HR+ Advanced or Metastatic Breast CancerNCT03706573

Managed Access Program to Provide Alpelisib for Patients With HR+ Advanced or Metastatic Breast Cancer

A study testing alpelisib in people with HR+ Advanced or Metastatic Breast Cancer, run by Novartis Pharmaceuticals.

No Longer Available

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Trial summary

The purpose of this Cohort Treatment Plan is to allow access to alpelisib for eligible patients diagnosed with hormone receptor positive, advanced or metastatic breast cancer harboring a phosphatidylinositol 3-kinase (PI3K)CA mutation. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: HR+ Advanced or Metastatic Breast Cancer
What is being tested?: alpelisib
Type of treatment: Pill or oral medicine
How many people: Not given
Where: No sites listed yet
Study dates: Start not givento not given
Phase: Expanded access
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
No healthy volunteers
Adult, Older adult

Who may be able to join

Patient can and will sign the Informed Consent (ICF) prior to eligibility evaluations being performed and is able to comply with treatment plan requirements
Patient is an adult and ≥ 18 years old at the time of informed consent
Patient has locally advanced or metastatic HR+ breast cancer and no comparable or satisfactory alternative therapy is available
PIK3CA mutation in tumor tissue or ctDNA as determined by a local laboratory
Patient is not:
eligible for participation in any ongoing clinical trials with alpelisib, or has recently completed a clinical trial with alpelisib that has been terminated

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being transferred from or participating in an ongoing clinical trial, and after considering other options (e.g. trial extensions, amendments, etc.), treating physician has determined that treatment is necessary and there are no other feasible alternatives for the patient
Patient has adequate bone marrow and organ function as defined by the following laboratory values:
Absolute Neutrophil Count (ANC) ≥ 1.5 x 109/L
Platelets ≥ 100 x 109/L (For patients with hematologic malignancies involving the bone marrow, platelet count > 75 x 109/L may be acceptable)
Hemoglobin ≥ 9.0 g/dL
INR ≤ 1.5
Potassium, magnesium and calcium (corrected for albumin), within normal limits for the institution, or ≤ Grade 1 severity according to NCI-CTCAE version 4.03 if judged clinically not significant by the investigator
Serum creatinine ≤ 1.5 x ULN and/or creatinine clearance > 50% LLN (Lower Limit of Normal)
Total serum bilirubin < ULN (or ≤ 1.5 x ULN if liver metastases are present; or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range in patients with well documented Gilbert's Syndrome, (defined as presence of several episodes of unconjugated hyperbilirubinemia with normal CBC results including normal reticulocyte count and peripheral blood smear, normal liver function test results, and absence of other contributing disease processes at the time of diagnosis)
Alanine aminotransferase (AST) and aspartate aminotransferase (ALT) ≤ 2.5 ULN (or < 5.0 x ULN if liver metastases are present)
Fasting plasma glucose (FPG) ≤ 140mg/dL or ≤ 7.7 mmol/L* or Glycosylated Hemoglobin (HbA1c)≤ 6.4% (both criteria have to be met).
Patient is deemed by the Treating Physician to have the initiative and means to be compliant with the treatment plan (treatment and follow-up requested by the Treating Physician)

Who cannot join

Patient has history of hypersensitivity to any drugs or metabolites of PI3K inhibitor.
Patient has not recovered to grade 1 or better (except alopecia) from side effects of any prior antineoplastic therapy
Patient has had major surgery within 14 days prior to starting treatment with alpelisib or has not recovered from major side effects
Patient is currently receiving high doses of systemic corticosteroids ≤ 2 weeks prior to starting treatment with alpelisib, or has not fully recovered from side effects of such treatment. (Note: low dose corticosteroids are permitted: single doses, topical applications, inhaled sprays, eye applications or local injections, stable low dose, for patients with CNS tumors, for at least 2 weeks prior to start of alpelisib treatment.)
Patient with uncontrolled diabetes mellitus.
Patient is being treated at start of treatment with alpelisib with any of the following drugs:

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Drugs known to be strong inhibitors or inducers of isoenzyme CYP3A4 including herbal medications (list of prohibited CYP3A4 inhibitors and inducers provided in Table 13-2 in Appendix) Note: The patient must have discontinued strong inducers for at least one week and must have discontinued strong inhibitors before the treatment with alpelisib is initiated. Switching to a different medication prior to starting treatment with alpelisib is allowed.
Patient is currently receiving warfarin or other coumarin derived anti-coagulant for treatment, prophylaxis or otherwise. Therapy with heparin, low molecular weight heparin (LMWH), or fondaparinux is allowed. Patients receiving therapies listed in Table 13-2 would not be allowed.
Patients who have other concurrent severe and/or uncontrolled medical conditions that would, in the Treating Physician's judgment, contraindicate patient participation in the individual patient program (eg. active or uncontrolled severe infection, chronic active hepatitis, immuno-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure, interstitial lung disease, etc.)
Patient has a known history of Steven Johnson's syndrome or toxic epidermal necrolysis
Patient with active HIV infection (testing not mandatory) infection
Patient has any of the following cardiac abnormalities:
symptomatic congestive heart failure
history of documented congestive heart failure (New York Heart Association functional classification III-IV), documented cardiomyopathy
Left Ventricular Ejection Fraction (LVEF) <50% as determined by Multiple Gated acquisition (MUGA) scan or echocardiogram (ECHO)
myocardial infarction ≤ 6 months prior to enrolment
unstable angina pectoris
serious uncontrolled cardiac arrhythmia
symptomatic pericarditis
Patient has impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of alpelisib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)

About this study

Official title: Managed Access Program (MAP) to Provide Alpelisib for Patients With HR+ Advanced or Metastatic Breast Cancer Resistant or Refractory to Available Treatment Options

Conditions: HR+ Advanced or Metastatic Breast Cancer
Being tested: alpelisib
Keywords: byl719, adult, breast cancer, HR+ advanced cancer, HR+ metastic cancer, resistant, refractory
Medical subject headings: Breast Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

alpelisibDrug
50 mg and 200 mg film coated tablets administered orally once daily. Starting dose is 350 mg daily. Starting dose as a combination agent with endocrine partner is 300mg daily.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

First posted16 Oct 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for HR+ Advanced or Metastatic Breast CancerStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Expanded access

IDs, dates and publications

ClinicalTrials.gov ID: NCT03706573
Sponsor's protocol ID: CBYL719A0US01CM
Status last verified: Jul 2021
First submitted: 10 Oct 2018
Last updated: 19 Jul 2021
Results posted: No

Publications

Hester A, Henze F, Travi C, Harbeck N, Wuerstlein R. First Experiences with Alpelisib in Clinical Routine: Case Reports from a German Breast Center. Breast Care (Basel). 2021 Apr;16(2):129-134. doi: 10.1159/000514794. Epub 2021 Mar 15.
PubMed 34012367

Study at a glance

From the registry record, last updated by the sponsor on 19 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Managed Access Program (MAP) to Provide Alpelisib for Patients With HR+ Advanced or Metastatic Breast Cancer Resistant or Refractory to Available Treatment Options
ClinicalTrials.gov ID: NCT03706573
Acronym / sponsor ID: CBYL719A0US01CM
Conditions: HR+ Advanced or Metastatic Breast Cancer
Keywords: byl719, adult, breast cancer, HR+ advanced cancer, HR+ metastic cancer, resistant, refractory
Study type: Expanded access
Recruitment status: No Longer Available
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
First posted: 16 Oct 2018 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Who may qualify

Ages: 18 years and older
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (18 listed)

Patient can and will sign the Informed Consent (ICF) prior to eligibility evaluations being performed and is able to comply with treatment plan requirements
Patient is an adult and ≥ 18 years old at the time of informed consent
Patient has locally advanced or metastatic HR+ breast cancer and no comparable or satisfactory alternative therapy is available
PIK3CA mutation in tumor tissue or ctDNA as determined by a local laboratory
Patient is not:

Key exclusion criteria (20 listed)

Patient has history of hypersensitivity to any drugs or metabolites of PI3K inhibitor.
Patient has not recovered to grade 1 or better (except alopecia) from side effects of any prior antineoplastic therapy
Patient has had major surgery within 14 days prior to starting treatment with alpelisib or has not recovered from major side effects
Patient is currently receiving high doses of systemic corticosteroids ≤ 2 weeks prior to starting treatment with alpelisib, or has not fully recovered from side effects of such treatment. (Note: low dose corticosteroids are permitted: single doses, topical applications, inhaled sprays, eye applications or local injections, stable low dose, for patients with CNS tumors, for at least 2 weeks prior to start of alpelisib treatment.)
Patient with uncontrolled diabetes mellitus.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
alpelisib50 mg and 200 mg film coated tablets administered orally once daily. Starting dose is 350 mg daily. Starting dose as a combination agent with endocrine partner is 300mg daily.	Drug	–

Primary outcome

Not reported in the source record.

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2021.
Derived from this study: PubMed 34012367Hester A, Henze F, Travi C, Harbeck N, Wuerstlein R. First Experiences with Alpelisib in Clinical Routine: Case Reports from a German Breast Center. Breast Care (Basel). 2021 Apr;16(2):129-134. doi: 10.1159/000514794. Epub 2021 Mar 15.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

A study testing alpelisib in people with HR+ Advanced or Metastatic Breast Cancer, run by Novartis Pharmaceuticals.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 19 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03706573, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jul 2021.