Ireland
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Latest
Active, not recruiting Phase 2

A Study of Berubicin in Adult Subjects With Recurrent Glioblastoma Multiforme

NCT04762069 · tracked via the Priya Life Science Italy tracker
Sponsor
CNS Pharmaceuticals, Inc
Phase
Phase 2
Started
2021-05-18
Last updated
2026-04-15

Condition(s) studied

Glioblastoma Multiforme, Adult

Investigational drug(s) / intervention(s)

BerubicinLomustine →

Berubicin: Berubicin HCl is a novel synthetic anthracycline with a chemical structure similar to doxorubicin HCl, a cytotoxic anthracycline topoisomerase II inhibitor isolated from cultures of Streptomyces peucetius var. caesius.

Lomustine: Lomustine is an anti-cancer ("antineoplastic" or "cytotoxic") chemotherapy drug. This medication is classified as an "alkylating agent.

Study summary

This is an open-label, multicenter, randomized, parallel, 2-arm, efficacy and safety study. Patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive berubicin or lomustine for the evaluation of OS. Additional endpoints will include response and progression outcomes evaluated by a blinded central reviewer for each patient according to RANO criteria.

A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Patients will be eligible for the study if they meet all of the following inclusion criteria and none of the exclusion criteria. Inclusion criteria 1. Written informed consent from the patient or their legally authorized representative (LAR) prior to any study-related procedure, and willing and able to comply with the protocol and aware of the investigational nature of this study. 2. At least 18 years of age. 3. KPS score of ≥ 60 4. A confirmed GBM diagnosis must be based on local review of tumor tissue from the initial biopsy, surgery, or re-resection. A formal pathology report confirming GBM is acceptable. It is not a requirement for slides to be sent to a central reviewer. 5. Recurrent or progressive GBM as evaluated by central review applying RANO criteria on contrast MRI scans of the Baseline/Screening MRI scan obtained up to six weeks prior to C1D1 and a historical scan taken before the Baseline/Screening scan that meets at least 1 of the following criteria: 1. In the case of measurable disease, progression will be documented by ≥ 25% increase in the sum of the perpendicular diameter products (SPDPs) of the measurable contrast-enhancing (target) lesions or any new measurable lesions. 2. If the SPDPs cannot be reliably estimated due to the lesion's complex conspicuity, shape, and contrast enhancement pattern, the volume of all measurable and non-measurable lesions may be used instead, applying the same threshold (≥ 25% increase) to confirm disease progression. 3. In the case of non-measurable lesions in the historical scan, any transformation into measurable lesions (≥10 mm in both maximum perpendicular diameters) in the Baseline/Screening scan will be evidence of progression. 4. If there are only non-measurable (non-target) lesions in the Baseline/Screening scan, additional lesions/sites will be considered evidence of progression based on the historical scan. Patients with new cerebrospinal fluid (CSF) seeding will not be considered eligible. 5. If historical scans are unavailable, a radiology report of a scan taken before the Baseline/Screening scan documenting the SPDPs from a previous scan of the enhancing disease or its volume can be used by the central reviewer to assess eligibility if it demonstrates the quality standards and acquisition guidelines required. 6. If the scan obtained during standard of care (prior to initiation of formal clinical screening and patient enrollment) is being used as the Baseline/Screening scan and does not entirely conform to central reader quality standards and acquisition guidelines (ie, artifacts or missing sequences), this can be used for the purpose of inclusion if the central reader in discussion with the sponsor and PI agree it provides evidence based on standard clinical practices of recurrence or progression. 7. Patients at first progression who are treated by re-resection or biopsy to confirm progression do not require measurable disease at their post-operative screening scan as their Baseline/Screening scan. These patients must be medically stable after the procedure as assessed by the PI and have the Baseline/Screening scan available by 7 days before starting treatment. 6. The tumor is localized supratentorially with no leptomeningeal (local or distant), spinal or CSF metastases, and no ventricular invasion (explicit documentation of the disease progression that would be problematic in evaluating the efficacy of this drug). 7. O\[6\] methylguanine-DNA methyltransferase (MGMT) methylation status must be available; results of routinely used methods for MGMT methylation testing (eg, methylation-specific polymerase chain reaction or quantitative polymerase chain reaction) are acceptable. 8. No more than 1 prior line of treatment (eg, surgery followed by radiation with concomitant chemotherapy, followed by adjuvant chemotherapy is considered as 1 line of treatment). In addition, treatment with tumor treating fields (TTFields; Optune) is acceptable if provided as first line therapy prior to progression or recurrence of disease. 9. A second debulking surgery, additional radiation or gamma knife surgery during the first line treatment or after progression, and for which the investigator does not suspect pseudoprogression is acceptable, as long as no chemotherapy or immunotherapy has been provided. 