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Efficacy and Safety Study of Talimogene Laherparepvec Compared to Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) in Melanoma

Phase 3
Completed
Conditions
Melanoma
Interventions
Biological: GM-CSF
Biological: Talimogene laherparepvec
Registration Number
NCT00769704
Lead Sponsor
BioVex Limited
Brief Summary

The objective of this study is to evaluate the efficacy and safety of treatment with talimogene laherparepvec compared to subcutaneously administered GM-CSF in patients with unresectable Stage IIIb, IIIc and Stage IV melanoma. The efficacy endpoints of the study aim to demonstrate overall clinical benefit for patients treated with talimogene laherparepvec as compared to GM-CSF.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
437
Inclusion Criteria
  • Males or females age ≥ 18 years
  • Stage IIIb, IIIc or stage IV disease that is not surgically resectable
  • Injectable disease (i.e. suitable for direct injection or through the use of ultrasound guidance)
  • At least 1 injectable cutaneous, subcutaneous or nodal melanoma lesion >= 10 mm in longest diameter or, multiple injectable melanoma lesions which in aggregate have a longest diameter of >= 10 mm
  • Serum lactate dehydrogenase (LDH) levels less than 1.5 x upper limit of normal (ULN)
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
  • Prolongation in International Normalized Ratio (INR), Prothrombin Time (PT), and Partial Thromboplastin Time (PTT) when the result is from therapeutic anticoagulation treatment are permitted for patients whose injectable lesions are cutaneous and/or subcutaneous such that direct pressure could be applied in the event of excessive bleeding
Exclusion Criteria
  • Clinically active cerebral or any bone metastases. Patients with up to 3 (neurological performance status of 0) cerebral metastases may be enrolled, provided that all lesions have been adequately treated with stereotactic radiation therapy, craniotomy, gammaknife therapy, with no evidence of progression, and have not required steroids, for at least two (2) months prior to randomization
  • Greater than 3 visceral metastases (this does not include lung metastases or nodal metastases associated with visceral organs). For patients with < 3 visceral metastases, no lesion > 3 cm, and liver lesions must meet Response Evaluation Criteria In Solid Tumors (RECIST) criteria for stable disease for at least 1 month prior to randomization

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
GM-CSFGM-CSFGranulocyte macrophage colony-stimulating factor (GM-CSF) was administered at a dose of 125 μg/m²/day subcutaneously for 14 days, followed by a 14-day rest period for 24 weeks. Participants could continue treatment until clinically relevant disease progression, intolerability, withdrawal of consent, complete remission, or lack of response by 12 months, for a maximum of 18 months.
Talimogene LaherparepvecTalimogene laherparepvecParticipants received talimogene laherparepvec on Days 1 and 15 of each 28-day cycle for 24 weeks. The initial dose of talimogene laherparepvec was at a concentration of 10⁶ plaque forming units (PFU)/mL, injected into 1 or more skin, subcutaneous or nodal tumors. Subsequent doses began at least 3 weeks after the first dose and consisted of talimogene laherparepvec at a concentration of 10⁸ PFU/mL. Participants could continue treatment until clinically relevant disease progression, intolerability, withdrawal of consent, complete remission, lack of response by 12 months, or disappearance of all injectable lesions, for a maximum of 18 months.
Primary Outcome Measures
NameTimeMethod
Durable Response RateFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

Durable response rate was defined as the percentage of participants with a complete response (CR) or partial response (PR) maintained continuously for at least 6 months from the time the objective response was first observed and initiating within 12 months of starting therapy as assessed by the Endpoint Assessment Committee (EAC). This reflects all new sites of disease as well as disease sites identified at baseline.

Disease assessments were performed at the beginning of each treatment cycle in accordance with modified World Health Organization criteria.

CR: Disappearance of all clinical evidence of tumor (both measurable and non-measurable but evaluable disease); PR: ≥ 50% reduction in the sum of the products of the perpendicular diameters of all measurable tumors at the time of assessment as compared to baseline.

Secondary Outcome Measures
NameTimeMethod
Overall SurvivalFrom randomization until the first 290 survival events had occurred (data cut-off date of 31 March 2014); median time on follow-up was 44 months.

