Hilar Cholangiocarcinoma
Conditions
Keywords
Cholangiocarcinoma, Unresectable perihilar cholangiocarcinoma, Klatskin tumor, Photodynamic therapy, Porfimer sodium, Photofrin, Gemcitabine, Cisplatin, Stents, CCA, PDT, Bile duct cancer, Bile duct tumor, Bile duct adenocarcinoma, Chemotherapy
Brief summary
Photodynamic therapy (PDT) is a combination of a drug, porfimer sodium (Photofrin), which is activated by a light from a laser that emits no heat. This technique works to allow the medical doctor to specifically target and destroy abnormal or cancer cells while limiting damage to surrounding healthy tissue. The activation of the drug is done by lighting the abnormal areas using a fiber optic device (very fine fiber like a fishing line that permits light transmission) inserted into a flexible tube with a light called cholangioscope for the bile duct. The light will activate the porfimer sodium concentrated in the abnormal tissue, leading to its destruction. This research study will evaluate the efficacy and safety of PDT with porfimer sodium administered with Standard Medical Care (SMC) compared to SMC alone on the overall survival time of patients with non-operable advanced cholangiocarcinoma, a rare cancer of the bile ducts. It will involve 200 patients across North America and Europe. Other countries may participate if needed. Participation will last at least 18 months.
Detailed description
Photodynamic therapy (PDT) is a combination of a drug, porfimer sodium (Photofrin), which is activated by a light from a laser that emits no heat. This technique works to allow the medical doctor to specifically target and destroy abnormal or cancer cells while limiting damage to surrounding healthy tissue. The activation of the drug is done by lighting the abnormal areas using a fiber optic device (very fine fiber like a fishing line that permits light transmission) inserted into a flexible tube with a light called cholangioscope for the bile duct. The light will activate the porfimer sodium concentrated in the abnormal tissue, leading to its destruction. Cholangiocarcinoma (CCA) is defined as primary malignant tumors of the bile ducts. The exact etiology remains unknown. These cancerous tumors block the bile flow and can be intrahepatic (IH) or extrahepatic (EH). The distinction between IH- and EH-CCA has become increasingly important, as the epidemiological features (i.e., incidence and risk factors), the biologic and pathologic characteristics and the clinical course are largely different. Unfortunately, most subjects are found to have metastases or unresectable disease at the time of diagnosis. Median survival for subjects with unresectable perihilar-CCA varies between five and eight months. The one-year survival is 50%, with 20% surviving at two years and 10% at three years. Unresected CCA is a rapidly fatal process with cholangitis being a significant cause of morbidity and mortality in these subjects. This study was designed to confirm the efficacy of PHOPDT + standard medical care (SMC) defined as stents plus gemcitabine/cisplatin chemotherapy regimen on the overall survival of subjects with unresectable cholestasis perihilar Bismuth type III or IV - tumor TNM stage III or IVa CCA.
Interventions
DRUGPhotodynamic therapy-Photofrin
Photodynamic therapy (PDT) involves the i.v. injection of Photofrin (2 mg/kg) followed by the illumination of the tumor using a fiber optic device during an endoscopic retrograde cholangiopancreatography (ERCP) or percutaneous transhepatic cholangiography (PTC). Two days after the injection, a laser light (180 J/cm(2)) will be applied to the tumor. A second light application will be given 96-120 hours after Photofrin injection if PDT could not initially be performed on all sides of the tumor. Post illumination, all patients will undergo stenting as part of standard medical care procedure. Up to 3 additional courses of PDT using a light dose of 120 J/cm(2) may be given at 3-month intervals.
PROCEDUREStenting procedure
As per standard medical procedures, stenting procedure consists in the placement of stents above the main tumors of the right and left hepatic bile ducts via endoscopic retrograde cholangiopancreatography (ERCP) or percutaneous transhepatic cholangiography (PTC) when the ERCP approach has been unsuccessful.
The regimen will comprise gemcitabine (1 000 mg/m(2)) followed by cisplatin (25 mg/m(2)), each administered on days 1 and 8 every 3 weeks (21 day-cycle) for four cycles. An additional 12 weeks of the same chemotherapy regimen may be administered if there is no disease progression or intolerable toxicity.
