MedPath

Phase Ib/II Trials of RAD001 in Triple Negative Metastatic Breast Cancer

Phase 1
Terminated
Conditions
Metastatic Breast Cancer
Interventions
Registration Number
NCT01939418
Lead Sponsor
National Cancer Center, Korea
Brief Summary

This study consists of two parts. In a phase Ib part, investigators will explore the recommended dose of gemcitabine, cisplatin, and RAD001 combination in patients with metastatic TNBC. After completing the phase Ib part, investigators will review the data and discuss with Novartis before the start of a phase II part. In the phase II part, investigators will compare the efficacy of the gemcitabine and cisplatin with or without RAD001 in patients with metastatic TNBC.

Detailed Description

PIK3CA active mutations are the most frequent genetic event in breast cancer, including in TNBC which presents activated PI3K/AKT signaling due to PIK3CA mutation or PTEN deficiency. TNBC cell lines having activated PI3K/AKT signaling showed a high sensitivity to PI3K/mTOR inhibitors. RAD001 is a potent mTOR complex 1 inhibitor and showed to enhance cisplatin or gemcitabine induced apoptosis by inhibiting p53 induced p21 expression.

This study consists of two parts. In a phase Ib part, investigators will explore the recommended dose of gemcitabine, cisplatin, and RAD001 combination in patients with metastatic TNBC. After completing the phase Ib part, investigators will review the data and discuss with Novartis before the start of a phase II part. In the phase II part, investigators will compare the efficacy of the gemcitabine and cisplatin with or without RAD001 in patients with metastatic TNBC.

Recruitment & Eligibility

Status
TERMINATED
Sex
Female
Target Recruitment
23
Inclusion Criteria
  • Females with histologically confirmed, metastatic or stage IV breast cancer
  • ER/PgR negative or poor (Allred score ≤ 3/8) and HER2 negative breast cancer
  • ECOG performance status 0-2
  • Age ≥ 20 years
  • Previously treated by anthracycline and taxane in adjuvant/neoadjuvant or metastatic setting
  • ≤ 2 chemotherapy regimens for metastatic disease
  • Radiological or objective evidence of recurrence or progression on or after the last systemic therapy prior to enrolment.
  • CNS metastasis is permitted if asymptomatic and not requiring treatment with steroids and is documented to be non-progressing at study entry
  • Presence of measurable or evaluable disease by RECIST 1.1 criteria
  • Adequate hematopoietic function: Absolute granulocyte count ≥1,500/mm3, platelet ≥100,000/mm3, hemoglobin ≥ 10g/mm3
  • Adequate hepatic function: total bilirubin ≤ 1.5 x upper normal limit (UNL), AST/ALT ≤2.5 x UNL or ≤5 x UNL if presented with hepatic metastasis
  • Fasting serum cholesterol ≤ 300mg/dl and fasting triglycerides ≤ 2.5 x UNL
  • Adequate renal function: Serum creatinine ≤1.5mg/dL
  • Patients should sign a written informed consent before study entry
  • Patients with positive HBV-DNA of HBsAg at screening must initiate prophylaxis with appropriate antiviral medication at least one week prior to treatment start
Read More
Exclusion Criteria
  • Known active CNS metastasis
  • Patients who received prior therapy with gemcitabine
  • Patients with only non-measurable lesions other than bone metastasis (e.g. pleural effusion, ascites).
  • Patients with more than 3 prior chemotherapy lines for treating metastatic breast cancer.
  • Patients who received prior therapy with mTOR inhibitor or PI3K inhibitor
  • Known hypersensitivity to mTOR inhibitors, e.g. Sirolimus (rapamycin).
  • Radiotherapy within four weeks prior to enrolment, except radiotherapy to the bone for analgesic purpose or for lytic lesions at risk of fracture. Patients must have recovered from radiotherapy toxicities prior to enrolment.
  • Patients who have history of cancer other than in situ uterine cervix cancer or nonmelanotic skin cancer
  • Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral everolimus
  • Active ulceration of upper gastrointestinal tract
  • Other concurrent severe and/or uncontrolled conditions (e.g. uncontrolled diabetes mellitus, active untreated or uncontrolled infection, chronic obstructive or chronic restrictive pulmonary disease including dyspnea at rest from any cause) that could cause unacceptable safety risks or compromise compliance with the protocol.
  • Patients with a known history of HIV seropositivity. Screening for HIV infection at baseline is not required.
  • Significant symptomatic deterioration of lung function. If clinically indicated, pulmonary function tests including measures of predicted lung volumes, DLco, O2 saturation at rest on room air should be considered to exclude restrictive pulmonary disease, pneumonitis or pulmonary infiltrates.
  • Patients being treated with drugs recognized as being strong inhibitors or inducers of the isoenzyme CYP3A at enrolment (rifabutin, rifampicin, clarithromycin, ketoconazole, itraconazole, voriconazole, ritonavir, telithromycin) continuously for at least 7 days during any time period in the last 2 weeks prior to enrolment
  • Known hypersensitivity to protocol treatment
  • Pregnant or breast feeding
  • Peripheral neuropathy ≥ grade 2 (NCI CTCAE version 4.0) at randomization
  • Patients unwilling to or unable to comply with the protocol
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
RAD001RAD001gemcitabine 800mg/m2, D1 and D8 iv. every 3 weeks. cisplatin 30mg/m2, D1 and D8 iv. every 3 weeks. RAD001 5mg QD. po.
RAD001Cisplatingemcitabine 800mg/m2, D1 and D8 iv. every 3 weeks. cisplatin 30mg/m2, D1 and D8 iv. every 3 weeks. RAD001 5mg QD. po.
RAD001Gemcitabinegemcitabine 800mg/m2, D1 and D8 iv. every 3 weeks. cisplatin 30mg/m2, D1 and D8 iv. every 3 weeks. RAD001 5mg QD. po.
Primary Outcome Measures
NameTimeMethod
The recommended dose of the combination of gemcitabine, cisplatin and RAD001 (everolimus) in patients with metastatic triple-negative breast cancerup to 1 year

phase IB part

Efficacy of gemcitabine and cisplatin with or without RAD001 in patients with metastatic triple-negative breast cancer by evaluating progression free survival (PFS)up to 5 years

phase II part

Secondary Outcome Measures
NameTimeMethod
check biomarkers associated with the response of RAD001: angiogenesis, metabolism, immune cells profilesup to 5 years

phaseIb and phaseII

number of patients with adverse events as a measure of safety and tolerabilityup to 5 years

phase Ib and phase II

The maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of gemcitabine/cisplatin/RAD001up to 1 year

phase Ib part

Overall survival (OS)up to 5 years

phase Ib and phase II

objective response rateup to 1 year

phase Ib and phase II

Trial Locations

Locations (1)

National cancer center

🇰🇷

Goyangsi, Gyeonggido, Korea, Republic of

© Copyright 2025. All Rights Reserved by MedPath