PCI Treatment/Gemcitabine & Chemotherapy vs Chemotherapy... | NCT04099888

NCT04099888

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
PCI treatment in conjunction with Standard of Care (SoC)	Experimental	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy	Drug: Fimaporfin and Gemcitabine Drug: Gemcitabine/Cisplatin chemotherapy
Standard of Care (SoC)	Active Comparator	Arm B: Gemcitabine/cisplatin chemotherapy	Drug: Gemcitabine/Cisplatin chemotherapy

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Fimaporfin and Gemcitabine	Drug	PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Progression-free Survival (PFS)	From date of randomisation to date of objective disease progression or death, whichever comes first (in months)	Up to 18 months

Secondary Outcomes

Measure	Description	Time Frame
Overall Survival (OS)	From date of randomisation to date of death from any cause (in months)	Up to 24 months
Best Overall Response (BOR)	Best response recorded from start of treatment until disease progression/recurrence (according to RECIST 1.1)

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Each patient must provide signed and witnessed written informed consent and agree to comply with study protocol requirements.
Histopathologically/cytologically verified adenocarcinoma consistent with cholangiocarcinoma (CCA). Must have biliary lesion causing bile obstruction that requires stenting and is accessible for PCI light treatment (ie, extrahepatic CCA [perihilar or distal] only).
CCA must be considered inoperable with respect to radical resection.
At least 1 radiologically evaluable lesion (measurable and/or non-measurable) that can be assessed at baseline and is suitable for repeated radiological evaluation.
If metastatic, metastases must be limited tissues other than bone or the central nervous system.
Must have adequate biliary drainage (at least 50% of the liver volume or at least 2 sectors) with no evidence of active uncontrolled infection (patients on antibiotics are eligible).
Must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
Estimated life expectancy of at least 12 weeks.

Exclusion Criteria:

Patients who have previously received any anti-tumor (either local or systemic) treatment for CCA, except for previous treatment of up to 2 cycles of gemcitabine/cisplatin.
Patients with severe visceral disease other than CCA.
A history of frequently recurring septic biliary events.
Patients with porphyria or hypersensitivity to porphyrins.
Patients with a second primary cancer with a disease-free interval of <5 years. A second primary cancer that has been treated with intent to cure may be allowed after consultation with the study Medical Monitor. Adequately treated basal cell carcinoma, squamous cell carcinoma or other non-melanomatous skin cancer, in-situ carcinoma of the uterine cervix, or prostate cancer that is controlled by hormone therapy (patients may continue hormone therapy while on study) are allowed.
Patients not able to undergo contrast-enhanced CT or MRI.
Patients currently participating in any other interventional clinical trial.
Planned surgery, endoscopic examination or dental treatment in the first 30 days after PCI treatment.
Co-existing ophthalmic disease likely to require slit-lamp examination within the first 90 days after PCI treatment.
Clinically significant and uncontrolled cardiac disease except for extra systoles or minor conduction abnormalities and controlled and well-treated chronic atrial fibrillation.
Known allergy or sensitivity to photosensitisers (active substance and/or any of the excipients); or chronic use of other photosensitising therapies; treatment with amiodarone during the last 12 months.
Known hypersensitivity to or contraindication to the use of gemcitabine (active substance and/or any of the excipients).
Known hypersensitivity to or contraindication to the use of cisplatin (active substance and/or any of the excipients).
Patients with ataxia telangiectasia.
Upon the Investigator's discretion, evidence of any other medical conditions (such as psychiatric illness, physical examination or laboratory findings) that may interfere with the planned PCI treatment, affect patient compliance or place the patient at high risk from treatment-related complications.
Patients planning to have or who have recently had vaccination with a live vaccine.
Patients concurrently receiving treatment with phenytoin.
Male patients unwilling to use highly effective contraception or female patients of childbearing potential unwilling to use highly effective form of contraception. Patients must continue the use of contraception during PCI treatment and subsequent chemotherapy for at least 6 months thereafter.
Women who are breastfeeding or who have a positive pregnancy test at baseline.
Patients with inadequate bone marrow function (absolute neutrophil count <1.5 x 10^9/L; platelet count <100 x 10^9/L; haemoglobin <6 mmol/L [transfusion allowed]).
Inadequate liver function despite satisfactory drainage (serum bilirubin persisting at >5 x upper limit of normal for the institution; aspartate aminotransferase or alanine aminotransferase >3.0 x upper limit of normal or >5 x upper limit of normal if liver metastases are present; alkaline phosphatase levels >5.0 x upper limit of normal).
Inadequate renal function, as determined by local practice for patients on fractionated platinum-based chemotherapy. Patients with creatinine clearance <45 mL/min (in France: <60 mL/min) must not be included.