Overall survival was defined as the time from the date of randomization to the date of death from any cause. Overall survival time was censored at the last date the patient was known to be alive when the confirmation of death was absent or unknown. Participants were censored at the date of randomization if no additional follow-up data were obtained.

Duration of ResponseFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

The duration of response is defined as the longest individual period from entering response (CR or PR as assessed by the EAC) to the first documented evidence of the patient no longer meeting the criteria for being in response or death, whichever is earlier. Responses were censored at the last assessment showing response.

Response OnsetFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

Response onset is defined as the time from the date of randomization to the date of the first documented evidence of response (CR or PR) per EAC assessment.

Objective Response RateFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

Objective response rate was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) assessed by the Endpoint Assessment Committee (EAC). Best overall response for a patient is the best overall response observed across all time points.

Disease assessments were performed at the beginning of each treatment cycle and assessed in accordance with modified World Health Organization criteria.

CR: Disappearance of all clinical evidence of tumor (both measurable and non-measurable but evaluable disease); PR: ≥ 50% reduction in the sum of the products of the perpendicular diameters of all measurable tumors at the time of assessment as compared to baseline.

Time to Treatment FailureFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

Time to treatment failure was assessed by the investigator, and calculated from randomization until the first clinically relevant disease progression where there is no response achieved after the progression, or until death if no such progression occurs. Participants who did not have clinically relevant progression or did not die were censored at the time of the their last tumor assessment. Participants who withdrew from treatment due to a clinically unacceptable toxicity were not considered as an event in the analysis.

Progressive disease (PD) is defined as a ≥ 25% increase in the sum of the products of the perpendicular diameters of all measurable tumors since baseline, or the unequivocal appearance of a new tumor since the last response assessment time point.

Clinically relevant progressive disease is PD that is associated with a decline in performance status and/or in the opinion of the investigator the patient requires alternative therapy.

Response IntervalFrom randomization until the data cut-off date of 21 December 2012; median follow-up time was 20 months.

Response interval is defined as the interval between the date of randomization and the date of the last documented evidence of response (CR or PR as assessed by the Investigator) prior to any new anti-cancer therapy. Response Interval post response onset was censored if a patient was still in response at the last observation.

Trial Locations

Locations (83)

MD Anderson Cancer Center Orlando

🇺🇸

Orlando, Florida, United States

New Mexico Cancer Care Alliance

🇺🇸

Albuquerque, New Mexico, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Institute for Translational Oncology Research

🇺🇸

Greenville, South Carolina, United States

Mount Sinai School of Medicine

🇺🇸

New York, New York, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

University of North Carolina At Chapel Hill School of Medicine

🇺🇸

Chapel Hill, North Carolina, United States

St Luke's Hospital & Health Network

🇺🇸

Bethlehem, Pennsylvania, United States

Columbia Medical University

🇺🇸

New York, New York, United States

Wake Forest University School of Medicine

🇺🇸

Winston Salem, North Carolina, United States

University of California San Diego, Moores Cancer Center

🇺🇸

La Jolla, California, United States

James Graham Brown Cancer Center

🇺🇸

Louisville, Kentucky, United States

Mount Sinai Medical Center CCOP

🇺🇸

Miami Beach, Florida, United States

Broomfield Hospital

🇬🇧

Chelmsford, United Kingdom

Jewish General Hospital

🇨🇦

Montreal, Quebec, Canada

Kansas City Cancer Center

🇺🇸

Kansas City, Missouri, United States

Addenbrookes Hospital

🇬🇧

Cambridge, United Kingdom

Mountainside Hospital

🇺🇸

Morristown, New Jersey, United States

Texas Cancer Center, Abilene

🇺🇸

Abilene, Texas, United States

Mary Potter Oncology Centre

🇿🇦

Pretoria, Guateng, South Africa

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Clatterbridge Centre for Oncology

