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Study of ThermoDox With Standardized Radiofrequency Ablation (RFA) for Treatment of Hepatocellular Carcinoma (HCC)

Phase 3
Completed
Conditions
Hepatocellular Carcinoma
Interventions
Drug: ThermoDox
Drug: Dummy infusion
Registration Number
NCT02112656
Lead Sponsor
Imunon
Brief Summary

The purpose of this study is to determine whether ThermoDox, a thermally sensitive liposomal doxorubicin, is effective in the treatment of non-resectable hepatocellular carcinoma when used in conjunction with standardized radiofrequency ablation (sRFA).

Detailed Description

This is a Phase III, randomized, double blind, dummy controlled safety and efficacy study of ThermoDox plus sRFA compared to sRFA plus dummy infusion using standardized treatment dwell time for solitary HCC lesions ≥ 3.0 cm to ≤ 7.0 cm. An sRFA treatment for this protocol is defined as the dwell time of ≥ 45 minutes measured from the first activation of the RFA probe through removal of the RFA probe after the final ablation cycle or deployment.

The 50 mg/m2 ThermoDox or dummy infusion will be administered IV over 30 minutes. As part of blinded pre-medication ThermoDox treated subjects will receive 20 mg of dexamethasone orally 24 hours prior to the drug infusion for infusion reaction prophylaxis. Subjects on the control arm will receive a matching dummy pre-medication pill orally at 24 hours prior to infusion of the study treatment. Thirty minutes prior to receiving the ThermoDox infusion, subjects will receive a blinded dose of 20 mg of IV dexamethasone, 50 mg IV diphenhydramine and either 50 mg of IV ranitidine or 20 mg of IV famotidine. Subjects on the control arm will receive a masked dummy pre-medication pill orally at 24 hours prior to infusion of the study medication, and a dummy infusion 30 minutes prior to dummy infusion of Sodium Chloride 0.9% or 5% Dextrose (D5W). RFA will be initiated approximately at a minimum of 15 minutes after the initiation of study drug infusion and should be completed no later than 3 hours after study drug infusion initiation. The goal is to reach a \> 45 minute dwell time which can be achieved by employing at least four ablation cycles or deployments in order to ablate the tumor as well as a 360º 1.0 cm tumor-free margin surrounding the tumor.with an estimated overall procedure time of less than 3 hours.

A subject who has an incomplete ablation is eligible for 1 retreatment procedure within 21 days after the radiological imaging exam showing residual disease at Day 28. Subjects will be retreated only once with the same RFA equipment and treatment assigned at randomization. Subjects with a complete ablation after retreatment will be followed both for PFS and for OS.

If after 2 ablations the subject has local, distant intrahepatic, or extrahepatic HCC, then the subject will be considered a treatment failure and will have met the PFS endpoint. The subject will be followed for OS every 3 months. Among subjects who are not treatment failures, five repeat treatments are permitted to treat a recurrent lesion or to treat newly-identified local or distant intrahepatic lesions at the Investigator's discretion after the PFS endpoint is reported and with agreement from the Sponsor. The subject must be eligible for retreatment consistent with the safety eligibility criteria and will be retreated with the same randomized treatment.

CT or MRI imaging will be used to assess the effectiveness of the ablation therapy. The blind will be maintained at the level of the imaging reads. Investigator determined radiological progression must be observed and recorded prior to beginning alternate treatments for HCC. Posttreatment imaging will be obtained at months 1, 5, 9, 13, 17, 21, 25, then every 6 months (+/- 2 weeks) until radiological progression is seen. Adverse event assessments and laboratory examinations will occur at each visit. All subjects will be monitored throughout the investigational period.

Patients that meet inclusion/exclusion criteria may be at risk for contrast-induced nephropathy (CIN) when undergoing the required CT with contrast procedures. The investigators must be mindful of the risk factors associated with CIN and employ strategies to reduce the risk of CIN. In subjects with diabetes or borderline renal function (creatinine greater than 1.5 mg/dL) special precautions (e.g. hydration, contrast dose reduction, follow up creatinine determination) should be employed. An accepted procedure is adequate intravenous volume expansion with isotonic saline (1.0 - 1.5 mL/kg per hour) for 3-12 hours before the procedure and continued for 6-24 hours if clinically indicated and based on the treating physician's medical judgment.

All randomized subjects will be followed for safety and overall survival.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
554
Inclusion Criteria
  1. Male or female ≥ 18 years of age.

  2. Diagnosed with a single HCC lesion ≥ 3.0 cm but ≤ 7.0 cm in maximum diameter based on diagnosis at screening.

    • Subjects meeting the American Association for the Study of Liver Disease (AASLD) criteria may be randomized without a biopsy, but will undergo a biopsy during the RFA procedure unless contraindicated or unattainable.
    • Subjects not meeting the AASLD criteria for HCC will need a biopsy to confirm HCC prior to randomization.
  3. Be an appropriate candidate for receiving RFA as a medically indicated treatment as evaluated by the following factors:

    • The position and accessibility of the target lesion allows for the safe administration of multiple ablation cycles or deployments to achieve a probe dwell time of ≥ 45 minutes.
    • Not a candidate for surgical resection according to the local guidelines for resection and in the Investigator's judgment.
  4. Child-Pugh Class A without either current encephalopathy or ascites.

