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NeoplasmsPhase 1NCT02859857

Phase 1 Study of BXQ-350 in Adult Patients With Advanced Solid Tumors

A Phase 1 study testing BXQ-350 in people with Neoplasms, run by Bexion Pharmaceuticals, Inc. at 4 sites in United States.

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

The objective of this study is to characterize the safety profile and determine the maximum tolerate dose (MTD) of BXQ-350, when given as a single agent at escalating doses, according to the investigational product (IP) related dose-limiting toxicities (DLTs) in patients with advanced solid tumors. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Neoplasms
What is being tested?
BXQ-350
Could I receive a placebo?
No, everyone receives an active treatment
How many people
86 participants
Where
4 sitesUnited States
Study dates
1 Sep 2016to 15 Jun 2020
Phase
Phase 1
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 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Each patient must meet the following criteria:
  • Provide signed, written informed consent prior to the initiation of any study-specific procedures
  • Have histologically or cytologically confirmed diagnosis of advanced solid tumor cancer (excluding lymphomas) for which there is no further standard therapy or when standard therapy is contraindicated. Patients with HGG must have shown unequivocal evidence for recurrence or progression by MRI scan or must have histologically proven tumor recurrence.
  • Patients with HGG: Have previously received radiotherapy and temozolomide
  • For patients with HGG and receiving glucocorticoid therapy, must be on stable or decreasing equivalent daily dose of glucocorticoids for 2 weeks (14 days) prior to dose assignment
  • Have measurable or non-measurable disease per RECIST 1.1 criteria for solid tumors and RANO criteria for HGG
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  • Are males or females aged ≥ 18 years
  • Have Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0 - 2
  • Have acceptable liver function defined as:
  • Total serum bilirubin ≤ 1.5 × upper limit of normal for the study site (ULN) (in patients with known Gilbert Syndrome, total bilirubin ≤ 3 × ULN, with direct bilirubin ≤ 1.5 × ULN)
  • Aspartate Transaminase (AST), Serum Glutamic Oxaloacetic Transaminase (SGOT), Alanine Transaminase (ALT), Serum Glutamic-Pyruvic Transamine (SGPT) ≤ 3 × ULN (if liver metastases are present, then ≤ 5 × ULN is allowed)
  • Serum albumin ≥ 3 g/dL
  • Have acceptable renal function defined as:
  • Serum creatinine ≤ 1.5 × ULN, OR calculated creatinine clearance ≥ 45 mL/min for patients with creatinine levels above 1.5 mg/dL
  • Have acceptable bone marrow function defined as:
  • Absolute neutrophil count (ANC) ≥ 1,500 cells/mm3
  • Platelet count ≥ 100,000 cells/mm3
  • Hemoglobin > 9.0 g/dL
  • Have acceptable coagulation parameters defined as:
  • International normalized ratio (INR) ≤ 2 × ULN
  • Activated partial thromboplastin time (aPTT) within normal limits
  • Have a negative serum pregnancy test result at screening (for females of child bearing potential (FCBP); not applicable to patients who are unable to become pregnant, including those with tubal ligation, bilateral oophorectomy and/or hysterectomy, post-menopausal is defined as > 12 months since last menstrual cycle)
  • FCBP and male patients whose sexual partner(s) are FCBP must agree to abstain from heterosexual activity or use a double barrier method of contraception (e.g., condom and occlusive cap with spermicide) or highly effective contraception (intrauterine device or system, established hormonal contraceptive methods on a stable dose from the time of the last menstrual cycle, or vasectomized partner with confirmed azoospermia) from the time of study entry to 1 month after the last day of treatment

