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A Double-blind Study of Paclitaxel in Combination With Reparixin or Placebo for Metastatic Triple-Negative Breast Cancer

Phase 2
Completed
Conditions
Metastatic Breast Cancer
Interventions
Registration Number
NCT02370238
Lead Sponsor
Dompé Farmaceutici S.p.A
Brief Summary

The Objectives of this study:

The primary objective of the study was to evaluate progression-free survival (PFS) (defined as the number of days between the date of randomization and the date of clinical disease progression (PD) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria version 1.1, as assessed by Independent Radiology Review, or death for any cause, whichever occured first) in patients with metastatic triple-negative breast cancer (TNBC) treated with the combination of paclitaxel and orally administered reparixin compared to paclitaxel alone.

The secondary objectives were:

* To determine overall survival (OS).

* To evaluate objective response rates (ORR).

* To determine median PFS (mPFS).

* To assess the safety of the combination of paclitaxel and orally administered reparixin (referred to as combination treatment).

Detailed Description

The study is a two arm, phase 2 study to evaluate the efficacy of the combination of paclitaxel and reparixin compared to paclitaxel and placebo in metastatic TNBC patients.

In the study two groups There were two groups:

Group 1: paclitaxel 80 mg/m2 intravenous (i.v.) (Days 1, 8, and 15 of 28-day cycle) + reparixin oral tablets 1200 mg three times a day (t.i.d.) continuing from Day 1 to Day 21.

Group 2: paclitaxel 80 mg/m2 i.v. (Days 1, 8, and 15 of 28-day cycle) + placebo oral tablets 1200 mg t.i.d. continuing from Day 1 to Day 21.

Study drug (reparixin/placebo) was administered with water prior to the start of the i.v. paclitaxel infusion on Cycle 1, Day 1 and then administered approximately every eight hours (six to ten hours) for 21 consecutive days during each cycle with seven days off-treatment between each cycle. It was preferable that reparixin was taken with food. However, if the patient was unable to eat, study drug was allowed to be administered. When in combination with paclitaxel (Day 1, 8 and 15 of each cycle), reparixin or placebo was administered every approximately eight hours with about 250 mL water and a light meal or snack. Paclitaxel was administered in combination with study drug (reparixin/placebo) as an i.v. infusion on Days 1, 8 and 15 of each 28-day cycle.

On Cycle 1, Day 1, paclitaxel was administered at the clinic after the administration of study drug (reparixin/placebo). From that point forward, study drug (reparixin/placebo) was self-administered t.i.d. for 21 days. Combination treatment (three weeks on and one week off) continued until PD according to RECIST criteria version 1.1, withdrawal of consent or unacceptable toxicity, whichever occurred first.

The next clinic visits were on Days 8 and 15 when a paclitaxel infusion was administered to the patient. The patients returned to the clinic again on Day 29/Day 1 of the next cycle.

Tumor response and/or progression assessments were performed and documented every eight weeks according to RECIST criteria version 1.1. Metastatic tissue samples were analyzed for evaluation of CD24-CD44+ and aldehyde dehydrogenase positive (ALDH+) CSCs.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
194
Inclusion Criteria
  1. Female aged ≥ 18 years.

  2. Patients with pathologically documented metastatic triple negative breast cancer (TNBC), eligible for treatment with paclitaxel. Paraffin-embedded tissue must be available from metastatic sites, if reasonably accessible, or from the primary tumor, to confirm the diagnosis of TNBC and for correlative studies (only on metastatic tissue). Fifteen slides can be obtained if the full block is not available to be sent or released.

    TNBC will be defined as breast cancer with <1% ER+ and <1% PgR+ cells, and HER2 immunohistochemistry score of 0 or 1+ and/or in situ hybridization (ISH) with HER2 gene copy number <4 or a ratio of less than 2 between HER2 gene copy number and centromere of chromosome 17. Patients whose metastatic disease is TNBC are eligible even when their primary tumor expressed hormone receptors and/or HER2.