  5. Left Ventricular Ejection Fraction (LVEF) ≥ 50%.

  6. Eastern Cooperative Oncology Group (ECOG) performance status 0.

  7. Willing to sign an informed consent form, indicating awareness of the investigational nature of this study that is in keeping with the policies of the institution.

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Exclusion Criteria
  1. Is scheduled for liver transplantation

  2. Expected ablation volume > 30% of total liver volume or removal of 3 hepatic segments

  3. More than 1 lesion identified during baseline.

  4. Have previously received therapeutic treatment for HCC outside the study protocol or is expected to receive concomitant HCC treatment prior to PFS event.

  5. Have serious medical illnesses including, but not limited to, congestive heart failure, myocardial infarction or cerebral vascular accident within the last six months, or life threatening cardiac arrhythmias.

  6. Have previously received any anthracycline outside the protocol

  7. Have extrahepatic metastasis.

  8. Have portal or hepatic vein tumor invasion/thrombosis.

  9. Have body temperature >101ºF (38.3ºC) immediately prior to study treatment.

  10. Baseline laboratories (repeat lab tests are permitted to evaluate eligibility during the Screening Period. Lab results must be within protocol range prior to study treatment.)

    • Absolute neutrophil count < 1500/mm3
    • Platelet count < 75,000/mm3
    • Hgb < 10.0 g/dL (unless the hemoglobin value has been stable, the subject is cardiovascularly stable, asymptomatic and judged able to withstand the RFA procedure) Note: If clinically indicated, subjects may receive platelets or packed red blood cell (RBC) transfusions and be re-evaluated after condition is treated.
  11. Baseline Chemistry

    • Serum creatinine ≥ 2.5 mg/dL or calculated creatinine clearance (CrCl) ≤25.0 mL/min.
    • Serum bilirubin > 3.0 mg/dL.
    • Serum albumin < 2.8 g/dL.
  12. Have any known allergic reactions to any of the drugs or liposomal components or intravenous imaging agents that prohibit the ability to complete the imaging requirements.

  13. Are pregnant or breast-feeding. In women of childbearing potential, a negative serum pregnancy test is required prior to study treatment.

  14. Women of childbearing potential and men who are not practicing an acceptable form of birth control (i.e. diaphragm, cervical cap, condom, surgical sterility or birth control pills. Women whose partner has or men who have undergone a vasectomy must use a second form of birth control).

  15. Have INR > 1.5 times the institution's upper normal limit (UNL), except in subjects who are therapeutically anticoagulated for medical conditions unrelated to HCC such as atrial fibrillation. Subjects may be re-screened after condition is treated or anticoagulant is withheld.

  16. Have contraindications to receiving doxorubicin hydrochloride (HCl).

  17. Are being treated with other investigational agents.

  18. Use of an investigational drug outside this study within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study medication.

  19. Have other concurrent malignancy (subjects with treated squamous cell carcinoma of the skin or basal cell carcinoma of the skin may be included), evidence of extrahepatic cancer from their primary malignancy, or ongoing, medically significant active infection.

  20. HIV positive.

  21. NYHA class III or IV functional classification for heart failure.

  22. Evidence of hemachromatosis.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ThermoDox 50 mg/m2ThermoDoxThermoDox plus standardized RFA using standardized treatment dwell time for solitary HCC lesions ≥ 3.0 cm to ≤ 7.0 cm
Dummy infusionDummy infusionstandardized RFA alone using standardized treatment dwell time for solitary HCC lesions ≥ 3.0 cm to ≤ 7.0 cm
Primary Outcome Measures
NameTimeMethod
Overall Survival (OS)All subjects to be contacted every 3 months after radiological progression for vital status reporting. Subjects were followed for OS up to 68 months from randomization.

Overall survival is defined as the time (in months) from the date of randomization to the death from any cause or the end of the study.

Secondary Outcome Measures
NameTimeMethod
Progression-free Survival (PFS)CT or MRI scan (Chest, Abdomen, Pelvis) done at Baseline and Day 28. Additional imaging done at months 5, 9, 13, 17, 21, 25, then every 6 months until disease progression is seen. Study subjects were followed up to 63 months after randomization.

