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Study of Safety and Tolerability of VLX600, an Iron Chelator, in Patients With Refractory Advanced Solid Tumors

Phase 1
Terminated
Conditions
Refractory Cancer
Interventions
Registration Number
NCT02222363
Lead Sponsor
Vivolux AB
Brief Summary

The purpose of this study is to evaluate the safety and tolerability of the investigational drug VLX600 in patients with refractory advanced solid tumors.

Detailed Description

This Phase I, open label dose escalation study of VLX600 in patients with refractory advanced solid tumors will determine the safety profile and maximum tolerated dose (MTD) of VLX600 when administered by intravenous infusion on Days 1, 8, and 15 of each 28-day treatment cycle. Dose escalation will proceed according to the standard "3 + 3" design using doubling doses. The doses are: 10, 20, 40, 80, 160, and 210 mg VLX600.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
19
Inclusion Criteria
  1. Men and women greater than or equal to 18 years of age.

  2. Histologic evidence of advanced solid tumors (excluding central nervous system (CNS) primary tumors) non-resectable, refractory to standard therapies, or patient cannot receive or refuses standard therapy.

  3. Solid tumors measurable according to RECIST 1.1 or solid tumors not measurable according to RECIST 1.1, but which express tumor markers (e.g., prostate cancer with prostate specific antigen (PSA) expression or ovarian cancer with cancer antigen-125 (CA-125) expression) are eligible.

  4. Eastern Cooperative Oncology Group (ECOG) Performance Status 0 or 1.

  5. Must have the following laboratory values, obtained less than or equal to 7 days prior to registration:

    • Absolute neutrophil count (ANC) greater than or equal to 1500/µL
    • Platelet (PLT) count greater than or equal to 100,000/µL
    • Total bilirubin less than 1.5 x upper normal limit (UNL)
    • Aspartate aminotransferase (AST) (SGOT) less than or equal to 3 x UNL
    • Creatinine less than 1.5 x UNL
  6. Women of childbearing potential must have a negative serum pregnancy test less than or equal to 7 days prior to registration.

  7. Life expectancy greater than 12 weeks.

  8. Must provide written informed consent.

  9. Must be willing to return to Mayo Clinic enrolling institution for follow-up.

Exclusion Criteria
  1. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, diabetes mellitus, seizure disorder or psychiatric illness/social situations that would limit compliance with study requirements.

  2. Treatment with any of the following prior therapies:

    • Chemotherapy less than or equal to 4 weeks prior to registration
    • Mitomycin C/nitrosoureas less than or equal to 6 weeks prior to registration
    • Immunotherapy less than or equal to 4 weeks prior to registration
    • Biologic therapy less than or equal to 4 weeks prior to registration
    • Radiation therapy less than or equal to 4 weeks prior to registration
    • Non-cytotoxic therapy less than or equal to 5 half-lives prior to registration
  3. Failure to fully recover from acute, reversible effects of prior chemotherapy regardless of interval since last treatment.

  4. Major surgery less than 28 days prior to study entry.

  5. Active other malignancy, except non-melanoma skin cancer or carcinoma-in-situ (e.g., of cervix, breast, prostate). If there is a history of prior malignancy, patient must not be receiving other specific treatment (other than hormonal therapy) for the cancer.

  6. CNS metastases if not previously treated and stable for at least 2 months per imaging and clinical assessment.

  7. Any of the following because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:

    • Pregnant women
    • Nursing women
    • Men or women of childbearing potential unwilling to employ adequate contraception
  8. Other concurrent chemotherapy, immunotherapy, radiotherapy, any ancillary therapy considered investigational (utilized for a non-FDA-approved indication and in the context of a research investigation) or receiving any other investigational agent which would be considered as a treatment for the primary neoplasm.

  9. Cardiac issues consisting of either symptomatic congestive heart failure (NYHA Class II or higher), unstable angina pectoris, myocardial infarction within 6 months, and/or cardiac arrhythmia, including atrial fibrillation (which is symptomatic or requires treatment).

  10. Corrected QT interval (QTc) interval greater than 450 msec (males) or greater than 470 msec (females).

  11. Immunocompromised patients (other than that related to the use of corticosteroids) including patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).

  12. Clinically relevant retinal abnormalities as per the medical history or ophthalmologic findings in the pretreatment evaluation (e.g., retinitis pigmentosa or macular degeneration).

  13. History of thromboembolism within the past 5 years, history of catheter-related thrombophlebitis or other clinically significant thrombophlebitis are excluded.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
VLX600VLX600Dose of VLX600 in patients with refractory advanced solid tumors
Primary Outcome Measures
NameTimeMethod
Determination of the maximum tolerated dose (MTD) of VLX600MTD will be determined during dose escalation phase of study, up to one year.
Determination of the recommended Phase II dose (RPTD) of VLX600Determination of RPTD will be based on adverse event profile, up to one year.
Assessment of adverse event (AE) profileAEs are assessed from first treatment day through at least 30 days after last treatment.
Secondary Outcome Measures
NameTimeMethod
Description of the tumor response to VLX600 treatmentTumor response will be evaluated by CT scans or MRI at pre-treatment baseline and on Cycle 2 Day 28, then after every 8 weeks, and then at off-study (if greater than 4 weeks since prior tumor assessment).

Tumor response will be determined using CT scans or magnetic resonance imaging (MRI) at pre-treatment, Cycle 2 Day 28, at every 8 weeks thereafter, and at off-study (if a scan has not been performed within the last prior 4 weeks). Patients in the MTD confirmation cohort only will have a pre-treatment baseline positron emission tomography- computerized tomography (PET-CT) scan and then at Cycle 2 Day 28. Tumor response will be determined by Response Criteria in Solid Tumors (RECIST) version 1.1. In patients with disease not measurable by RECIST 1.1, blood samples for biomarkers of tumor response will be collected at pre-treatment baseline and then after every 8 weeks.

Description of progression-free survivalProgression-free survival is calculated from date of first treatment until date of first documented progression or date of death from any cause, whichever comes first, assessed up to 200 days (through 6 treatment cycles).
Description of the pharmacokinetics (PK) of VLX600PK assessed in Cycle 1 only. Day 1: pre-dose, 15, 30, 60 min, and 2, 4, 4.25, 4.5, 5, 8 hrs post start of infusion. Day 2: 24 hr post-start of infusion. Day 8: pre-dose, Day 15 (same as Day 1), Day 16: 24 hr post-start of infusion, and Day 22: pre-dose

Blood samples will be collected for PK analysis at the specified time points in Cycle 1 only.

Description of overall survival.Overall survival will be determined from date of first study treatment until death due to any cause, up to 72 weeks (duration of study).

Trial Locations

Locations (3)

Mayo Clinic Rochester

🇺🇸

Rochester, Minnesota, United States

Mayo Clinic Scottsdale

🇺🇸

Scottsdale, Arizona, United States

Mayo Clinic Jacksonville

🇺🇸

Jacksonville, Florida, United States

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