  3. Patients must be newly diagnosed metastatic or must have relapsed following a prior (neo)adjuvant chemotherapy regimen. If a taxane (i.e., paclitaxel or docetaxel) was administered as part of the (neo)adjuvant regimen, PD must have occurred > 12 months from the end of previous (neo)adjuvant treatment. For non-taxane (neo)adjuvant regimen, PD must have occurred > 6 months from the end of previous (neo)adjuvant treatment

  4. Patients with at least one baseline measurable lesion according to RECIST criteria version 1.1.

  5. Zubrod (Eastern Co-operative Oncology Group [ECOG]) Performance Status (PS) of 0-1.

  6. Life expectancy of at least three months.

  7. Patients must be able to swallow and retain oral medication (intact tablet).

  8. Able to undergo all screening assessments outlined in the protocol.

  9. Adequate organ function (defined by the following parameters):

    1. Serum creatinine < 140 μmol/L (< 1.6 mg/dL) or creatinine clearance > 60 mL/min.
    2. Serum hemoglobin ≥ 9 g/dL; absolute neutrophil count ≥ 1.5 x 109/L; platelets ≥ 100 x 109/L.
    3. Serum bilirubin ≤ 1.5 x upper normal limit (UNL) except patients with Gilbert's syndrome
    4. Serum alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≤ 2.5 x UNL but ≤ 5.0 x UNL in case of liver metastases; alkaline phosphatase (ALP) ≤ UNL but i) ≤ 2.5 x UNL in case of liver metastases and ii) ≤ 5 UNL in case of bone metastases; albumin ≥ 2.5 g/dl.
  10. No history or evidence by CT scan or MRI, of brain metastases or leptomeningeal disease.

  11. No known hepatitis B virus (not due to immunization), hepatitis C virus, human immunodeficiency virus-I and -II positive status.

  12. Dated and signed IEC/IRB-approved informed consent.

Exclusion Criteria
  1. Prior therapy for metastatic TNBC (chemotherapy, hormone therapy or biological therapy), Patients may receive bisphosphonates and other therapies to treat bone metastases, however if used, bone lesions will not be considered as measurable disease.

  2. Less than four weeks since last radiotherapy (excluding palliative radiotherapy).

  3. Pregnancy or lactation or unwillingness to use adequate method of birth control.

  4. Neurological or psychiatric disorders which may influence understanding of study and informed consent procedures.

  5. Active or uncontrolled infection.

  6. Malabsorption syndrome, disease significantly affecting gastrointestinal function.

  7. G>1 pre-existing peripheral neuropathy

  8. Any other invasive malignancy from which the patient has been disease-free for less than 5 years with the exception of curatively treated basal or squamous cell skin cancer

  9. Hypersensitivity to:

    1. paclitaxel
    2. ibuprofen or to more than one non-steroidal anti-inflammatory drug.
    3. medications belonging to the class of sulfonamides, with the exception of sulfanilamides (e.g., sulfamethoxazole).

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
paclitaxel+placeboplacebopaclitaxel 80 mg/m2 i.v. (Days 1, 8, and 15) + placebo oral tablets 1200 mg t.i.d. continuing from D 1 to Day 21 of 28-day cycle
paclitaxel+reparixinpaclitaxelpaclitaxel 80 mg/m2 i.v. (Days 1, 8, and 15) + reparixin oral tablets 1200 mg t.i.d. continuing from D 1 to Day 21 of 28-day cycle
paclitaxel+reparixinReparixinpaclitaxel 80 mg/m2 i.v. (Days 1, 8, and 15) + reparixin oral tablets 1200 mg t.i.d. continuing from D 1 to Day 21 of 28-day cycle
paclitaxel+placebopaclitaxelpaclitaxel 80 mg/m2 i.v. (Days 1, 8, and 15) + placebo oral tablets 1200 mg t.i.d. continuing from D 1 to Day 21 of 28-day cycle
Primary Outcome Measures
NameTimeMethod
Progression Free Survival (PFS)Baseline up to every 8 weeks until disease progression or death, whichever occurs first, up to 721 days

PFS was defined as the number of days between the date of randomization and the date of clinical disease progression, according to RECIST criteria version 1.1, as assessed by Independent Radiology Review, or to death due to any cause, whichever occurred first.

Patients must have completed at least one course of treatment and performed at least one disease assessment to be considered evaluable for response.

Secondary Outcome Measures
NameTimeMethod
Best Overall Response (BOR)From the start of treatment, every 8 weeks, up to 56 months

BOR is defined as the best response among all overall responses (in the order complete response \[CR\], partial response \[PR\], stable disease \[SD\], and progressive disease \[PD\]) recorded as an independent review response from the start of reparixin or placebo until disease progression/recurrence or end of treatment, or death, whichever comes first. The status of BOR of PR or CR needs to be confirmed by repeat tumor assessment within no less than 4 weeks according to RECIST version 1.1. Complete Response (CR) = Disappearance of all target lesions; Partial Response (PR) = \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. If the status of CR or PR cannot be confirmed by repeat tumor assessment, the best overall response of unconfirmed CR and PR will be PR and SD, respectively. Patients must have completed at least one course of treatment and performed at least one disease assessment to be considered evaluable for response.

