judgment, contraindicate participation in the MAP (e.g., chronic active hepatitis [testing not mandatory unless required by local regulations or requirements], severe hepatic impairment, etc.).

11. Participant is currently receiving any of the following medications and cannot be discontinued 7 days prior to the start of the treatment: - Strong CYP3A4 inducers - Inhibitors of BCRP.

12. Participant has a history of acute pancreatitis within 1 year of screening or past medical history of chronic pancreatitis.

13. Participant with unresolved osteonecrosis of the jaw.

14. Participant has a history of severe cutaneous reaction, such as Stevens-Johnson Syndrome (SJS), Erythema Multiforme (EM), Toxic Epidermal Necrolysis (TEN), or Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS).

15. Participant has received radiotherapy ≤ 4 weeks or limited field radiation for palliation ≤ 2 weeks prior to treatment, and who has not recovered to grade 1 or better from related side effects of such therapy (with the exception of alopecia).

16. Participant is currently receiving or has received systemic corticosteroids ≤ 2 weeks prior to starting drug, or who have not fully recovered from side effects of such treatment. Note: The following uses of corticosteroids are permitted: single doses, topical applications (e.g., for rash), inhaled sprays (e.g., for obstructive airways diseases), eye drops or local injections (e.g., intra-articular).

17. Participant has not recovered from all toxicities related to prior anticancer therapies to NCI CTCAE version 4.03 Grade ≤1. Exception to this criterion: participant with any grade of alopecia are allowed to enter the MAP.

18. Participant has a concurrent malignancy or malignancy within 3 years of start of treatment, with the exception of adequately treated, basal or squamous cell carcinoma, non-melanomatous skin cancer, or curatively resected cervical cancer.

19. Participant has central nervous system (CNS) involvement which was not previously treated and not fulfilling the following 3 criteria to be eligible for the MAP: - Completed prior therapy (including radiation and/or surgery) for CNS metastases ≥ 28 days prior to the treatment and - CNS tumor is clinically stable at the time of treatment start and - Participant is not receiving steroids and/or enzyme inducing anti-epileptic medications for brain metastases.

20. Subject has any other concurrent severe and/or uncontrolled medical condition that would, in the physician's judgment, contraindicate subject participation in MAP (e.g., chronic active hepatitis [testing not mandatory unless required by local regulations or requirements], severe hepatic impairment.

21. Participant is not able to understand and to comply with instructions and requirements, including oral administration of treatment.

22. Participant is a nursing (lactating) or pregnant as confirmed by a positive serum (hCG) test prior to initiating treatment.

23. Participant is a woman of child-bearing potential defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during treatment and at least for 1 week after the last dose of the treatment. Highly effective contraception methods include: - Total abstinence (when this is in line with the preferred and usual lifestyle of the participant). Periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception - Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least 6 weeks before taking 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 participants the vasectomized male partner should be the sole partner for that participant - Use of oral (estrogen and progesterone), injected or implanted combined hormonal method of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or forms of hormonal contraception that have comparable efficacy (failure rate

Eligibility Gender
Eligibility Age
Minimum: 18 Years~Maximum: 99 Years

Novartis Pharmaceuticals

Novartis Pharmaceuticals

Novartis Pharmaceuticals
NCT Number
hormone receptor positive
breast cancer
PIK3CA gene
Expanded Access
advanced breast cancer
stage IV
MeSH Terms
Breast Neoplasms