Who cannot join

  • Patients must not meet any of the following criteria:
  • Have a concurrent malignancy or have had another malignancy within 1 year prior to initiation of screening (with the exception of adequately treated basal or squamous cell carcinoma, melanoma in situ, early-stage prostate cancer (T1a-cN0M0), ductal carcinoma in situ of the breast or cervical carcinoma in situ)
  • Patients with solid tumors: Have received anticancer therapies, including radiation therapy, cytotoxic agents, targeted agents or endocrine therapy within 2 weeks prior to dose assignment
  • Patients with HGG: Have received anticancer therapies including: radiation therapy to current site of disease within 12 weeks of dose assignment, targeted agent therapy within 2 weeks of dose assignment, nitrosoureas within 6 weeks of dose assignment, procarbazine within 3 weeks of dose assignment, or other cytotoxic agents within 4 weeks of dose assignment
  • Have not recovered from toxicity of prior therapy defined as a return to < grade 1 at the time of dose assignment, graded according to CTCAE v4.03 (excluding alopecia, neuropathy, and lymphopenia)
  • Have received prior treatment with any investigational drug within 4 weeks prior to dose assignment
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  • Have had major surgery other than a minor outpatient procedure within 4 weeks prior to dose assignment or have not recovered from major side effects of the surgery if more than 4 weeks have elapsed since surgery
  • Have a history of cardiac dysfunction including:
  • Myocardial infarction within 6 months prior to initiation of screening
  • History of documented congestive heart failure (New York Heart Association functional classification III-IV) within 6 months prior to initiation of screening
  • Active cardiomyopathy
  • ECG with correctd QT interval (QTc) >450 msec in males or >470 msec in females at screening
  • Have a known history of HIV seropositivity
  • Are pregnant or nursing (lactating), where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive serum human chorionic gonadotropin (hCG) laboratory test
  • Have symptomatic brain metastases or leptomeningeal disease
  • Have active (acute or chronic) or uncontrolled severe infections
  • Have active poor wound healing (delayed healing, wound infection or fistula)
  • Have poorly controlled hypertension defined as blood pressure >160/90 on at least 2 repeated determinations on separate days within 2 weeks (14 days) prior to initiation of screening
  • Have evidence of active clinically significant bleed (e.g., gastrointestinal bleed, hemoptysis, or gross hematuria) at screening
  • Have other concurrent severe and/or uncontrolled medical condition that would, in the site Investigator's judgment contraindicate the patient's participation in the clinical study

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • University of New Mexico Cancer Center

    Albuquerque, New Mexico, 87102

  • University of Cincinnati Barrett Center

    Cincinnati, Ohio, 45219

  • The Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

  • University of Kentucky Markey Cancer Center

    Lexington, Kentucky, 40536

Other trials in United States

About this study

Official title: Phase 1, Dose-Escalation, Open-label, Safety and Pharmacokinetic, First in Human Study of BXQ-350 Administered as a Single Agent by Intravenous Infusion in Adult Patients With Advanced Solid Tumors and Recurrent High-Grade Gliomas

The objective of this study is to characterize the safety profile and determine the maximum tolerate dose (MTD) of BXQ-350, when given as a single agent at escalating doses, according to the investigational product (IP) related dose-limiting toxicities (DLTs) in patients with advanced solid tumors. Secondarily to assess the preliminary antitumor activity of BXQ-350 in solid tumors and recurrent high grade gliomas.

Read the detailed description

This is a first in man study of BXQ-350, a novel anti-neoplastic therapeutic agent composed of two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes. When both the components are assembled together forming stable SapC-DOPS nanovesicles (clinical formulation BXQ-350), the agent exhibits the propensity to enter the body and brain, target cells in the tumor mass, and induce cell death.

The study is divided into 3 parts:

  1. Dose Escalation Scheme Sequential cohorts of adult patients with advanced solid tumors and recurrent high-grade gliomas will be treated with escalating doses of BXQ-350 until the MTD is established, or in the absence of a MAD, the highest planned DL.
  2. During Part 2, patients with advanced solid tumors and recurrent high-grade gliomas will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at the highest planned DL, if the MAD is not reached.
  3. During Part 3, patients with either ependymoma, GI tumors , or advanced solid tumors other than HGG, will be enrolled and administered BXQ-350 at the 2.4 mg/kg dose level.
Conditions
Neoplasms
Being tested
BXQ-350
Keywords
Glioblastoma Multiforme, Solid Tumors, Ependymoma
Medical subject headings
Neoplasms, Glioblastoma, Ependymoma

What participants receive

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

  • BXQ-350Drug

    Also called SapC-DOPS

    BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes. When both the components are assembled together stable SapC-DOPS nanovesicles are formed(clinical formulation BXQ-350).

