GINECO-EN102b - BKM120 as Monotherapy in the Treatment of Initial or Recurrent Metastatic Endometrial Cancer (ENDOPIK)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01397877
Recruitment Status : Completed
First Posted : July 20, 2011
Last Update Posted : March 17, 2016
Information provided by (Responsible Party):

Brief Summary:

This study is to determine the clinical efficacy of BKM120 as monotherapy in the treatment of initial or recurrent metastatic endometrial cancer after first line radio chemotherapy.

Clinical efficacy will be determined by the non-progression rate at 3 or 2 months depending on the group of patients. The primary endpoint is the non-progression rate at 3 months (12 weeks) for the patient group whose disease is painless (low grade tumor = stratum 1) and the non-progression rate at 2 months (8 weeks) for the group of patients with an aggressive disease (high grade tumor = stratum 2).

Disease progression is defined by the RECIST 1.1 criteria

Condition or disease Intervention/treatment Phase
Endometrial Cancer Drug: BKM120 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Multicenter Study to Assess the Safety and Efficacy of BKM120 as Monotherapy in Treatment of Initial or Recurrent Metastatic Endometrial Cancer After 1st Line Therapy in Patients Who Cannot Undergo Local Surgery and/or Radiotherapy
Study Start Date : December 2011
Actual Primary Completion Date : October 2014
Actual Study Completion Date : March 2016

Arm Intervention/treatment
Experimental: stratum 1
Patients with low grade disease (grade 1 or 2) with positive or negative mutational status
Drug: BKM120
per os, 60mg/j, until progression or unacceptable toxicity

Primary Outcome Measures :
  1. Clinical Efficacy [ Time Frame: 3 months ]
    To determine the clinical efficacy of BKM120 as monotherapy in the treatment of initial or recurrent metastatic endometrial cancer after first line radio chemotherapy.

Secondary Outcome Measures :
  1. Safety according to CTCAE v4.0 criteria and mood questionnaires : PHQ-9 and GAD-7 [ Time Frame: Patient will be followed for the duration of the study, an expected average of 75 days ]
    To assess patient safety and the tolerance of BKM120 administered as monotherapy at the daily dose of 100 mg.

  2. Efficacy: PFS [ Time Frame: 6 months ]
    To evaluate progression-free survival

  3. Efficacy: ORR [ Time Frame: Patient will be followed for the duration of the study, an expected average of 75 days ]
    To evaluate the objective response rate according to RECIST 1.1

  4. Efficacy: overall survival [ Time Frame: Patients will be followed for an expected average of 1 year and 75 days ]
    To evaluate overall survival.

  5. Efficacy: duration of response [ Time Frame: Patients will be followed for an expected average of 1 year and 75 days ]

    To evaluate the duration of the response. For patients in complete remission, the duration of the response will be calculated from the day on which a complete response is determined for the first time up to progression.

    For patients in partial remission, the duration of the response will be the overall response period calculated from the administration of the first treatment cycle up to the date of progression.

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Female ≥ 18 years
  • ECOG ≤ 2
  • Histologically confirmed endometrial cancer
  • Not eligible for exclusive curative treatment by surgery and/or radiotherapy
  • Initial metastatic endometrial cancer not treated with chemotherapy or radiotherapy prior to inclusion OR
  • Recurrent endometrial cancer previously treated with adjuvant CT and RT, presenting with a disease-free interval of at least 12 months
  • Presence of one or more measurable lesion(s) outside the irradiated areas
  • Availability at inclusion of samples of tumor tissue (a block or at least 20 unstained slides) for tumor sub-classification and for routine molecular analysis
  • Satisfactory biological functions: PNN ≥ 1.5 x 109/L, platelets ≥ 100 x 109/L, hemoglobin ≥ 9.0 g/dL, INR ≤ 2, standard normal values for potassium, calcium and magnesium, serum creatinine ≤ 1.5 x ULN or creatinine clearance > 50 mL/min, ALT and AST within normal range (or ≤ 3.0 x ULN if liver metastases present), Alkaline phosphatase ≤ 2.5 x ULN, serum bilirubin within normal range (or ≤ 1.5 x ULN if liver metastases present; or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range in patients with well documented Gilbert Syndrome), fasting glycemia ≤ 120 mg/dL or ≤ 6.7 mmol/L
  • Life expectancy 3 months
  • Post menopausal woman with at least 12 months of natural (spontaneous) amenorrhea
  • Negative serum pregnancy test ≤ 72 hours prior to initiating treatment for woman of child-bearing potential
  • Consent form signed before any procedure performed

Exclusion Criteria:

  • Previous treatment with PI3K inhibitors and/or mTOR
  • Presence of symptomatic CNS metastases. Patient must have completed any prior treatment for CNS metastases ≥ 28 days and, if on corticosteroid therapy, should be receiving a stable low dose
  • Concomitant presence or history of another malignant tumor in the past 3 years prior to inclusion (except spinocellular or cutaneous basal cell epithelioma or non-melanomatous skin cancer treated successfully)
  • Suffering from mood disorders based on an evaluation by the investigator or a psychiatrist OR with a given score according to the PHQ-9 or GAD-7 mood evaluation scale (cf protocol)
  • Concomitant administration of another approved or investigational anticancer agent
  • Pelvic and/or para-aortic radiotherapy within ≤ 28 days prior to inclusion or persistent side effects from this treatment on implementation of the selection procedures
  • Major surgery during the 28 days prior to starting investigational drug or persistent side effects from surgery
  • Uncontrolled diabetes (HbA1c > 8 %)
  • Presence of an active heart disease, especially: LVEF < 50 % determined by MUGA or ECHO, QTc > 480 msec on ECG recorded during selection (with QTcF formula), angina warranting the administration of anti-angina treatment, ventricular arrhythmia except for benign premature ventricular contractions, supraventricular and nodal arrhythmias warranting a pacemaker or not controlled by a treatment, conduction anomalies warranting a pacemaker, valvular disease with documented involvement of cardiac function, symptomatic pericarditis
  • History of heart disease
  • Currently receiving treatment to prolong QT interval accompanied by a known risk of triggering wave burst arrhythmia. Impossible to stop treatment or to replace it before starting study medication
  • GI dysfunction or disease that could significantly interfere with absorption of BKM120
  • Chronic treatment with corticosteroids or other immunosuppressants
  • Any other severe and/or uncontrolled concomitant disease, which is likely to contraindicate the patient's participation
  • Known treatment non-compliance
  • Currently receiving treatment known to be inhibitors or moderate and strong inducers of isoenzyme CYP3A. Impossible to stop this treatment or to replace it with a different treatment before starting the study product
  • Severe pneumonitis
  • Grade ≥ 3 biological anomalies
  • Known history of HIV infection
  • Pregnant woman or nursing mother

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01397877

Clinique Bonnefon
Ales, France
Centre Paul Papin
Angers, France
Institut Ste Catherine
Avignon, France
Hôpital jean Minjoz
Besancon, France
Centre Hospitalier de Blois
Blois, France
Clinique Tivoli
Bordeaux, France
Institut Bergonié
Bordeaux, France
Polyclinique Bordeaux Nord
Bordeaux, France
centre Francois baclesse
Caen, France
Centre jean Perrin
Clermont Ferrand, France
Hôpitaux Civils de Colmar
Colmar, France
Centre Georges François leclerc
Dijon, France
Group Hospitalier Mutualiste de Grenoble
Grenoble, France
Hôpital Michallon - CHU Grenoble
Grenoble, France
CHD Les Oudairies
la Roche sur Yon, France
Hôpital André Mignot
Le Chesnay, France
Centre jean Bernard
Le Mans, France
Centre Oscar Lambret
Lille, France
CHU Dupuytren
Limoges, France
Centre Léon bérard
Lyon, France
Hôpital Prové Clairval
Marseille, France
institut Paoli Calmette
Marseille, France
CRLC Val d'Aurelle
Montpellier, France, 34298
Groupement de coopération sanitaire
Montpellier, France
Centre Alexis Vautrin
Nancy, France
Centre d'oncologie de Gentilly
Nancy, France
Centre Catherine de Sienne
Nantes, France
Centre Antoine Lacassagne
Nice, France
CHU Caremeau
Nimes, France
Clinique Valdegour
Nimes, France
Centre Hospitalier Régional
Orléans, France
Hopital Hotel Dieu
Paris, France, 75004
Hopital Tenon
Paris, France, 75020
Centre Eugene Marquis
Rennes, France
Centre Frederic Joliot
Rouen, France
Centre Henri Becquerel
Rouen, France
Clinique Armoricaine de Radiologie
Saint Brieuc, France
Hôpital rené Huguenin
St Cloud, France
ICO René Gauducheau
St Herblain, France
Centre Etienne DOLET
St Nazaire, France
Institut cancérologuie de la loire
St Priest en Jarez, France
Hôpital Civil
Strasbourg, France
Centre Claudius Régaud
Toulouse, France, 31052
CHU Bretonneau
Tours, France
Institut Gustave Roussy
Villejuif, France
Sponsors and Collaborators
Principal Investigator: Isabelle Ray-Coquard, MD GINECO - Centre Léon Bérard

Responsible Party: ARCAGY/ GINECO GROUP Identifier: NCT01397877     History of Changes
Other Study ID Numbers: ENDOPIK
First Posted: July 20, 2011    Key Record Dates
Last Update Posted: March 17, 2016
Last Verified: March 2016

Keywords provided by ARCAGY/ GINECO GROUP:
endometrial cancer
initial treatment

Additional relevant MeSH terms:
Endometrial Neoplasms
Uterine Neoplasms
Genital Neoplasms, Female
Urogenital Neoplasms
Neoplasms by Site
Uterine Diseases
Genital Diseases, Female