Sponsors
Concordia Laboratories Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males or females aged 18 or older * Diagnosed with radiologically and biopsy or cytology confirmed inoperable perihilar cholangiocarcinoma Bismuth Tumor Stage III/IV * Non-menopausal or non-sterile female subjects of childbearing potential must have a negative serum beta-HCG and use a medically acceptable form of birth control * Able to sign an informed consent
Exclusion criteria
* Diagnostic of cholangiocarcinoma made more than 45 days prior to randomization * Cholangiocarcinoma with extra-hepatic metastasis or concurrent non-solid malignancy * Presence or history of other neoplasms (treated during the last five years prior to study entry) other than carcinoma in situ of the cervix or basal carcinoma of the skin * Previously received photodynamic therapy for cholangiocarcinoma * Previously undergone surgical resection of the cholangiocarcinoma * Previously undergone chemotherapy, brachytherapy, or radiotherapy prior to entering the study * Previously undergone metal stent insertion * Porphyria or hypersensitivity to porphyrins (constituents of porfimer sodium), gemcitabine, cisplatin or other platinum-containing compounds * Presence of infection other than the infection of the bile duct (cholangitis) * Acute or chronic medical or psychological illnesses that prevent endoscopy procedures * Abnormal blood test results * Severe impairment of your kidney or liver function * Decompensated cirrhosis * Pregnant or intend to become pregnant, breastfeeding or intend to breast-feed during this study * Participated in another drug study within 90 days before this one * Unable or unwilling to complete the follow-up evaluations required for the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival Time | Up to 26 months | Time from the date of randomization until the date of death or the last date the subject was known to be alive |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Best Overall Tumor Response as Measured by the RECIST 1.1 Criteria (Response Evaluation Criteria in Solid Tumors) | Up to 26 months | From the start of the treatment until disease progression or recurrence the RECIST 1.1 criteria are applied (Response Evaluation Criteria in Solid Tumors) |
| Time-to-tumor Progression | Up to 26 months | From the date of first documented response until the date that tumor progression was assessed |
| Change From Baseline on Karnofsky Performance Scale (KPS) | Baseline, 7 days | The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease. |
| Time-to-bilirubin Response | Up to 30 days | From the date of randomization until the date of first documented bilirubin response |
| Change From Baseline in Health-related Quality of Life on the 4- and 7-point European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) | Baseline, 7 days | Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life. |
| Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | Baseline, up to 4 weeks | Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life. |
| Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | Baseline, up to 4 weeks | The Karnofsky Performance Scale (KPS) scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. A score of 100% means there are no complaints and no evidence of disease. A score of 80% means there is normal activity with effort and some signs or symptoms of disease. A score of 0% means death. |
Countries
Canada, Germany, South Korea, Switzerland, United States
Participant flow
Pre-assignment details
An independent expert panel acted as central assessors for subjects across all sites. Central assessors prospectively interpreted all abdominal CT and cholangiogram images taken at screening to establish the final diagnosis and to confirm the randomization. Central assessors had the authority to overrule the investigator's diagnosis.
Participants by arm
| Arm | Count |
|---|---|
| Photodynamic Therapy-Photofrin Plus SMC Photodynamic therapy (PDT) involved the i.v. injection of Photofrin (2 mg/kg) followed by the illumination of the tumor using a fiber optic device. Two days after the injection, a laser light (180 J/cm(2)) was applied to the tumor. A second light application was given 96-120 hours after Photofrin injection if PDT could not initially be performed on all sides of the tumor. Post illumination, all patients underwent stenting. Up to 3 additional courses of PDT using a light dose of 120 J/cm(2) could be given at 3-month intervals.