10. Recovery from toxicity/side effects of all prior therapy to Grade 1 or less, subject to the investigator's discretion, except for alopecia; the following time intervals from previous treatments are required to be eligible: 1. 12 weeks from the completion of radiation (to reduce risk of pseudoprogression), unless progression is confirmed by biopsy 2. 4 weeks from the end of any previous of chemotherapy 3. 2 weeks from tumor biopsy if wound completely healed 4. 4 weeks from any major surgery (maximal debulking surgery, either gross total resection or partial resection), gamma knife surgery or significant traumatic injury. Any surgery incisions or wounds must be completely healed 11. A stable or decreasing dose of corticosteroids (or none) for brain edema for at least 5 days prior to baseline MRI and enrollment in the study to document disease progression such that changes in the MRI are not related to the use of corticosteroids. 12. Eligible for chemotherapy based on adequate bone marrow function and organ function within 2 weeks of study treatment as defined by the following laboratory guidelines, subject to the investigator's discretion: 1. Hematopoietic function: total white blood cell (WBC) count ≥3 × 103/µL, absolute neutrophil count (ANC) ≥1.5 × 10³/µL, platelet count ≥75 × 10³/µL, hemoglobin ≥10 g/dL 2. Hepatic function: bilirubin ≤1.5 × × the upper limit of normal (ULN) (excluding Gilberts Syndrome, for which bilirubin must be ≤4 × ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<3 × ULN, and alkaline phosphatase ≤2.5 × ULN 3. Renal function: serum creatinine ≤1.5 × ULN or for patients with creatinine levels above the ULN, estimated creatinine clearance of ≥60 mL/min, calculated using the Cockcroft-Gault equation35 4. Activated partial thromboplastin time (aPTT) ≤1.5 × ULN 13. Women of childbearing potential must agree to practice a highly effective method of contraception beginning at least 28 days before the start of treatment until at least 6.25 months after the last dose of study drug. Male study patients and their female sexual partners of childbearing potential must agree to practice a highly effective method of contraception starting from the time of informed consent until at least 3.5 months (no less than 104 days) after the last dose of study drug. 1. A woman of childbearing potential is defined as a woman who is not permanently sterilized or postmenopausal. Postmenopausal is defined as 12 months with no menses without an alternative medical cause. 2. Women of childbearing potential must have a negative serum or urine pregnancy test at Screening. 3. A highly effective method of birth control is defined as one which results in a low failure rate (ie, less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence, or vasectomized partner. For patients using a hormonal contraceptive method, information regarding all medications being administered to the patient and their potential effects on the contraceptive should be addressed. 14. Patients with prior malignancies must be disease-free for ≥5 years. Curatively treated basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix, breast, or bladder; or prostate cancer as well as benign tumors that will not interfere with the treatment plan at the time of screening are allowed. Exclusion Criteria 1. Unable or not willing to comply with the protocol regulations. 2. Any additional chemotherapy (including but not limited to TMZ or immunotherapy) for recurrent or progressive GBM after a first line treatment. 3. Prior treatment with bevacizumab. 4. Prior treatment with lomustine. 5. Known to have an IDH mutation prior to enrollment 6. Screening/Baseline MRI showing a mass effect defined as significant compression of the ventricular system and/or a midline shift with associated clinical symptoms deemed inappropriate for the patient to enter a clinical trial. If there is otherwise asymptomatic compression and/or midline shift and the patient fulfills all other criteria, these patients are considered eligible. 7. Any condition (medical, social, psychological) that would prevent adequate information and follow-up, including but not limited to clinically relevant psychiatric disorders, legal incapacity, dementia, adults protected by law or altered mental status. 8. Presence of poorly controlled seizures, defined as occurring despite SOC or requiring hospitalization. 9. Prior anthracycline cumulative dose more than 550 mg/m2. 10. Heart disease: 1. LVEF \<50% 2. Unstable angina 3. CHF with New York Heart Association (NYHA) classification of 3 or 4 4. Patients with baseline QT/QTc interval \>480 msec, a history of additional risk factors for torsades de pointes (TdP) (eg, heart failure, hypokalemia, family history of long QT syndrome) and using concomitant medications that significantly prolong the QT/QTc interval 5. History of myocardial infarction within 12 months of enrollment 6. Severe arrhythmia not controlled by medication 11. Uncontrolled hypertension (systolic blood pressure \[BP\] \>150 mmHg and/or diastolic BP \>100 mmHg) sustained over 2 measurements. 12. Known to be positive for hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV), human immunodeficiency virus (HIV), COVID-19 (currently positive at time of screening), or any other acute viral, bacterial, or fungal infection (testing not required unless symptomatic or suspected disease). 13. Patients with any other uncontrolled intercurrent medical conditions, including but not limited to diabetes mellitus or chronic obstructive pulmonary disease that have not been well controlled by medical management over the prior 3 months are ineligible unless approved by the sponsor. 14. Women who are lactating or breastfeeding

