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Single-arm Study With Bimiralisib in Patients With HNSCC Harboring NOTCH1 Loss of Function Mutations (HNSCC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03740100
Recruitment Status : Completed
First Posted : November 14, 2018
Last Update Posted : February 25, 2021
Information provided by (Responsible Party):
PIQUR Therapeutics AG

Tracking Information
First Submitted Date  ICMJE November 1, 2018
First Posted Date  ICMJE November 14, 2018
Last Update Posted Date February 25, 2021
Actual Study Start Date  ICMJE January 25, 2019
Actual Primary Completion Date August 5, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 12, 2018)
Assessment of change of tumor size from baseline [ Time Frame: Every 6 weeks until all patients have been treated for 6 months ]
Radiological tumor assessments will be performed by computed tomography (CT) or magnetic resonance imaging (MRI) according to a standard protocol
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Single-arm Study With Bimiralisib in Patients With HNSCC Harboring NOTCH1 Loss of Function Mutations
Official Title  ICMJE Open-label, Single Arm, Two-stage Study, Evaluating the Efficacy and Safety of Bimiralisib in Patients With Recurrent or Metastatic Head and Neck Squamous Cell Carcinomas (HNSCC) Harboring NOTCH1 Loss of Function (LOF) Mutations
Brief Summary

Preclinical data and limited clinical evidence suggest that Head and Neck Squameous Cell Carcinoma tumors harboring certain mutations may respond well to PI3K/mTOR inhibition (phosphatidylinositol-3-kinase/ mammalian target of rapamycin inhibition).

The current study enrolls patients with refractory and / or metastatic Head and Neck Squameous Cell Carcinoma based on the mutational status of their disease to assess the response to treatment with bimiralisib, an orally available pan-PI3K/mTOR inhibitor (phosphatidylinositol-3-kinase/ mammalian target of rapamycin inhibitor).

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Oral administration
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE HNSCC
Intervention  ICMJE Drug: Bimiralisib
Bimiralisib capsules
Other Name: PQR309, PI3K/mTOR inhibitor
Study Arms  ICMJE Open single arm
Bimiralisib capsules orally
Intervention: Drug: Bimiralisib
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 23, 2021)
Original Estimated Enrollment  ICMJE
 (submitted: November 12, 2018)
Actual Study Completion Date  ICMJE August 5, 2020
Actual Primary Completion Date August 5, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Histologically or cytological confirmed diagnosis of Head and Neck Squameous Cell Carcinoma, for which no standard curative or life prolonging therapy is available
  2. Available CLIA-certified sequencing results of the NOTCH gene in Head and Neck Squameous Cell Carcinoma (HNSCC) tumor material. The tumor must harbor a NOTCH1 LOF mutation as confirmed by central review (MD Anderson Cancer Center, MDACC)
  3. ECOG performance status of ≤ 2
  4. Adequate bone marrow, liver, and renal functions
  5. Measurable disease according to RECIST version 1.1
  6. Patients of reproductive potential must agree to use effective contraception from screening until 90 days after discontinuing study treatment.

Exclusion Criteria:

  1. Has received any anti-cancer treatment including hormonal and investigational agents within 21 days prior to first dose of bimiralisib.
  2. Major surgery within 28 days prior to first dose of bimiralisib or persisting side effects that have not improved to NCI-CTCAE grade 1 or better.
  3. Pregnant or nursing (lactating) women.
  4. Poorly controlled diabetes mellitus, steroid-induced diabetes mellitus
  5. Has other active malignancies that require systemic treatment.
  6. Has a known history of HIV infection
  7. Any of the following cardiac abnormalities:

    • History of, or current, documented congestive heart failure (New York heart association functional classification iii - iv), documented cardiomyopathy
    • Symptomatic (NYHA class II or higher) left ventricular ejection fraction (LVEF) < 40% as determined by multiple gated acquisition (MUGA) scan or echocardiogram (echo)
    • Myocardial infarction ≤ 6 months prior to enrolment
    • Unstable angina pectoris
    • Serious uncontrolled cardiac arrhythmia
    • Symptomatic pericarditis
  8. Impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug.
  9. Patient has a history of non-compliance to medical regimen or inability to grant consent.
  10. Medically documented history of an active major depressive episode, bipolar disorder (I or II), obsessive-compulsive disorder, schizophrenia, a history of suicidal attempt or ideation, or homicidal ideation (immediate risk of doing harm to others) or ≥ CTCAE grade 3 anxiety
  11. History of interstitial pneumonitis or patients who require chronic oxygen supplementation.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT03740100
Other Study ID Numbers  ICMJE PQR309-009
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party PIQUR Therapeutics AG
Study Sponsor  ICMJE PIQUR Therapeutics AG
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Faye M Johnson, MD,PhD MD Anderson Cancer Center Recruiting, Houston, Texas, US 77030
PRS Account PIQUR Therapeutics AG
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP