Compassionate Use of BYL 719 Alpelisib

NCT03941782 | Unknown

• 1 other identifier: INST UNM 1601
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This is a compassionate use protocol of BYL719 (alpelisib) treatment for a single patient with locally advanced lymphangioma positive PI3K alpha H1047R mutation.

Conditions

Lymphangioma

Interventions

Alpelisib DRUG

As a single agent, alpelisib will be administered at a starting dose of 350 mg orally once daily on a continuous dosing schedule and can be adjusted for toxicity per the recommendations in this protocol. No pretreatment will be given prior to the medication.

Also known as: BYL719

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has signed the Informed Consent (ICF) prior to any eligibility evaluations being performed and is able to comply with protocol requirements
• Patient is an adult and ≥ 18 years old at the time of informed consent
• Patient has locally advanced or metastatic cancer resistant or refractory to available standard of care treatment options and has no other available comparable or satisfactory alternative treatment options
• PIK3CA mutation, or other molecular alteration known to activate PI3K, in tumor tissue as determined by a standard 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, and after considering other options (e.g. trial extensions, amendments, etc.), the treating physician has determined that treatment is necessary and there are no other feasible alternatives for the patient
• Patient is not being transferred from an ongoing clinical trial for which they are still eligible
• 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)

You may not qualify if:

• Patients eligible for this Treatment Plan must not meet any of the following criteria:
• Patient has history of hypersensitivity to any drugs or metabolites of similar chemical classes as alpelisib
• Patient has not recovered to grade 1 or better (except alopecia) from related side effects of any prior antineoplastic therapy
• Patient has had major surgery within 4 weks prior to starting treatment with alpelisib or has not recovered from major side effects
• Patient is currently receiving or has received systemic corticosteroids ≤ 2 weeks prior to starting treatment with alpelisib, or has not fully recovered from side effects of such treatment
• Patient with clinically manifest diabetes mellitus, or documented steroid induced diabetes mellitus
• Patient is being treated at start of treatment with alpelisib with any of the following drugs:
• Drugs known to be strong inhibitors or inducers of isoenzyme CYP3A4 including herbal medications, or drugs with a known risk to induce Torsades de Pointes (See Appendix 1, Table 2 for a full list of prohibited medications).
• 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 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 HIV infection (testing not mandatory) infection
• Patient has any of the following cardiac abnormalities:
• a. Symptomatic congestive heart failure i. History of documented congestive heart failure (New York Heart Association functional classification III-IV), documented cardiomyopathy ii. Left Ventricular Ejection Fraction (LVEF) \<50% as determined by Multiple Gated acquisition (MUGA) scan or echocardiogram (ECHO) b. Myocardial infarction ≤ 6 months prior to enrolment c. Unstable angina pectoris Acute coronary angioplasty, or stenting), \< 3 months prior to screening coronary syndromes (including myocardial infarction, unstable angina, coronary artery bypass graft (CABG).
• d. Serious uncontrolled cardiac arrhythmia History or current evidence of clinically significant cardiac arrhythmias, atrial fibrillation and/or conduction abnormality, e.g. congenital long QT syndrome, high-grade/complete AV-blockage: per guidelines.

Sponsors & Collaborators

• New Mexico Cancer Research Alliance: lead

Study Sites (1)

University of New Mexico - Cancer Center

Albuquerque, New Mexico, 87106, United States

Location

Related Publications (1)

• Shaheen MF, Tse JY, Sokol ES, Masterson M, Bansal P, Rabinowitz I, Tarleton CA, Dobroff AS, Smith TL, Bocklage TJ, Mannakee BK, Gutenkunst RN, Bischoff J, Ness SA, Riedlinger GM, Groisberg R, Pasqualini R, Ganesan S, Arap W. Genomic landscape of lymphatic malformations: a case series and response to the PI3Kalpha inhibitor alpelisib in an N-of-1 clinical trial. Elife. 2022 Jul 5;11:e74510. doi: 10.7554/eLife.74510.

  PMID: 35787784 DERIVED

MeSH Terms

Conditions

Lymphangioma

Interventions

Alpelisib

Condition Hierarchy (Ancestors)

Neoplasm, Lymphatic Tissue; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Ian Rabinowitz, MD

  University of New Mexico Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2019
• First Posted: May 8, 2019
• Last Updated: January 11, 2024

Record last verified: 2024-01

Locations

US (1)