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NeoplasmsPhase 1KOURAGENCT03967093

A Study of BXQ-350 in Children and Young Adults With Relapsed Solid Tumors

A Phase 1 study testing BXQ-350 in people with Neoplasms, run by Bexion Pharmaceuticals, Inc. at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children and young adults with relapsed solid tumors, including recurrent malignant brain tumors. All patients will receive BXQ-350 by intravenous (IV) infusion. Read more about this study

Who is it for?
Ages 1 year to 30 years
Conditions the study is for
Neoplasms
What is being tested?
BXQ-350
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
2 sitesUnited States
Study dates
15 Apr 2019to 17 Jan 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 1 year to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Each subject must meet the following criteria:
  • Provide signed, written informed consent prior to the initiation of any study-specific procedures (Consent from Guardians for minor children and patient assent according to Institution and Institutional Review Board (IRB) standards)
  • Are male or female aged ≥ 1 to 30 years
  • Have histologically or cytologically confirmed relapsed solid tumor cancer, including recurrent malignant brain tumors, for which there is no further standard therapy or when standard therapy is contraindicated • Recurrent malignant brain tumors: must have shown unequivocal evidence for recurrence or progression by MRI scan or must have histologically proven tumor recurrence • Recurrent malignant brain tumors: must have previously received standard of care treatment at initial diagnosis (radiation and/or chemotherapy)
  • Recurrent malignant brain tumors receiving glucocorticoid therapy: must be on stable or decreasing equivalent daily dose of glucocorticoids for 2 weeks (14 days) prior to dose assignment
  • Recurrent embryonal tumors or atypical teratoid rhabdoid tumors (AT/RT): must have previously received standard of care therapy including either chemotherapy and radiation therapy or high dose chemotherapy with autologous hematopoietic stem cell support
Show all 32Show fewer
  • Grade II or III recurrent ependymoma, including RELA fusion-positive ependymoma: must have previously received radiation therapy
  • Have measurable or non-measurable disease per RECIST v1.1 for relapsed solid tumors, RRC for recurrent malignant brain tumors, and INRC for recurrent neuroblastomas
  • Have Lansky (age 1 - 15) / Karnofsky (age ≥ 16) Performance Score of >50 or Eastern Cooperative Oncology Group Performance Status (ECOG PS) (age ≥ 18) of 0 - 2
  • Have acceptable liver function defined as:
  • Total serum bilirubin ≤ 1.5 × upper limit of normal (ULN) for the study site (in subjects 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:
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥70 mL/min/1.73m² or a maximum serum creatinine (mg/dL)* based on age/gender as follows:
  • 1 to < 2 years: 0.6 (male); 0.6 (female)
  • 2 to < 6 years: 0.8 (male); 0.8 (female)
  • 6 to < 10 years: 1 (male); 1 (female)
  • 10 to < 13 years: 1.2 (male); 1.2 (female)
  • 13 to < 16 years: 1.5 (male); 1.4 (female)
  • ≥ 16 years: 1.7 (male); 1.4 (female)
  • Threshold creatinine values derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the CDC (Schwartz 2009)
  • Have acceptable bone marrow function defined as:
  • Absolute neutrophil count (ANC) ≥ 750 cells/mm3
  • Platelet count ≥ 75,000 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
  • Chronic/prophylactic anticoagulation for a distant thrombus that occurred ≥3 months ago is allowed
  • Have a negative serum pregnancy test result at screening (for females of child bearing potential (FCBP); not applicable to subjects who are unable to become pregnant, including those with tubal ligation, bilateral oophorectomy and/or hysterectomy)
  • FCBP and male subjects 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

  • Subjects must not meet any of the following criteria:
  • Have a concurrent or second malignancy
  • Have lymphoma
  • Have Grade I ependymoma
  • Relapsed solid tumors: have symptomatic brain metastases or leptomeningeal disease
  • Relapsed solid tumors: have received
Show all 32Show fewer
  • anticancer therapies within 2 weeks prior to dose assignment (including radiation therapy, cytotoxic agents, targeted agents or endocrine therapy)
  • myelosuppressive agents within 3 weeks prior to dose assignment
  • monoclonal antibodies within 4 weeks prior to dose assignment
  • growth factors within 2 weeks of dose assignment
  • other immunotherapy (tumor vaccine, cytokines) within 4 weeks of dose assignment
  • Recurrent malignant brain tumors: have received
  • anticancer therapies including: radiation therapy to current site of disease within 3 weeks 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; other cytotoxic agents withing 4 weeks of dose assignment
  • myelosuppressive agents within 4 weeks prior to dose assignment
  • monoclonal antibodies within 4 weeks prior to dose assignment
  • other immunotherapy (tumor vaccine, cytokines, or growth factor) within 2 weeks prior to 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 v5.0 (excluding alopecia, neuropathy, and lymphopenia)
  • Have had major surgery other than a minor outpatient procedure within 28 days 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 poorly controlled hypertension despite the use of antihypertensive agents defined as blood pressure ≥95th percentile for age and weight or >160/90 on at least 2 repeated determinations on separate days within 2 weeks (14 days) prior to initiation of screening
  • Have a history of cardiac dysfunction including any of the following:
  • Myocardial infarction within 6 months prior to initiation of screening
  • History of documented congestive heart failure (New York Heart Association functional classification III-IV, see Appendix 6) within 6 months prior to initiation of screening
  • Active cardiomyopathy
  • Electrocardiogram (ECG) with QTc >480 msec at screening
  • Echocardiogram with ejection fraction <50% or a decrease in the left ventricular shortening fraction to <27%
  • Have a known history of Human Immunodeficiency Virus (HIV) seropositivity
  • Have active (acute or chronic) or uncontrolled severe infections
  • Have active poor wound healing (delayed healing, wound infection or fistula)
  • Have evidence of active clinically significant bleed (e.g., gastrointestinal bleed, hemoptysis, or gross hematuria) at initiation of screening
  • 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 received prior treatment with any investigational drug within 28 days prior to dose assignment
  • Have other concurrent severe and/or uncontrolled medical condition that would, in the site Investigator's judgment contraindicate the subject's participation in the clinical study

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45226

  • Nationwide Children's

    Columbus, Ohio, 43205

Other trials in United States

About this study

Official title: A Phase 1 Safety Study of BXQ-350 Administered as a Single Agent by Intravenous Infusion in Children and Young Adults With Relapsed Solid Tumors, Including Recurrent Malignant Brain Tumors

This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children and young adults with relapsed solid tumors, including recurrent malignant brain tumors. All patients will receive BXQ-350 by intravenous (IV) infusion. The study is divided into two parts: Part 1 will enroll patients at increasing dose levels of BXQ-350 in order to determine the MTD. Part 2 will use the MTD to further assess the safety of BXQ-350 as well as preliminary anti-tumor activity.

Read the detailed description

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 (clinical formulation BXQ-350). Given via intravenous (IV) infusion, data indicate that 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 2 parts:

Part 1: Dose Escalation and Safety - Sequential cohorts of patients 1-30 years of age with relapsed solid tumors, including recurrent malignant brain tumors will be treated with escalating doses of BXQ-350 until the MTD is established, or in the absence of a Maximum Administered Dose, the highest planned dose level is reached.

Part 2: Safety and Preliminary Anti-tumor Activity - Patients will be enrolled into one of four cohorts and administered BXQ-350 at the established MTD or at the highest planned dose level. The four cohorts will consist of: recurrent ependymoma, recurrent malignant brain tumor, recurrent Diffuse Intrinsic Pontine Glioma (DIPG), and relapsed non-central nervous system (non-CNS) solid tumor.

Conditions
Neoplasms
Being tested
BXQ-350
Keywords
solid tumor, brain tumor, DIPG, ependymoma
Medical subject headings
Neoplasms, Brain Neoplasms, 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 (clinical formulation BXQ-350). BXQ-350 is administered by intravenous (IV) infusion over a minimum of six 28-day cycles.

Study groups in NCT03967093
Study groupRoleWhat they receive
Part 1 Dose Escalation: Safety and ToleranceExperimentalSequential cohorts of patients with relapsed solid tumors, including malignant brain tumors, will be treated with escalating doses of BXQ-350 until the maximum tolerated dose (MTD) is established, or in the absence of a maximum administered dose (MAD), the highest planned dose level (3.2 mg/kg) is reached.
Receives: Drug: BXQ-350
Part 2: Ependymoma PatientsExperimentalCohort of patients with recurrent ependymoma will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at 3.2 mg/kg if the MAD is not reached.
Receives: Drug: BXQ-350
Part 2: Brain Tumor PatientsExperimentalCohort of patients with recurrent malignant brain tumors will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at 3.2 mg/kg if the MAD is not reached.
Receives: Drug: BXQ-350
Part 2: DIPG PatientsExperimentalCohort of patients with recurrent diffuse intrinsic pontine glioma (DIPG) will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at 3.2 mg/kg if the MAD is not reached.
Receives: Drug: BXQ-350
Part 2: Other Solid Tumor PatientsExperimentalCohort of patients with relapsed non-central nervous system (CNS) solid tumors will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at 3.2 mg/kg if the MAD is not reached.
Receives: Drug: BXQ-350

What is being measured

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

  1. Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v5.0

    To determine the safety of BXQ-350 in children and young adults with relapsed solid tumors, including recurrent malignant brain tumors, as evidenced by the incidence of treatment emergent adverse events assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

    Measured: 6 months

  2. Part 1 - Maximum Tolerated Dose

    To determine the maximum tolerated 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 children and young adults with relapsed solid tumors, including recurrent malignant brain tumors

    Measured: 6 months

  3. Part 2 - RECIST

    To assess the preliminary antitumor activity of BXQ-350, given as a single agent at the MTD, or highest planned dose level (DL), 3.2 mg/kg, in the absence of a Maximum Administered Dose (MAD). Antitumor activity is defined as maximal radiological response during treatment using:

    • Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1) criteria for relapsed solid tumors

    Measured: 6 months

  4. Part 2 - RANO

    To assess the preliminary antitumor activity of BXQ-350, given as a single agent at the MTD, or highest planned dose level (DL), 3.2 mg/kg, in the absence of a Maximum Administered Dose (MAD). Antitumor activity is defined as maximal radiological response during treatment using:

    • Radiographic Response Criteria (RRC) for recurrent malignant brain tumors

    Measured: 6 months

  5. Part 2 - INRC

    To assess the preliminary antitumor activity of BXQ-350, given as a single agent at the MTD, or highest planned dose level (DL), 3.2 mg/kg, in the absence of a Maximum Administered Dose (MAD). Antitumor activity is defined as maximal radiological response during treatment using:

    • International Neuroblastoma Response Criteria (INRC) for recurrent neuroblastomas

    Measured: 6 months

Secondary outcomes (3)

  1. Progression-Free Survival (PFS-6)

    • To evaluate progression-free survival at 6 months (PFS-6) in subjects with relapsed solid tumors, including recurrent malignant brain tumors

    Measured: 6 months

  2. Time to Response

    • To evaluate time to response, and duration of response in subjects with relapsed solid tumors, including recurrent malignant brain tumors

    Measured: 6 months

  3. Duration of Response

    • To evaluate duration of response in subjects with relapsed solid tumors, including recurrent malignant brain tumors

    Measured: 6 months

Timeline

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

  1. First posted30 May 2019
  2. Study start15 Apr 2019
  3. Primary completion3 Jan 2020
  4. Study completion17 Jan 2020

Why it stopped: Bexion will not move forward with the Part 2 expansion portion of the trial in order to focus resources on the further development of BXQ-350 as an earlier treatment measure in the pediatric population.

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
Sequential
Model description
Part 1 dose escalation - sequential cohorts of patients will be treated with escalating doses of BXQ-350 until the maximum tolerated dose (MTD) is established, or in the absence of a maximum administered dose, the highest planned dose level. Part 2 - patients will be enrolled and administered BXQ-350 at the Part 1 MTD or at the highest planned dose level
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03967093
Sponsor's protocol ID
BXQ-350.AB
Acronym
KOURAGE
Status last verified
Jul 2021
First submitted
21 May 2019
Last updated
22 Jul 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
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 2 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 32 inclusion and 32 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: stopped early, 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 2 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 was stopped early (reason given: Bexion will not move forward with the Part 2 expansion portion of the trial in order to focus resources on the further development of BXQ-350 as an earlier treatment measure in the pediatric population.). Results are not available in the source record.

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?

Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v5.0 (time frame: 6 months). The study lists 5 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 NCT03967093.

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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