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Primary Central Nervous System LymphomaPhase 2NCT02669511

PQR309 in Patients With Relapsed or Refractory Primary Central Nervous System Lymphoma

A Phase 2 study testing PQR309 in people with Primary Central Nervous System Lymphoma, run by PIQUR Therapeutics AG.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Primary Central Nervous System Lymphoma
What is being tested?
PQR309
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
No sites listed yet
Study dates
12 Nov 2015to 12 Jan 2018
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥18 years of age.
  • Patient with histologically/cytologically confirmed Primary Central Nervous System Lymphoma (PCNSL)
  • Relapsed or refractory Primary Central Nervous System Lymphoma (PCNSL) demonstrated by cranial MRI.
  • 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.
  • Maximum one prior systemic therapy regimen.
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  • 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.
  • Karnofsky Performance Score (KPS) ≥ 70%.
  • More than 4 weeks from any investigational agent.
  • Adequate haematological, liver and renal function
  • Able and willing to swallow and retain oral medication.
  • Female and male patients of reproductive potential must agree to use effective contraception from screening until 90 days after discontinuing study treatment.
  • Willing and able to sign the informed consent and to comply with the protocol for the duration of the study.

Who cannot join

  • Central Nervous System (CNS) Lymphoma or chronic immunosuppression-associated central nervous system (CNS) lymphoma.
  • Previous allogeneic hematopoietic stem cell transplant (HSCT transplant).
  • Previous whole brain radiotherapy (WBRT)
  • Other concomitant anti-tumor therapy as determined by the study team.
  • Patients unable to undergo contrast-enhanced MRI.
  • Prior treatment with a phosphoinositide -3 kinase (PI3K) inhibitor, Protein Kinase B Inhibitor is known as AKT inhibitor, or mammalian target of rapamycin (mTOR) inhibitor.
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  • Patient taking enzyme-inducing anti-epileptic drug (EIAED) < 7 days of the first dose of PQR309.
  • Patient is taking a drug with a risk to promote QT prolongation and Torsades de Pointes.
  • 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.
  • 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.
  • Anxiety ≥ Common Terminology Criteria (CTC) of adverse events (AE) grade 3.
  • Patient has an uncontrolled intercurrent illness, including, but not limited to, ongoing or active infection, 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.
  • Presence of gastrointestinal disease or any other condition that could interfere significantly with the absorption of the study drug.
  • Concomitant treatment with medicinal products that increase the potential hydrogen (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 washout period sufficient to terminate their effect.
  • Patient has a history of invasive malignancy other than Primary Central Nervous System Lymphoma (PCNSL). Patients are eligible, if they are disease-free for at least 3 years and deemed to be at low risk for recurrence by the investigator. Patients diagnosed with cervical cancer in situ, basal cell or squamous cell carcinoma of the skin and treated within the past 3 years are eligible.
  • Women who are pregnant or breast feeding.
  • 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 serum pregnancy test within 7 days prior to first dose of PQR309).
  • Fasting glucose > 7.0 mmol/L (126 mg/dL). or HbA1c > 6.4%.

About this study

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

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).

Read the detailed description

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.

Being tested
PQR309
Keywords
DLBCL

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PQR309Drug

    Also called bimiralisib

    Oral PQR309, 80mg or 60mg daily or intermittent dosing

Study groups in NCT02669511
Study groupRoleWhat they receive
PQR309OtherA single arm study with PQR309, a phosphoinositide-3-kinases (PI3K) and inhibitor of the mammalian target of rapamycin (mTOR), 60mg/80mg given once a day, orally.
Receives: Drug: PQR309

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (ORR)

    ORR including complete response (CR, unconfirmed complete(CRu) and partial response (PR) according to the 2005 Response Criteria of the Central Nervous System (CNS) Lymphoma Collaborative Group (IPCG)

    Measured: Every 8 weeks up to 6 months

Secondary outcomes (14)

  1. Number of adverse events (AE) as related to the study medication.

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 to 30 days after last dose up to 12 months

  2. Changes in puls rate

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8,Day 15 and 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  3. Changes in blood pressure

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8,Day 15 and 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  4. Changes in body weight

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8,Day 15 and 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  5. Changes in temperature

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8,Day 15 and 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  6. Changes in Physical examination according to Karnofsky Performance Status (KPS)

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8,Day 15 and 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  7. Generalized anxiety disorder mood scale score (GAD7)

    Continous Dosing and Intermittent Dosing

    Measured: Treatment on Day 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  8. Depression Test PHQ-9

    Continous Dosing and Intermittent Dosing

    Measured: Treatment on 22, Day 43 and every subsequent 3 weeks, at the end of treatment and 30days after last dose

  9. Changes in haematology

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15,Day 22, Day 36 and Day 43 and every subsequent 3 weeks, at the end of treatment and 30 days after last dose

  10. Changes in Routine blood chemistry

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15,Day 22, Day 36 and Day 43 and every subsequent 3 weeks, at the end of treatment and 30 days after last dose

  11. Changes of Insulin/Glucose/C-Peptide

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 8, Day15,Day 22, Day 36 and Day 43 and every subsequent 3 weeks, at the end of treatment and 30 days after last dose

  12. Changes of haemostasis

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 22 and Day 43 and every subsequent 3 weeks, at the end of treatment

  13. Changes of urinanalysis

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 22 and Day 43 and every subsequent 3 weeks, at the end of treatment

  14. Changes of ECG

    Continous Dosing and Intermittent Dosing

    Measured: Week 1 Day 1 prior to treatment, Treatment on Day 22 and Day 43 and every subsequent 3 weeks, at the end of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Feb 2016 (estimated)
  2. Study start12 Nov 2015
  3. Primary completion12 Jan 2018
  4. Study completion12 Jan 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02669511
Sponsor's protocol ID
PQR309-005
Status last verified
Jul 2019
First submitted
7 Jan 2016
Last updated
9 Jul 2019
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PQR309 in people with Primary Central Nervous System Lymphoma, run by PIQUR Therapeutics AG.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) (time frame: Every 8 weeks up to 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02669511.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2019.

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