🇬🇧

Wirral, United Kingdom

Dr. Fourie & Bonnet

🇿🇦

Bloemfontein, Free State, South Africa

University of Pretoria

🇿🇦

Pretoria, Guateng, South Africa

Royal Free Hospital

🇬🇧

London, United Kingdom

Churchill Hospital

🇬🇧

Oxford, United Kingdom

Royal Marsden Hospital

🇬🇧

London, United Kingdom

Wits Donald Gordon Clinical Trial Site

🇿🇦

Parktown, Guateng, South Africa

Wilgers Oncology Center

🇿🇦

Hatfield, Pretoria, South Africa

Cross Cancer Institute

🇨🇦

Edmonton, Alberta, Canada

Palm Beach Cancer Institute

🇺🇸

West Palm Beach, Florida, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

UCLA Medical Center

🇺🇸

Los Angeles, California, United States

Hubert H Humphrey Cancer Center

🇺🇸

Robbinsdale, Minnesota, United States

Gabrail Cancer Center

🇺🇸

Canton, Ohio, United States

Cleveland Clinic Foundation, Taussig Cancer Center

🇺🇸

Cleveland, Ohio, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

Cancer Care Center at Lutheran General Hospital

🇺🇸

Park Ridge, Illinois, United States

Mary Crowley Medical Research Center

🇺🇸

Dallas, Texas, United States

Princess Margaret Hospital

🇨🇦

Toronto, Ontario, Canada

Medical Oncology Centre of Rosebank

🇿🇦

Johannesburg, Gauteng, South Africa

GVI Onocology Clinical Trials Unit

🇿🇦

Cape Town, Western Cape, South Africa

GVI Oncology Centre

🇿🇦

Cape Town, Western Cape, South Africa

Massachusetts General Hospital Cancer Center

🇺🇸

Boston, Massachusetts, United States

Indiana University

🇺🇸

Indianapolis, Indiana, United States

Investigative Clinical Research of Indiana

🇺🇸

Indianapolis, Indiana, United States

University of Texas - MD Anderson

🇺🇸

Houston, Texas, United States

Intermountain Medical Center

🇺🇸

Murray, Utah, United States

John Wayne Cancer Institute

🇺🇸

Santa Monica, California, United States

University of Arizona Cancer Center

🇺🇸

Tucson, Arizona, United States

University of Arkansas for Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

Redwood Regional Medical Group Inc, North Bay Melanoma Program

🇺🇸

Sebastopol, California, United States

Baptist Cancer Institute

🇺🇸

Jacksonville, Florida, United States

Lakeland Regional Cancer Center

🇺🇸

Lakeland, Florida, United States

University of Iowa Hospitals & Clinics

🇺🇸

Iowa City, Iowa, United States

Texas Oncology, Allison Cancer Center

🇺🇸

Midland, Texas, United States

Oncology and Hematology Associates of Southwest Virginia, Inc.

🇺🇸

Salem, Virginia, United States

Hopelands Cancer Centre

🇿🇦

Pietermaritzburg, Kwa-Zulu Natal, South Africa

University of Birmingham

🇬🇧

Birmingham, United Kingdom

Freeman Hospital

🇬🇧

Newcastle upon Tyne, United Kingdom

Southampton General Hospital

🇬🇧

Southampton, United Kingdom

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

San Francisco Oncology Associates

🇺🇸

San Francisco, California, United States

Northern California Melanoma Center, St. Mary's Medical Center

🇺🇸

San Francisco, California, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

Barrett Cancer Center

🇺🇸

Cincinnati, Ohio, United States

Vanderbilt Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

St. George's University of London

🇬🇧

London, United Kingdom

St. James's University Hospital

🇬🇧

Leeds, United Kingdom

Nottingham City Hospital

🇬🇧

Nottingham, United Kingdom

St. Louis University Hospital

🇺🇸

St. Louis, Missouri, United States

University of Colorado Cancer Center

🇺🇸

Aurora, Colorado, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

H. Lee Moffitt Cancer Center

🇺🇸

Tampa, Florida, United States

Methodist Estabrook Cancer Center

🇺🇸

Omaha, Nebraska, United States

Aurora/St. Luke's Medical Center

🇺🇸

Milwaukee, Wisconsin, United States

Earle A Chiles Research Institute, Providence Cancer Center

🇺🇸

Portland, Oregon, United States

University of Miami

🇺🇸

Miami, Florida, United States

GVI Oncology

🇿🇦

Port Elizabeth, Eastern Cape, South Africa

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

Rhode Island Hospital

🇺🇸

Providence, Rhode Island, United States

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