As per SMC, stenting procedure consisted of the placement of stents above the main tumors of the right and left hepatic bile ducts. The chemotherapy regimen comprised gemcitabine (1 000 mg/m(2)) followed by cisplatin (25 mg/m(2)), each administered on days 1 and 8 every 3 weeks (21 day-cycle) for four cycles. An additional 12 weeks of the same chemotherapy regimen could be administered if there was no disease progression or intolerable toxicity. | 15 |
| Standard Medical Care (SMC) Standard Medical Care (SMC) was defined as stenting procedure plus chemotherapy regimen. The chemotherapy regimen comprised gemcitabine (1 000 mg/m(2)) followed by cisplatin (25 mg/m(2)), each administered on days 1 and 8 every 3 weeks (21 day-cycle) for four cycles. An additional 12 weeks of the same chemotherapy regimen could be administered if there was no disease progression or intolerable toxicity. As per SMC, stenting procedure consisted of the placement of stents above the main tumors of the right and left hepatic bile ducts. | 13 |
| Total | 28 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Chemotherapy change | 1 | 3 |
| Overall Study | Death | 1 | 0 |
| Overall Study | Disease progression | 2 | 2 |
| Overall Study | Lost to Follow-up | 1 | 0 |
| Overall Study | Physician Decision | 3 | 1 |
| Overall Study | Sponsor termination | 5 | 3 |
| Overall Study | Withdrawal by Subject | 2 | 4 |
Baseline characteristics
| Characteristic | Photodynamic Therapy-Photofrin Plus SMC | Standard Medical Care (SMC) | Total |
|---|---|---|---|
| Age, Continuous | 63.4 years STANDARD_DEVIATION 11.84 | 66.6 years STANDARD_DEVIATION 7.69 | 64.9 years STANDARD_DEVIATION 10.08 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 12 Participants | 10 Participants | 22 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 3 Participants | 3 Participants | 6 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 5 Participants | 8 Participants | 13 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 9 Participants | 4 Participants | 13 Participants |
| Region of Enrollment Germany | 1 participants | 1 participants | 2 participants |
| Region of Enrollment South Korea | 5 participants | 8 participants | 13 participants |
| Region of Enrollment United States | 9 participants | 4 participants | 13 participants |
| Sex: Female, Male Female | 3 Participants | 5 Participants | 8 Participants |
| Sex: Female, Male Male | 12 Participants | 8 Participants | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 15 | 0 / 13 |
| other Total, other adverse events | 13 / 15 | 10 / 10 |
| serious Total, serious adverse events | 11 / 15 | 10 / 10 |
Outcome results
Primary
Overall Survival Time
Time from the date of randomization until the date of death or the last date the subject was known to be alive
Time frame: Up to 26 months
Population: All participants randomized (intent-to-treat population)
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Overall Survival Time | 444 days |
| Standard Medical Care (SMC) | Overall Survival Time | 387 days |
Secondary
Best Overall Tumor Response as Measured by the RECIST 1.1 Criteria (Response Evaluation Criteria in Solid Tumors)
From the start of the treatment until disease progression or recurrence the RECIST 1.1 criteria are applied (Response Evaluation Criteria in Solid Tumors)
Time frame: Up to 26 months
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Best Overall Tumor Response as Measured by the RECIST 1.1 Criteria (Response Evaluation Criteria in Solid Tumors) | 56 percentage of participants |
| Standard Medical Care (SMC) | Best Overall Tumor Response as Measured by the RECIST 1.1 Criteria (Response Evaluation Criteria in Solid Tumors) | 75 percentage of participants |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 78 weeks
Population: No data collected
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, up to 4 weeks
Population: Not collected
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 13 weeks
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -7.1 score on a scale | Standard Deviation 30.59 |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | 6.2 score on a scale | Standard Deviation 35.84 |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 16 weeks
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -21.3 score on a scale | Standard Error 33.88 |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | 10.4 score on a scale | Standard Error 31.1 |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 29 weeks
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -12.5 score on a scale | Standard Deviation 35.03 |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | 5.0 score on a scale | Standard Deviation 18.26 |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 41 weeks
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | 8.3 score on a scale | Standard Deviation 11.79 |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -16.7 score on a scale | Standard Deviation 23.57 |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 54 weeks
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -8.3 score on a scale |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30 | -33.3 score on a scale |
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point EORTC QLQ-C30
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 66 weeks
Population: No data collected
Secondary
Change From Baseline in Health-related Quality of Life on the 4- and 7-point European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30)
Final European Organisation for Research and Treatment of Cancer (EORTC) scores for multi-item scales and single-item measures will range from 0 to 100. This questionnaire assesses the quality of life of cancer patients. A high score represents a high/healthy level of functioning, basically a high Quality of Life.
