Clinical Trial: PQR309 in Phase 2 in Patients With Relapsed or Refractory Primary Central Nervous System Lymphoma

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Open-label, Non-randomized, Phase 2 Study Evaluating Efficacy and Safety of PQR309 in Patients With Relapsed or Refractory Primary Central Nervous System Lymphoma

Brief Summary: An open-label, non-randomized, two-stage, multicenter study evaluating clinical efficacy, safety and pharmacokinetics of PQR309 in patients with relapsed or refractory Primary Central Nervous System Lymphoma (PCNSL).

Detailed Summary:

An open-label, non-randomized, two-stage, multicenter study evaluating clinical efficacy, safety, and pharmacokinetics effects of PQR309 in patients with relapsed or refractory Primary Central Nervous System Lymphoma (PCNSL).

The first stage of the study will enroll a minimum of 12 patients with relapsed or refractory Primary Central Nervous System Lymphoma (PCNSL) evaluable for the primary study objective. If during the first stage of the study data emerge that 80 mg p.o. qd is not adequately tolerated or is inefficacious in patients with relapsed or refractory Primary Central Nervous System Lymphoma (PCNSL), additional patients may be enrolled in the study to evaluate alternative dosing regimens, either a lower daily dose (eg. 60 mg) or a lower weekly dose with administration on 2 consecutive days followed by 5 days without treatment in 7-day treatment cycles (intermittent dosing schedule A).In all cases data from at least 12 evaluable patients will be required on the selected dosing regimen (daily or weekly) before the decision is made to proceed with this regimen into the second stage of the study.Nine (9) additional patients will be enrolled for the second stage of the study, for a minimum of 21 patients on the selected dosing regimen in total, evaluable for the final primary endpoint analysis.All patients evaluable for the primary endpoint will be followed until disease progression or death.

Secondary objectives, PQR309 treatment safety and pharmacokinetics (PK) will be evaluated in all enrolled patients in both study stages.


Sponsor: PIQUR Therapeutics AG

Current Primary Outcome: Overall Response Rate [ Time Frame: Every 8 weeks up to 6 months ]

ORR (Overall Response Rate) 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) [1].


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of Adverse Events as related to the study medication [ Time Frame: Week 1 Day 1 to 30 days after last dose up to 12 months ]
    Continous Dosing and Intermittent Dosing
  • Changes in puls rate [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes in blood pressure [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes in body weight [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes in temperature [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Physical examination according to Karnofsky Performance Status (KPS) [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Depression Test PHQ-9 [ Time Frame: Treatment Day 22, Day 43 and every subsequent 3 weeks later up to 1 year, at the end of treatment ]
    Continous Dosing and Intermittent Dosing
  • Generalized anxiety disorder mood scale score (GAD7) [ Time Frame: Treatment Day 22, Day 43 and every subsequent 3 weeks later up to 1 year, at the end of treatment ]
    Continous Dosing and Intermittent Dosing
  • Changes in haematology [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15,Day 22, Day 36 and Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes in routine blood chemistry [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15, Day 22, Day 36 and Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes of Insulin/Glucose/ C-peptide [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15 and 22, Day 36, Day 43 and every subsequent 3 weeks up to 1 year, at the end of treatment and 30 days after last dose ]
    Continous Dosing and Intermittent Dosing
  • Changes of haemostasis [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 22, Day 43 and every subsequent 3 weeks ]
    Continous Dosing and Intermittent Dosing
  • Changes of urinanalysis [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 22, Day 43 and every subsequent 3 weeks up to 1 year ]
    Continous Dosing and Intermittent Dosing
  • Changes of ECG [ Time Frame: Week 1 Day 1 prior to treatment, Treatment on Day 22, Day 43 and every subsequent 3 weeks up to 1 year ]
    Continous Dosing and Intermittent Dosing


Original Secondary Outcome: Same as current

Information By: PIQUR Therapeutics AG

Dates:
Date Received: April 10, 2017
Date Started: June 15, 2017
Date Completion: April 2018
Last Updated: May 22, 2017
Last Verified: May 2017