Number of Treatment-Emergent Adverse Events (TEAEs), Overall and by GradeThroughout the study, until off-treatment visit (performed 14 to 28 days following the last dose of study drug), up to 985 days

Treatment-emergent adverse events (TEAEs) are those which first occur or increase in severity or relationship to study drug after the first dose of study drug and before 30 days after the last dose of study treatment, reparixin/placebo. In the case of missing or partial dates, any AE that could have started on or after first dose date was assumed to be treatment-emergent. In the case of missing or partial dates, imputed dates (see section 10.1 AE date imputation) were used.

Serious AEs and Fatal AEsThroughout the study, until off-treatment visit (performed 14 to 28 days following the last dose of study drug), up to 985 days.

A serious adverse event (SAE) in human drug trials is defined as any untoward medical occurrence that at any dose

1. - results in death, (fatal)

2. - is life-threatening

3. - requires inpatient hospitalization or causes prolongation of existing hospitalization

4. - results in persistent or significant disability/incapacity,

5. - may have caused a congenital anomaly/birth defect, or

6. - requires intervention to prevent permanent impairment or damage.

Overall Survival (OS)Baseline until death due to any cause, up to 985 days

OS was defined as the time from randomization until death due to any cause. For patients who did not die, time of death was censored at the date of last contact.

Patients must have completed at least one course of treatment and performed at least one disease assessment to be considered evaluable for response.

Objective Response Rate (ORR)Baseline up to every 8 weeks until documented disease progression, up to 56 months

The ORR was defined as the percentage of patients achieving CR or PR in the Evaluable Population. The response rate was calculated from the independently reviewed assessment best response. In case of PR or CR, only confirmed cases were considered to be responses.

Complete Response (CR) = Disappearance of all target lesions; Partial Response (PR) = \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. Patients with unknown or missing response, including response of "not all evaluated" or "unable to determine", were treated as non-responders; i.e., they were included in the denominator when calculating the percentages.

Patients must have completed at least one course of treatment and performed at least one disease assessment to be considered evaluable for response.

Median Progression-free Survival (mPFS)At screening and every 8 weeks, up to 721 days

PFS was defined as the time from randomization to first documentation of disease progression, according to RECIST criteria version 1.1, as assessed by Independent Radiology Review, or to death due to any cause, whichever occurred first. For each treatment group, the Kaplan-Meier estimates for the median PFS time, the first and third quartiles were presented, along with approximate 95% confidence intervals if there were a sufficient number of progressions or deaths.

Patients must have completed at least one course of treatment and performed at least one disease assessment to be considered evaluable for response.

Duration of Overall Response (DOR)Baseline up to every 8 weeks until documented disease progression, up to 557 days

Duration of overall response (DOR) in days for the investigator assessments is measured from the time response criteria are first met for CR or PR (whichever is first recorded on the "Disease Response" page on the CRF) until either death or the first date that recurrent or PD is objectively documented (on the "Disease Response" p. on the CRF or the Follow-Up Disease Evaluation page indicates disease progression and there is supporting information in the Disease Status pages) per RECIST version 1.1. Complete Response (CR) = Disappearance of all target lesions; Partial Response (PR) = \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. If a patient is lost to follow-up with no documentation of PD, DOR was censored at the last evaluable tumor assessment. DOR was calculated only for responding patients (PR or CR) as recorded on the CRF page "Disease Response" based upon the RECIST version 1.1.

Duration of overall response wa

Trial Locations

Locations (66)

AZ St Elisabeth

🇧🇪

Namur, Belgium

Southeastern Regional Medical Center

🇺🇸

Newnan, Georgia, United States

Regional Cancer Care Associates

🇺🇸

Sparta, New Jersey, United States

Fakultni nemocnice Hradec Králové

🇨🇿

Hradec Králové, Czechia

Centre Paul Papin

🇫🇷

Angers, France

Atlanta Cancer Care

🇺🇸

Jonesboro, Georgia, United States

Azienda Ospedaliero-Universitaria

🇮🇹

Cagliari, Italy

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

🇮🇹

Meldola, Italy

Overlake Medical Center

🇺🇸

Bellevue, Washington, United States

Nemocnice Horovice a.s.

