A Study to Investigate Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) of PQR309

Phase I Study of Oral PQR309 in Patients With Advanced Solid Tumors

This study is a Phase I, open label study to determine the Maximum Tolerated Dose(MTD) and the Recommended Phase II Dose (RP2D) in patients with advanced solid tumors.

Study Overview

• Status: Completed
• Conditions: Advanced Solid Tumors
• Intervention / Treatment: Drug: PQR309

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 1

Contacts and Locations

Study Locations

• Switzerland
  • Basel, Switzerland, 4031
    • University Hospital Basel

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Histologically or cytologically confirmed diagnosis of advanced solid tumor for which no therapy of proven efficacy is available.
• Age ≥ 18
• Evidence of tumor progression with measurable or evaluable disease.
• Use of adequate contraceptive measures for male patients.
• Eastern Cooperative Oncology Group (ECOG) Performance Score of 0-1.
• Signed informed consent.

Exclusion Criteria:

• Concomitant anticancer therapy (e.g., chemotherapy, radiotherapy, hormonal, immunotherapy, biological response modifier, signal transduction inhibitors)
• Patients with a history of myocardial infarction or coronary artery bypass within the last 3 years.
• Patients with severe/unstable angina, coronary/peripheral arterial bypass, symptomatic congestive heart failure NYHA Class 3 or 4, hypertension BP>150/100mmHg.
• Pre-diagnosed diabetes mellitus.
• Fasting glucose > 7.0 mmol/L or HbA1c > 6%.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PQR309; Different dose evaluation	Drug: PQR309; Dosing will be orally, once a day for the duration of the trial.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Maximum Tolerated Dose (MTD) and the Recommended Phase II Dose (RP2D)	In average 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine overall safety	Incidence of SAEs, incidence and severity of all AEs, changes of vital signs, physical examinations, body weight, changes of routine laboratory assessments.	1 year

Collaborators and Investigators

• Sponsor: PIQUR Therapeutics AG
• Investigators: Study Chair: Andreas Wicki, Dr., University Hospital, Basel, Switzerland

Publications and helpful links

General Publications

• Wicki A, Brown N, Xyrafas A, Bize V, Hawle H, Berardi S, Cmiljanovic N, Cmiljanovic V, Stumm M, Dimitrijevic S, Herrmann R, Pretre V, Ritschard R, Tzankov A, Hess V, Childs A, Hierro C, Rodon J, Hess D, Joerger M, von Moos R, Sessa C, Kristeleit R. First-in human, phase 1, dose-escalation pharmacokinetic and pharmacodynamic study of the oral dual PI3K and mTORC1/2 inhibitor PQR309 in patients with advanced solid tumors (SAKK 67/13). Eur J Cancer. 2018 Jun;96:6-16. doi: 10.1016/j.ejca.2018.03.012. Epub 2018 Apr 13.

Study record dates

Study Major Dates

• Study Start: January 1, 2014
• Primary Completion (Actual): March 1, 2015
• Study Completion (Actual): December 1, 2015

Study Registration Dates

• First Submitted: September 6, 2013
• First Submitted That Met QC Criteria: September 6, 2013
• First Posted (Estimate): September 12, 2013

Study Record Updates

• Last Update Posted (Actual): April 12, 2017
• Last Update Submitted That Met QC Criteria: April 10, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Other Study ID Numbers: PQR309-001; 2013-003441-42 (EudraCT Number)