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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.
ClinicalTrials.gov Identifier: NCT03740100
Recruitment Status : Terminated (The trial was closed because the sponsor became insolvent.)
First Posted : November 14, 2018
Results First Posted : January 20, 2022
Last Update Posted : January 20, 2022
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

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).

Condition or disease Intervention/treatment Phase
HNSCC Drug: Bimiralisib Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Oral administration
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : January 25, 2019
Actual Primary Completion Date : August 5, 2020
Actual Study Completion Date : August 5, 2020

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Open single arm
Bimiralisib capsules orally
Drug: Bimiralisib
Bimiralisib capsules
Other Name: PQR309, PI3K/mTOR inhibitor

Primary Outcome Measures :
  1. Objective Response Rate (ORR) [ Time Frame: At 6 and 12 weeks after the start of therapy (± 3 days) ]

    Radiological tumor assessments were performed by computed tomography (CT) or magnetic resonance imaging (MRI) according to a standard protocol.

    ORR: comprised of all patients who achieved a confirmed partial or a confirmed complete response per RECIST 1.1

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03740100

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United States, Texas
M.D. Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
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Principal Investigator: Faye M Johnson, MD,PhD MD Anderson Cancer Center Recruiting, Houston, Texas, US 77030
  Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:
Study Protocol  [PDF] April 24, 2020
Statistical Analysis Plan  [PDF] September 24, 2018
Informed Consent Form  [PDF] May 11, 2020

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT03740100    
Other Study ID Numbers: 2018-1003 (PQR309-009)
First Posted: November 14, 2018    Key Record Dates
Results First Posted: January 20, 2022
Last Update Posted: January 20, 2022
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by M.D. Anderson Cancer Center:
Head and Neck Squamous Cell Carcinoma
Notch 1 loss of function mutation
PI3K/mTOR Inhibitor
Refractory metastatic
Additional relevant MeSH terms:
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Squamous Cell Carcinoma of Head and Neck
Carcinoma, Squamous Cell
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Head and Neck Neoplasms
Neoplasms by Site
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs