Phase 2 study evaluating efficacy and safety of PQR309 in patients with relapsed or refractory primary CNS lymphoma

Open-label, non-randomized, phase 2 study evaluating efficacy and safety of PQR309 in patients with relapsed or refractory primary CNS lymphoma

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001306-33-ES

Enrollment

Registered

2017-01-04

Start date

2017-03-08

Completion date

Unknown

Last updated

2018-05-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Relapsed or refractory primary CNS lymphoma (PCNSL) MedDRA version: 19.0 Level: LLT Classification code 10036685 Term: Primary central nervous system lymphoma System Organ Class: 100000004864

Interventions

Product Name: PQR309 Product Code: PQR309 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not available yet CAS Number: 1225037-39-7 Current Sponsor code: PQR309 Other descriptive name: 5-(4,6

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. = 18 years of age. 2. Patient with histologically/cytologically confirmed PCNSL at first progression. 3. Relapsed or refractory PCNSL demonstrated by cranial MRI. 4. Presence of at least one lesion of bi-dimensionally measurable disease on baseline MRI with a contrast-enhancing tumor of at least 1 cm (10 mm) in the longest diameter. 5. Maximum two prior systemic therapy regimens excluding high dose chemotherapy at first relapse. 6. If receiving corticosteroids, patients must have been on a stable or decreasing dose of corticosteroids and no more than 8 mg dexamethasone (or equivalent) for at least 5 days prior to date of enrollment. 7. Karnofsky Performance Score (KPS) = 70%. 8. More than 4 weeks from any investigational agent (at the judgment of the investigator and in agreement with lead investigator and PIQUR). Adequate haematological, 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. 9. Able and willing to swallow and retain oral medication. 10. Female and male patients of reproductive potential must agree to use effective contraception from screening until 90 days after discontinuing study treatment. 11. Willing and able to sign the informed consent and to comply with the protocol for the duration of the study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3

Exclusion criteria

Exclusion criteria: 1. Secondary CNS lymphoma or chronic immunosuppresion-associated CNS lymphoma. 2. Previous allogeneic hematopoietic stem cell transplant (HSCT transplant). 3. Previous whole brain radiotherapy (WBRT). 4. Other concomitant anti-tumor therapy as determined by the study team. 5. Patients unable to undergo contrast-enhanced MRI. 6. Prior treatment with a PI3K inhibitor, AKT inhibitor, or mTOR inhibitor. 7. Patient taking enzyme-inducing anti-epileptic drug (EIAED) 7.0 mmol/L (126 mg/dL).

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the clinical efficacy of PQR309 in the treatment of patients with relapsed or refractory primary CNS lymphoma.;Secondary Objective: To evaluate: - Overall safety and tolerability of PQR309 - Overall clinical efficacy of PQR309 - Pharmacokinetics (PK) of PQR309 in plasma;Primary end point(s): ORR including complete response (CR), unconfirmed complete (CRu) and partial response (PR) according to the 2005 Response Criteria of the International Primary CNS Lymphoma Collaborative Group (IPCG);Timepoint(s) of evaluation of this end point: 4 and 8 weeks after first intake of PQR309, subsequently every 8 weeks	—

Measure

Time frame

Main Objective: To evaluate the clinical efficacy of PQR309 in the treatment of patients with relapsed or refractory primary CNS lymphoma.;Secondary Objective: To evaluate: - Overall safety and tolerability of PQR309 - Overall clinical efficacy of PQR309 - Pharmacokinetics (PK) of PQR309 in plasma;Primary end point(s): ORR including complete response (CR), unconfirmed complete (CRu) and partial response (PR) according to the 2005 Response Criteria of the International Primary CNS Lymphoma Collaborative Group (IPCG);Timepoint(s) of evaluation of this end point: 4 and 8 weeks after first intake of PQR309, subsequently every 8 weeks

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Secondary

Measure	Time frame
Secondary end point(s): Safety: - Incidence and severity of AEs including SAEs - Changes in vital signs (pulse rate, blood pressure, body temperature), Karnofsky performance status (KPS), physical examinations, body weight, ECG, PHQ-9 questionnaire and GAD-7 mood scale score - Changes of routine laboratory assessments (haematology, blood chemistry, urinalysis) - Changes in Mini-Mental State Examination (MMSE) score Additional Clinical Efficacy: - Time to response (TTR), duration of response (DOR), time to treatment failure (TTF), progression-free survival (PFS) and survival rate at 1 year Pharmacokinetics: - PQR309 plasma concentration, PK parameters;Timepoint(s) of evaluation of this end point: AE monitoring: at Weeks 1, 2, 3, 4, 6 and 7 (and every three weeks later), and at 30 days after the last dose Vital signs: at Screening, at Weeks 1, 2, 3, 4 and 7 (and every three weeks later), at the end of treatment and 30 days after the last dose Haematology & Blood chemistry: at Screening, at Weeks 1, 2, 3, 4, 6 and 7 (and every three weeks later), and at 30 days after the last dose Urinalysis: at Screning, at Weeks 1, 4 and 7 (and every three weeks later), and the end of treatment MMSE: at Screening and at the end of treatment PK sampling: at Weeks 1 (Day 1 and 2), 2, 3, 4 and 7 (and every three weeks later) (depending on dosing schedule)	—

Measure

Time frame

Secondary end point(s): Safety: - Incidence and severity of AEs including SAEs - Changes in vital signs (pulse rate, blood pressure, body temperature), Karnofsky performance status (KPS), physical examinations, body weight, ECG, PHQ-9 questionnaire and GAD-7 mood scale score - Changes of routine laboratory assessments (haematology, blood chemistry, urinalysis) - Changes in Mini-Mental State Examination (MMSE) score Additional Clinical Efficacy: - Time to response (TTR), duration of response (DOR), time to treatment failure (TTF), progression-free survival (PFS) and survival rate at 1 year Pharmacokinetics: - PQR309 plasma concentration, PK parameters;Timepoint(s) of evaluation of this end point: AE monitoring: at Weeks 1, 2, 3, 4, 6 and 7 (and every three weeks later), and at 30 days after the last dose Vital signs: at Screening, at Weeks 1, 2, 3, 4 and 7 (and every three weeks later), at the end of treatment and 30 days after the last dose Haematology & Blood chemistry: at Screening, at Weeks 1, 2, 3, 4, 6 and 7 (and every three weeks later), and at 30 days after the last dose Urinalysis: at Screning, at Weeks 1, 4 and 7 (and every three weeks later), and the end of treatment MMSE: at Screening and at the end of treatment PK sampling: at Weeks 1 (Day 1 and 2), 2, 3, 4 and 7 (and every three weeks later) (depending on dosing schedule)

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Countries

Belgium, Canada, France, Germany, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States

Contacts

Public ContactChief Operating Officer

PIQUR Therapeutics AG

frances.betts@piqur.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026