Time frame: Baseline, 7 days
Population: Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Health-related Quality of Life on the 4- and 7-point European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) | -2.2 score on a scale | Standard Deviation 30.29 |
| Standard Medical Care (SMC) | Change From Baseline in Health-related Quality of Life on the 4- and 7-point European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) | 0.0 score on a scale | Standard Deviation 0 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 66 weeks
Population: Data not collected
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 78 weeks
Population: Not collected
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 54 weeks
Population: At 54 weeks, only 1 subject in each group was measured. Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0.0 score on a scale | Standard Deviation 0 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0.0 score on a scale | Standard Deviation 0 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale (KPS) scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. A score of 100% means there are no complaints and no evidence of disease. A score of 80% means there is normal activity with effort and some signs or symptoms of disease. A score of 0% means death.
Time frame: Baseline, 16 weeks
Population: At 16 weeks, 9 subjects in the PDT +SMC group were analysed and 9 in the SMC Alone group were analysed. Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | -6.7 score on a scale | Standard Deviation 15 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 3.3 score on a scale | Standard Deviation 5 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 29 weeks
Population: At 29 weeks, 6 subjects in the PDT +SMC group were analysed and 5 in the SMC Alone group were analysed. Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0.0 score on a scale | Standard Deviation 8.94 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 2.0 score on a scale | Standard Deviation 4.47 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 41 weeks
Population: At 41 weeks, 2 subjects in the PDT +SMC group were analysed and 1 in the SMC Alone group was analysed. Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 10.0 score on a scale | Standard Deviation 14.14 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0.0 score on a scale | Standard Deviation 0 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale (KPS) scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. A score of 100% means there are no complaints and no evidence of disease. A score of 80% means there is normal activity with effort and some signs or symptoms of disease. A score of 0% means death.
Time frame: Baseline, up to 4 weeks
Population: At 4 weeks, 12 subjects in the PDT +SMC group were analysed and 9 in the SMC Alone group were analysed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 1.7 score on a scale | Standard Deviation 5.77 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0 score on a scale | Standard Deviation 0 |
Secondary
Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale (KPS) scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. A score of 100% means there are no complaints and no evidence of disease. A score of 80% means there is normal activity with effort and some signs or symptoms of disease. A score of 0% means death.
Time frame: Baseline, 13 weeks
Population: At 13 weeks, 8 subjects in the PDT +SMC group were analysed and 8 in the SMC Alone group were analysed. Due to the premature termination of the study, there was insufficient data to allow statistical analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 0.0 score on a scale | Standard Deviation 7.56 |
| Standard Medical Care (SMC) | Change From Baseline in Performance Status on the Karnofsky Performance Scale (KPS) | 2.5 score on a scale | Standard Deviation 4.63 |
Secondary
Change From Baseline on Karnofsky Performance Scale (KPS)
The Karnofsky Performance Scale scores range from 0% to 100%. The lower the Karnofsky score, the worse likelihood of survival. However, the premature termination of the study does not allow for a meaningful analysis of the scale where 100% means no complaints with no evidence of disease, 80% is normal activity with effort and some signs or symptoms of disease.
Time frame: Baseline, 7 days
Population: At Day 7, all 15 subjects in the PDT +SMC group were analysed and 9/13 in the SMC Alone group were analysed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Photodynamic Therapy-Photofrin Plus SMC | Change From Baseline on Karnofsky Performance Scale (KPS) | -0.7 score on a scale | Standard Deviation 7.99 |
| Standard Medical Care (SMC) | Change From Baseline on Karnofsky Performance Scale (KPS) | 0 score on a scale | Standard Deviation 0 |
Secondary
Time-to-bilirubin Response
From the date of randomization until the date of first documented bilirubin response
Time frame: Up to 30 days
Population: Data not collected
Secondary
Time-to-tumor Progression
From the date of first documented response until the date that tumor progression was assessed
Time frame: Up to 26 months
Population: Not collected