Trial Outcomes & Findings for A Phase 2a Study of Modified Vaccinia Virus to Treat Sorafenib-naïve Advanced Liver Cancer (NCT NCT01636284)
NCT ID: NCT01636284
Last Updated: 2021-01-20
Results Overview
Number of Participants with Complete response \[CR\] or partial response \[PR\] per modified Response Evaluation Criteria in Solid Tumors (mRECIST) for target and non-target lesions assessed by enhanced CT scan: CR, disappearance of intratumoral enhancing area; PR, \>=30% decrease in sum of diameters of enhancing area; Radiographic Response = CR + PR
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
CT scans every 6 week from Week 6 up to 12 months
Results posted on
2021-01-20
Participant Flow
Participant milestones
| Measure |
JX-594 Recombinant Vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
16
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase 2a Study of Modified Vaccinia Virus to Treat Sorafenib-naïve Advanced Liver Cancer
Baseline characteristics by cohort
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 Participants
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
14 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
54.3 Years
STANDARD_DEVIATION 13.14 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
South Korea
|
13 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: CT scans every 6 week from Week 6 up to 12 monthsNumber of Participants with Complete response \[CR\] or partial response \[PR\] per modified Response Evaluation Criteria in Solid Tumors (mRECIST) for target and non-target lesions assessed by enhanced CT scan: CR, disappearance of intratumoral enhancing area; PR, \>=30% decrease in sum of diameters of enhancing area; Radiographic Response = CR + PR
Outcome measures
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 Participants
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Number of Participants With Radiographic Response
|
4 Participants
|
SECONDARY outcome
Timeframe: CT scans every 6 week from Week 6 up to 12 months.TTP (in months) was defined as the number of months from the date of first Pexa-Vec infusion to the date of disease progression. If the patient had no progression then TTP was censored at the date of last evaluable tumor assessment. TTP was summarized and a Kaplan Meier (KM) curve was constructed.
Outcome measures
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 Participants
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Time to Progression (TTP)
|
1.38 months
Interval 1.35 to 2.73
|
SECONDARY outcome
Timeframe: CT scans every 6 week from Week 6 up to 12 monthsOS was defined as the time from first dose of Pexa-Vec until death from any cause. For patients not known to have died at the time of the analysis, OS was censored on the date they were last known to be alive. OS was summarized and a KM curve was constructed.
Outcome measures
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 Participants
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Overall Survival (OS)
|
7.47 months
Interval 4.44 to 10.64
|
Adverse Events
JX-594 Recombinant Vaccina GM-CSF
Serious events: 8 serious events
Other events: 16 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 participants at risk
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic Neoplasm Malignant
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Liver Carcinoma Ruptured
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases To Central Nervous System
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Hepatobiliary disorders
Hepatic Failure
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Hepatobiliary disorders
Hepatorenal Syndrome
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Ascites
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
Other adverse events
| Measure |
JX-594 Recombinant Vaccina GM-CSF
n=16 participants at risk
JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
|
|---|---|
|
General disorders
Chills
|
81.2%
13/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Influenza Like Illness
|
81.2%
13/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Pyrexia
|
56.2%
9/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Fatigue
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Oedema Peripheral
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Pain
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
General disorders
Chest Discomfort
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Abdominal Pain
|
37.5%
6/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Diarrhoea
|
37.5%
6/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Nausea
|
37.5%
6/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
31.2%
5/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Ascites
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
4/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Constipation
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Dyspepsia
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Abdominal Distension
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Gastrointestinal disorders
Stomatitis
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Nervous system disorders
Dizziness
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Nervous system disorders
Headache
|
43.8%
7/16 • From the date when ICF signed up to 12 months
|
|
Infections and infestations
Rash Pustular
|
31.2%
5/16 • From the date when ICF signed up to 12 months
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Infections and infestations
Herpes Zoster
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Infections and infestations
Acute Tonsillitis
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Infections and infestations
Skin Infection
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Aspartate Aminotransferase Increased
|
25.0%
4/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Platelet Count Decreased
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Alanine Aminotransferase Increased
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Blood Bilirubin Increased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Eosinophil Count Increased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Lymphocyte Count Decreased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Neutrophil Count Decreased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
Neutrophil Count Increased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Investigations
White Blood Cell Count Decreased
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Pain
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Pain In Extremity
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Flank Pain
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Productive Cough
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Skin and subcutaneous tissue disorders
Cold Sweat
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Skin and subcutaneous tissue disorders
Rash Generalised
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Vascular disorders
Hypotension
|
25.0%
4/16 • From the date when ICF signed up to 12 months
|
|
Blood and lymphatic system disorders
Anaemia
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Cardiac disorders
Tachycardia
|
18.8%
3/16 • From the date when ICF signed up to 12 months
|
|
Hepatobiliary disorders
Jaundice
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Hepatobiliary disorders
Hepatic Failure
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Hepatobiliary disorders
Hepatorenal Syndrome
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic Neoplasm Malignant
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Liver Carcinoma Ruptured
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases To Central Nervous System
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases To Spine
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Renal and urinary disorders
Dysuria
|
12.5%
2/16 • From the date when ICF signed up to 12 months
|
|
Injury, poisoning and procedural complications
Contusion
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Psychiatric disorders
Insomnia
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Muscular Weakness
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
25.0%
4/16 • From the date when ICF signed up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
6.2%
1/16 • From the date when ICF signed up to 12 months
|
Additional Information
Kyoung Soo Ha, Head of Clinical Development
Sillajen Biotherapeutics Inc.
Phone: +1-415-281-8886
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place