Study groups in NCT02859857
Study groupRoleWhat they receive
Rising dose; safety and toleranceExperimentalSequential cohorts of patients with advanced solid tumors and recurrent high-grade gliomas will be be treated with escalating doses of BXQ-350 until the MTD is established, or in the absence of a MAD, the highest planned DL is reached.
Receives: Drug: BXQ-350
Solid tumor patientsExperimentalCohort of patients with advanced solid tumors administered BXQ-350 at the MTD determined in Part 1 or at the highest planned DL if the MAD is not reached.
Receives: Drug: BXQ-350
Glioblastoma Multiforme patientsExperimentalCohort of patients with recurrent high-grade gliomas administered BXQ-350 at the MTD determined in Part 1 or at the highest planned DL if the MAD is not reached.
Receives: Drug: BXQ-350
Gastrointestinal tumor patientsExperimentalCohort of patients with Gastrointestinal tumors as defined in the protocol and administered BXQ-350 at the 2.4 mg/kg dose level.
Receives: Drug: BXQ-350
Ependymoma tumor patientsExperimentalCohort of patients with ependymoma administered BXQ-350 at the 2.4 mg/kg dose level.
Receives: Drug: BXQ-350
Solid tumor patients other than HGGExperimentalCohort of patients with advanced solid tumors other than HGG administered BXQ-350 at the 2.4 mg/kg dose level.
Receives: Drug: BXQ-350

What is being measured

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

  1. Part 1-MTD

    • To determine the maximum tolerate dose (MTD) of BXQ-350, when given as a single agent at escalating doses, according to the investigational product (IP) related dose-limiting toxicities (DLTs) in patients with advanced solid tumors

    Measured: 12 months

  2. Part 2-RECIST

    ·To assess preliminary antitumor activity, defined as maximal radiological response during treatment using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1 (v1.1) criteria for solid tumors.

    Measured: 12 months

  3. Part 2-RANO

    To assess preliminary antitumor activity, defined as maximal radiological response during treatment Revised Assessment in Neuro-Oncology (RANO) criteria for recurrent high grade glioma (HGG), of BXQ-350 given as a single agent at the MTD, or highest planned dose level (DL), in the absence of a Maximum Administered Dose (MAD).

    Measured: 12 months

  4. Part 3 - RECIST

    To assess preliminary antitumor activity, defined as maximal radiological response during treatment using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1 (v1.1) criteria for solid tumors

    Measured: 12 months

Secondary outcomes (7)

  1. Part 2- Area under Curve (AUC)

    ·To evaluate the AUC of BXQ-350

    Measured: 12 months

  2. Part 2-Cmax

    To evaluate the Cmax of BXQ-350

    Measured: 12 months

  3. Part 2-half life

    To evaluate the half-life (t1/2) of BXQ-350

    Measured: 12 months

  4. Part 2-CL

    To evaluate the clearance (CL) of BXQ-350

    Measured: 12 months

  5. Part 2-Progression-free survival (PFS-6)

    To evaluate progression free survival at 6 months

    Measured: 12 months

  6. Part 2-time to response

    To evaluate time to response

    Measured: 12 months

  7. Part 2-duration of response

    To evaluate duration of response

    Measured: 12 months

Timeline

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

  1. First posted9 Aug 2016 (estimated)
  2. Study start1 Sep 2016
  3. Primary completion17 Jun 2019
  4. Study completion15 Jun 2020

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
86 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02859857
Sponsor's protocol ID
BXQ-350.AA
Status last verified
Jul 2021
First submitted
29 Jul 2016
Last updated
22 Jul 2021
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BXQ-350 in people with Neoplasms, run by Bexion Pharmaceuticals, Inc. at 4 sites in United States.

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 23 inclusion and 20 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 22 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Part 1-MTD (time frame: 12 months). The study lists 4 primary outcomes in all. 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 NCT02859857.

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 22 Jul 2021.

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