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A Multicenter Phase II Pilot Open Label Study to Evaluate the Efficacy and Safety of BKM120 in the Treatment of Patients With Advanced or Metastatic Differentiated Thyroid Cancers

Phase 2
18 Years
Open (Enrolling)
Thyroid Cancers

Thank you

Trial Information

A Multicenter Phase II Pilot Open Label Study to Evaluate the Efficacy and Safety of BKM120 in the Treatment of Patients With Advanced or Metastatic Differentiated Thyroid Cancers

The primary objective of the study will be to determine efficacy of BKM120 as measured by
the progression free survival (PFS rate) at 6 months in patients with progressive,
metastatic, refractory, follicular or poorly differentiated thyroid cancers.

Inclusion Criteria:

1. Histologically confirmed differentiated follicular or poorly differentiated thyroid

2. Patients refractory to radio iodine: i.e.; absence of radioiodine uptake in at least
one target lesion on a post-therapeutic whole body scan, presence of a target lesion
after a cumulative radio-iodine activity of at least 600 mCi, patients with
radio-iodine uptake who have progression of the disease within 12 months after
radioactive iodine (RAI) treatment

3. Metastatic or locally invasive disease

4. Patients must have at least one site of measurable disease per RECIST (version 1.1.)

5. Documented progression as per RECIST (version 1.1.) based on 2 comparative imagings
performed within the last 12 months (+20%)

6. Patients may have received two previous treatment with tyrosine kinase inhibitors but
must be off treatment within at least 4 weeks

7. Patient has signed the informed consent before any trial related activities and
according to local guidelines

8. Patient (male or female) is ≥ 18 years at the day of consenting to the study

9. Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 which
the investigator believes is stable at the time of screening

10. Patient has adequate bone marrow and organ function as defined by the following
laboratory values:

- Absolute Neutrophil Count (ANC) ≥ 1.0 x 109/L

- Platelets ≥ 100 x 109/L

- Hemoglobin ≥ 9.0 g/dL

- INR ≤ 1,5

- Potassium, calcium,and magnesium within normal limits (WNL) for theinstitution

- Serum Creatinine ≤ 1.5 x ULN

- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) within
normal range

- Total Serum bilirubin within normal range normal liver function test results,
and absence of other contributing disease processes at the time of diagnosis

- Fasting plasma glucose (FPG) ≤ 120 mg/dL or ≤ 6.7 mmol/L

- HbA1c ≤ 8 %

11. Patient has no legal protection measure

12. Patient has a health coverage

Exclusion Criteria:

1. Other histological subtypes of thyroid tumors: papillary, anaplastic, medullary,
lymphoma or sarcoma

2. Patient has received previous treatment with PI3K and/or mTOR inhibitors or AKT

3. Patient has symptomatic central nervous system (CNS) metastases. Patients with
controlled and asymptomatic CNS metastases may participate in this trial. The patient
must have completed any prior local treatment for CNS metastases ≥ 28 days (including
radiotherapy and/or surgery) prior to enrollment in this study

4. Patient has a concurrent malignancy or malignancy within 3 years of study enrollment,

5. Patient has a score ≥ 12 on the PHQ-9 questionnaire

6. Patient selects a response of "1, 2 or 3" to question number 9 on the PHQ-9
questionnaire regarding potential for suicidal thoughts or ideation

7. Patient has a GAD-7 mood scale score ≥ 15

8. Patient has a medically documented history of or active major depressive episode,
bipolar disorder (I or II), obsessive-compulsive disorder, schizophrenia, a history
of suicidal attempt or ideation, or homicidal ideation

9. Patient is concurrently using other approved or investigational antineoplastic agent

10. Patient who received wide field radiotherapy ≤ 4 weeks or limited field radiation for
palliation ≤ 2 weeks prior to starting study drug or who have not recovered to grade
1 or better from related side effects of such therapy

11. Patient has had major surgery within 14 days prior to starting study drug or has not
recovered from major side effects of the surgery

12. Patient has active cardiac disease or a history of cardiac dysfunction including any
of the following:Unstable angina pectoris within 6 months prior to study
entry,Symptomatic pericarditis,Documented myocardial infarction within 6 months prior
to study entry,History of documented congestive heart failure,Documented

13. Patient has a Left Ventricular Ejection Fraction (LVEF) < 50% as determined by
Multiple Gated acquisition (MUGA) scan or echocardiogram (ECHO)

14. Patient has any of the following cardiac conduction abnormalities:

15. Patient is currently receiving treatment with medication that has a known risk to
prolong the QT interval or inducing Torsades de Pointes, and the treatment cannot be
discontinued or switched to a different medication prior to randomization.

16. Patient has impairment of gastrointestinal (GI) function or GI disease that may
significantly alter the absorption of BKM120

17. Patient is currently receiving increasing or chronic treatment (> 5 days) with
corticosteroids or another immunosuppressive agent, as chronic administration of
corticosteroids (> 5 days) can induce CYP3A4 The following uses of corticosteroids
are permitted: single doses; topical applications, inhaled sprays, eye drops or local

18. Patient has any other concurrent severe and/or uncontrolled medical condition that
would, in the investigator's judgment contraindicate patient participation in the
clinical study

19. Patient has a history of non-compliance to medical regimen or inability to grant

20. Patient is currently receiving treatment with drugs known to be moderate and strong
inhibitors or inducers of isoenzyme CYP3A, The patient must have discontinued strong
inducers for at least one week and must have discontinued strong inhibitors before
the treatment is initiated. Switching to a different medication prior to entry in the
treatment phase is allowed.

21. Patient has a known history of HIV (testing not mandatory) infection

22. Pregnant or nursing (lactating) woman where pregnancy is defined as the state of a
female after conception and until the termination of gestation, confirmed by a
positive beta hCG laboratory test (> 5 mIU/mL)

23. Patient who does not apply highly effective contraception during the study and
through the duration as defined below after the final dose of study treatment.

24. Patient has a known hypersensitivity to any of the excipients of BKM120

25. Patient has not recovered to grade 1 or better (except alopecia) from related side
effects of any prior antineoplastic therapy

26. 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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

progression-free survival (PFS)

Outcome Description:

The progression-free survival is defined as a lack of objective tumor progression and death. Tumor progression is documented by the CT or MRI scans. Progression is defined according to RECIST criteria (version 1.1.).

Outcome Time Frame:

at 6 months after the onset of the treatment

Safety Issue:


Principal Investigator


Investigator Role:

Study Director

Investigator Affiliation:

Hospices Civils de Lyon


France: Agence Nationale de Sécurité du Médicament et des produits de santé

Study ID:




Start Date:

April 2013

Completion Date:

January 2017

Related Keywords:

  • Thyroid Cancers
  • thyroid cancers
  • refractory
  • PI3K inhibitor
  • Thyroid Neoplasms
  • Thyroid Diseases