Clinical Trial Details
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NCT02850744 : Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamic Effects of PQR309 in Glioblastoma Patients
PhasePhase 2
AgesMin: 18 Years Max: N/A
Eligibility
Inclusion Criteria:

1. Patients with histologically confirmed glioblastoma at first progression following or
during standard temozolomide chemoradiotherapy (TMZ/RTTMZ)

2. older than 18 years of age

3. Radiographic demonstration of disease progression by RANO criteria

4. Only for patients of the surgical cohort:

- Eligible for open resection of progressive tumor according to standard practice
of the study center

- Availability of adequate surgical tissue sample for the evaluation of
concentration of PQR309 in the tumor and its PD effect

- Patients treated with PQR309 after incomplete surgical resection may still have
measurable disease according to RANO criteria and may therefore be evaluable for
evaluation of response to treatment with PQR309 according to RANO criteria. The
best response in patients treated with PQR309 after complete surgical resection
is stable disease. All patients can be assessed for PFS6.

5. Only for patients of the non-surgical cohort:

- Presence of at least one lesion of bi-dimensionally measurable disease by MRI with
a contrast-enhancing tumor of at least 1 cm (10 mm) in the longest diameter on
baseline MRI is required for patients who do not undergo surgery at relapse. For
patients who undergo surgery for recurrence but do not participate in the presurgical
PQR309 dosing cohort, the same rules regarding response assessment as in the surgical
cohort apply. All patients can be assessed for PFS6.

6. Patient must have at least 1 formalin-fixed paraffin-embedded archival tumor tissue
block representative of glioblastoma available from the first surgical resection of
glioblastoma.

7. One prior systemic therapy regimen: patients must have received at least one dose of
TMZ in the first line therapy. More than 6 cycles and alternative dosing regiments of
TMZ are allowed.

8. If receiving corticosteroids, patients must have been on a stable or decreasing dose
of corticosteroids and no more than 8 mg dexamethasone equivalent for ? 5 days prior
to baseline MRI.

9. Karnofsky Performance Score (KPS) >70%.

10. More than 12 weeks from radiotherapy (RT)

11. More than 4 weeks from last administration of TMZ

12. More than 4 weeks from any investigational agent (at the judgment of the investigator
and in agreement with lead investigator and PIQUR)

13. Adequate hematological, liver and renal function defined as follows:

Absolute neutrophil count (ANC) ?1.5x109/l, platelets ? 100x109/l, hemoglobin ?
100g/L. Total bilirubin ? 1.5 times the upper limit of normal (ULN). Alanine
aminotransferase (ALT) and aspartate aminotransferase (AST) ? 2.5 times ULN Serum
Creatinine ? 1.5 times ULN

14. Able and willing to swallow and retain oral medication

15. Female and male patients of reproductive potential must agree to use effective
contraception from screening until 90 days after discontinuing study treatment*

16. Willing and able to sign the informed consent and to comply with the protocol for the
duration of the study

Exclusion Criteria:

1. Second or later glioblastoma relapse

2. Received more than one systemic treatment regimen for glioblastoma

3. Patients receiving enzyme-inducing anti-epileptic drug (EIAED) within 7 days of the
first dose of PQR309

4. Patient is taking a drug with known risk to promote QT prolongation and Torsades de
Pointes.

5. Patient is currently using herbal preparations or medications. Patient should stop
using herbal medications 7 days prior to the first dose of the study drug

6. Patients with glioblastoma known to contain IDH1 or 2 mutation

7. Other concomitant anti-tumor therapy as determined by the study team

8. Prior treatment with intracerebral agents, e.g. prolifeprospan 20 with carmustine
wafer

9. Patients unable to undergo contrast-enhanced MRI

10. Fasting glucose > 7.0 mmol/L or HbA1c > 6.4%.

11. 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 (e.g. risk of doing harm to self or
others), or patients with active severe personality disorders.

12. Anxiety ?CTC AE grade 3

13. Patient has an uncontrolled intercurrent illness, including but not limited to,
ongoing or active infection, known HIV infection, chronic liver disease, chronic
renal disease, pancreatitis, chronic pulmonary disease, active cardiac disease or
cardiac dysfunction, interstitial lung disease, active autoimmune disease,
uncontrolled diabetes, neuropsychiatric or social situations that would limit
compliance with the study requirements

14. Presence of gastrointestinal disease or any other condition that could interfere
significantly with the absorption of the study drug.

15. Concomitant treatment with medicinal products that increase the pH (reduce acidity)
of the upper gastrointestinal tract, including, but not limited to, proton-pump
inhibitors (e.g. omeprazole), H2-antagonists (e.g. ranitidine) and antacids. Patients
may be enrolled in the study after a wash-out period sufficient to terminate their
effect (See section 11.2.2.8).Women who are pregnant or breast feeding,

16. Women able to conceive and unwilling to practice an effective method of birth
control* from screening until 90 days after discontinuing study treatment (women of
childbearing potential** must have a negative urine or serum pregnancy test within 7
days prior to first dose of PQR309

- Adequate contraception is defined as surgical sterilization (e.g., bilateral
tubal ligation, vasectomy), hormonal contraception (implantable, patch, oral),
or double-barrier methods (any double combination of: IUD, male or female condom
with spermicidal gel, diaphragm, sponge, cervical cap). Male patients must agree
to use condoms as contraception method.

- Child-bearing potential for the sake of this study is defined as sexually
mature women who have not undergone a hysterectomy, have not been naturally
postmenopausal for at least 12 consecutive months or have a serum FSH < 40
mIU/ml.
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