Primary outcome measure(s)

  • Overall Survival — Through study completion an average of 4 years.
    To assess the effect of berubicin compared with lomustine on overall survival (OS) in adult patients with GBM that has recurred or progressed after standard initial therapy

Trial sites (48)

FacilityCityRegionStatus
University of Arkansas Little Rock Arkansas
Southern California Permanente Medical Group Los Angeles California
University of California Irvine Orange California
University of Califonia San Diego Moores Cancer Center San Diego California
University of California San Francisco San Francisco California
Saint John's Cancer Institute at Providence Saint John's Health Center Santa Monica California
HCA Healthcare Research Institute Englewood Colorado
Baptist MD Anderson Cancer Center Jacksonville Florida
Mayo Clinic Florida Jacksonville Florida
Baptist Miami Miami Florida
Piedmont Healthcare Atlanta Georgia
Rush University Cancer Center Chicago Illinois
University of Kentucky Lexington Kentucky
Tulane Cancer Center Clinic New Orleans Louisiana
Tufts Medical Center Boston Massachusetts
UMass (ACC) - Hollings Cancer Center (HCC) Worcester Massachusetts
Mayo Clinic Rochester Minnesota
University of Nebraska Medical Center Omaha Nebraska
Hackensack Meridian Health Hackensack New Jersey
Rutgers University Piscataway New Jersey
Atlantic Healthcare Summit New Jersey
Roswell Park Cancer Center Buffalo New York
Duke University School of Medicine Durham North Carolina
Ohio State University Columbus Ohio
Providence Health Portland Oregon
Milton S. Hershey Medical Center Hershey Pennsylvania
Texas Oncology PA Austin Texas
Baylor Research Institute Dallas Texas
University of Texas Health Science Center at Houston Houston Texas
Huntsman Cancer Center Salt Lake City Utah
Swedish Medical Center Seattle Washington
University of Wisconsin Hospital and Clinics Madison Wisconsin
Hopital Pierre Wertheimer Lyon France
Hopital de La Timone Marseille France
Institut de Recherche en Cancerologie de Montpellier Montpellier France
Hopital Pitie-Salpetriere Paris France
Institut de Cancerologie de l'Ouest Saint-Herblain France
nstitut Universitaire du Cancer de Toulouse- Toulouse France
Institut de Cancerologie Gustave-Roussy Villejuif France
Servizio Sanitario Regionale Emilia-Romagna - Azienda USL di Bologna - Ospedale Bellaria Bologna Italy

+ 8 more sites — see the full list on the official registry below.

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04762069 on ClinicalTrials.gov ↗ ← All trials in Italy