The protocol incorporates modified RECIST developed for HCC clinical research as a basis to evaluate tumor response. PFS here is defined as the time (in months) from the date of randomization to the first date on which one of the following occurs (as determined by CT or MRI scan):

* Death of any cause

* Treatment failure (inability to achieve CR after two RFA ± ThermoDox treatment sessions)

* Progression due to local tumor recurrence after initial CR

* Progression due to distant intrahepatic tumor recurrence

* Progression due to extrahepatic tumor recurrence

Trial Locations

Locations (61)

Zhongshan Hospital, Fudan University

🇨🇳

Shanghai, China

302 Military Hospital of China

🇨🇳

Beijing, China

Peking University First Hospital

🇨🇳

Beijing, China

Xijing Hospital

🇨🇳

Xi'an, Shaanxi, China

UCLA Department of Medicine

🇺🇸

Los Angeles, California, United States

Toronto General Hospital

🇨🇦

Toronto, Ontario, Canada

Mengchao Hepatobiliary Hospital of Fujian Medicatl University

🇨🇳

Fuzhou, Fujian, China

Beijing Hospital of the Ministry of Health

🇨🇳

Beijing, China

Beijing Cancer Hospital, School of Oncology, Peking

🇨🇳

Beijing, China

Chinese PLA General Hospital

🇨🇳

Beijing, China

West China Hospital of Sichuan University

🇨🇳

Chengdu, China

The Second Hospital of Dalian Medical University

🇨🇳

Dalian, China

Hunan Cancer Hospital

🇨🇳

Hunan, China

Guangdong General Hospital

🇨🇳

Guangdong, China

The First Hospital of Jilin University

🇨🇳

Jilin, China

The Sixth People's Hospital of Shenyang

🇨🇳

Shenyang, China

The 3rd Hospital of Tianjing

🇨🇳

Tianjin, China

The First Hospital of Zhejiang

🇨🇳

ZheJiang, China

Zhejiang Cancer Hospital

🇨🇳

Zhejiang, China

Universitaetsklinikum des Saarlandes, Klik fuer Allgemeine Chirurgie, Viszeral-, Gefaess und Kinderchirurgie

🇩🇪

Homburg, Germany

Institut für Diagnostische und Radiologische Therapie del Uniklinik Frankfurt

🇩🇪

Frankfurt, Germany

Klinikum rechts der Isar, II. Medizinische Klinik und Poliklinik (Gastroenterologie)

🇩🇪

München, Germany

Universitätsklinikum Regensburg, Institut für Röntgendiagnostik

🇩🇪

Regensburg, Germany

Department of Radiological Sciences and Bioimaging Catholic University of Rome, "A. Gemelli" Hospital

🇮🇹

Rome, Italy

Cisanello Hospital, Division of Diagnostic Imaging and Intervention

🇮🇹

Pisa, Italy

Pusan National University Hospital

🇰🇷

Busan, Korea, Republic of

Kyungpook National University Hospital

🇰🇷

Daegu, Korea, Republic of

Kyungpook National University Medical Center

🇰🇷

Daegu, Korea, Republic of

Severance Hospital, Yonsei University Health System

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Inha University Hospital

🇰🇷

Incheon, Korea, Republic of

The Catholic University of Korea, Seoul St.Mary's Hospital

🇰🇷

Seoul, Korea, Republic of

Chinese General Hospital and Medical Center

🇵🇭

Manila, Philippines

University Malaya Medical Centre

🇲🇾

Kuala Lumpur, Malaysia

Cardinal Santos Medical Center

🇵🇭

San Juan, Philippines

Hospital Universitario Marqués de Valdecilla

🇪🇸

Santander, Spain

Hospital Madrid Norte Sanchinarro

🇪🇸

Madrid, Spain

Taichung Veteran General Hospital

🇨🇳

Taichung, Taiwan

National Cheng Kung University (NCKU) Hospital

🇨🇳

Tainan, Taiwan

Srinagarind Hospital

🇹🇭

Khon Kaen, Thailand

Thammasat University Hospital

🇹🇭

Pathumthani, Thailand

Bach Mai Hospital

🇻🇳

Hà Nội, Dong Da District, Vietnam

108 Military Central Hospital

🇻🇳

Hà Nội, Hai Ba Trung District, Vietnam

Hue Central Hospital

🇻🇳

Huế, Vin Ninh Ward, Vietnam

Bach Mai Hospital (Hepato-gastroenterology Department)

🇻🇳

Hanoi, Vietnam

Viet Duc University Hospital

🇻🇳

Hanoi, Vietnam

National Cancer Hospital

🇻🇳

Hanoi, Vietnam

Queen Mary Hospital

🇭🇰

Hong Kong, Hong Kong

St. Lukes Medical Center

🇵🇭

Quezon City, Philippines

Singapore General Hospital

🇸🇬

Singapore, Singapore

Chang Gung Memorial Hospital - Kaohsiung

🇨🇳

Kaohsiung, Taiwan

Maharaj Nakorn Chiang Mai Hospital

🇹🇭

Chiang Mai, Thailand

Can Tho Oncology Hospital

🇻🇳

Hanoi, Vietnam

Taipei Medical University-Shuang Ho Hospital

🇨🇳

New Taipei City, Taiwan

Siriraj Hospital

🇹🇭

Bangkok, Thailand

People's Hospital 115

🇻🇳

Ho Chi Minh City, Vietnam

National Taiwan University Hospital

🇨🇳

Taipei City, Taiwan

Chang Gung Memorial Hospital - Linkou

🇨🇳

Taoyuan, Taiwan

National Taiwan University Hospital, Yun-Lin Branch

🇨🇳

Yuanlin, Taiwan

Songklanagarind Hospital

🇹🇭

Songkhla, Thailand

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