🇨🇿

Horovice, Czechia

Fakultní nemocnice v Motole, Onkologická klinika 2. LF UK a FN Motol

🇨🇿

Praha 5, Czechia

Masaryk Memorial Cancer Institute

🇨🇿

Brno, Czechia

University of Michigan Cancer Center

🇺🇸

Ann Arbor, Michigan, United States

Nuovo Ospedale

🇮🇹

Prato, Italy

Complejo Hospitalario Universitario La Coruña

🇪🇸

La Coruña, Spain

Centre hospitalier de Saint-Brieuc, Yves Le Foll

🇫🇷

La Roche sur Yon Cedex 9, France

Onkologická klinika VFN a 1.LF UK

🇨🇿

Prague 2, Czechia

Cliniques Universitaires Saint- LUC UCL

🇧🇪

Brussels, Belgium

Ospedale "Di Summa-Perrino"

🇮🇹

Brindisi, Italy

Azienda Ospedaliera, Ospedale San Carlo Borromeo

🇮🇹

Milan, Italy

Institut Paoli Calmettes

🇫🇷

Marseille cedex 9, France

Hospital Universitario Vall d'Hebron

🇪🇸

Barcelona, Spain

C. Hospital Xeral-Cies

🇪🇸

Vigo, Spain

Centre François Baclesse

🇫🇷

Caen, France

Istituto Europeo di Oncologia

🇮🇹

Milan, Italy

Wojewódzkie Centrum Onkologii

🇵🇱

Gdansk, Poland

Mrukmed. Lekarz Beata Madej Mruk i Partner. Spólka Partnerska Oddzial nr 1 w Rzeszowie

🇵🇱

Rzeszów, Poland

CHU Ambroise Paré

🇧🇪

Mons, Belgium

Hospital General Universitario Gregorio Marañon

🇪🇸

Madrid, Spain

Centro Integral Oncológico Clara Campal, Hospital de Madrid Norte-San Chinarro

🇪🇸

Madrid, Spain

Magodent Sp. z o. o.

🇵🇱

Warsaw, Poland

Centro Oncológico Regional de Galicia, Servicio de Oncologia Medica

🇪🇸

La Coruña, Galizia, Spain

Florida Cancer Specialists

🇺🇸

West Palm Beach, Florida, United States

Northside Hospital, Inc.

🇺🇸

Atlanta, Georgia, United States

CBCC Global Research a Comprehensive Blood and Cancer Center

🇺🇸

Bakersfield, California, United States

Tennessee Oncology PLLC

🇺🇸

Chattanooga, Tennessee, United States

Swedish Covenant

🇺🇸

Chicago, Illinois, United States

Waverly Hematology Oncology

🇺🇸

Cary, North Carolina, United States

Fox Valley Hematology and Oncology, SC

🇺🇸

Appleton, Wisconsin, United States

Centrum Onkologii Ziemi Lubelskiej im. sw. Jana z Dukli

🇵🇱

Lublin, Poland

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu

🇵🇱

Poznan, Poland

Bialostockie Centrum Onkologii im. Marii Sklodowskiej - Curie

🇵🇱

Bialystok, Poland

Ospedale dell'Angelo

🇮🇹

Mestre, Italy

Fondazione IRCCS Policlinico S. Matteo

🇮🇹

Pavia, Italy

Azienda Ospedaliera Ospedali Riuniti Marche Nord

🇮🇹

Pesaro, Italy

Azienda Opspedaliero Universitaria Santa Maria della Misericordia

🇮🇹

Udine, Italy

Centre Hospitalier Universitaire (CHU) De Limoges - Hopital Dupuytren

🇫🇷

Limoges, France

Medicale Centre René Gauducheau

🇫🇷

Saint Herblain cedex, France

Krajská nemocnice T.Bati, a. s.

🇨🇿

Zlin, Czechia

Centre Antoine Lacassagne

🇫🇷

Nice Cedex 2, France

Southern Cancer Center

🇺🇸

Mobile, Alabama, United States

Northside Hospital, Inc.-Georgia Cancer Specialists

🇺🇸

Sandy Springs, Georgia, United States

Mid Illinois Hematology & Oncology Associates, Ltd.

🇺🇸

Normal, Illinois, United States

Summit Medical Group

🇺🇸

Morristown, New Jersey, United States

Hematology and Oncology Associates of Northeast PA

🇺🇸

Dunmore, Pennsylvania, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

The Methodist Hospital

🇺🇸

Houston, Texas, United States

Algemeen Ziekenhuis Klina

🇧🇪

Brasschaat, Belgium

Universitair Ziekenhuis Antwerpen

🇧🇪

Edegem, Belgium

Fakultní nemocnice Královské Vinohrady

🇨🇿

Praha 10, Czechia

Azienda Ospedaliero - Universitaria, Policlinico Vittorio Emanuele

🇮🇹

Catania, Italy

Hôpital Européen Georges Pompidou

🇫🇷

Paris Cedex 15, France

Ospedale SS Giovanni e Paolo

🇮🇹

Venezia, Italy

Hospital del Mar

🇪🇸

Barcelona, Spain

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

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