Other protocol-defined criteria may apply.

Contacts/Locations Module

Name	Affiliation	Role
PCI Biotech	PCI Biotech	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
City of Hope National Medical Center		Duarte	California	91010	United States
Emory University Hospital, 1365C Clifton Road NE

References Module

No data available

No data is available for this block.

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Uploaded Document Information

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Jul 13, 2020

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

Masking Info

Intent-to-treat (ITT) Analysis Set

Arm/Group Information

ID	Title	Description
BG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy. Length of cycle is 3 weeks with PCI treatment at Day 1. Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
BG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy. Length of cycle is 3 weeks. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
BG002	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00068.1± 7.30 BG00164.4± 11.43 BG00266.3± 9.61

Age, Customized

Count of Participants

Participants

Title

Denominators

Categories

<50 years of age

Title	Measurements
	BG0000 BG0012 BG002

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0007 BG00111 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

White

Title	Measurements
	BG00011 BG00111 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Progression-free Survival (PFS)

From date of randomisation to date of objective disease progression or death, whichever comes first (in months)

Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).

Posted

Median

95% Confidence Interval

Months

Up to 18 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants	OG00019 OG00115

Title

Denominators

Categories

Title	Measurements
	OG0007.29(6.00 to 12.00) OG0018.08(6.00 to 12.00)

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001										2-Sided	95							Superiority		The HR was estimated using an unstratified cox-proportional hazards model using the Efron approach for handling ties (Efron 1977), together with the associated 95% confidence intervals (CI) for the HR based on the Wald method. The effect of treatment is summarized by the hazard ratio (HR) together with its corresponding 95% Wald CI for the mITT population. No p-value will be reported.

Secondary

Overall Survival (OS)

From date of randomisation to date of death from any cause (in months)

Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).

Posted

Count of Participants

Participants

Up to 24 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units

Secondary

Best Overall Response (BOR)

Best response recorded from start of treatment until disease progression/recurrence (according to RECIST 1.1)

Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).

Posted

Count of Participants

Participants

Up to 18 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Secondary

Objective Response Rate (ORR)

Proportion of patients with measurable disease at baseline who have at least one visit response with a complete response (CR) or partial response (PR) noted (according to RECIST 1.1)

Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).

Posted

Count of Participants

Participants

Up to 18 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Secondary

Duration of Response (DoR)

From first documented tumour response until first documented disease progression, or death in the absence of disease progression (in months)

Posted

Number

Days

Up to 24 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants

Secondary

Overall Disease Control Rate (DCR)

Proportion of patients with BOR of CR, PR or stable disease (SD) (according to RECIST 1.1) at or after the first follow-up scan, partial response or complete response

Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).

Posted

Count of Participants

Participants

6 months and 12 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Secondary

Change in Tumor Size

Best overall percentage change in tumour size from baseline

Only patients with measurable disease at baseline are included in this summary.

Posted

Mean

Standard Deviation

Percentage Change in Tumor Size

Up to 18 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts

Secondary

Loco-regional Tumour-related Events and Biliary Complications

Frequency and severity of loco-regional tumour related events and biliary complications

Posted

Count of Participants

Participants

Up to 12 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants

Secondary

Adverse Events (AEs)/Serious Adverse Events (SAEs)

Number and proportion of patients with AEs/SAEs

Posted

Count of Participants

Participants

Up to 12 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants

Secondary

Area Under the Plasma Concentration Curve (AUC) Was Performed for Patients in Arm A.

A non-compartmental analysis (NCA) was applied on the data. AUC from time zero to the last measured concentration (AUC 0-t) was initially estimated by the linear trapezoidal method.

AUC was calculated for each single patient and not for the population since the study was terminated and the amount of data was limited. No mean and standard deviation was calculated for the population

Posted

Number

(ng/ml)*hrs

Time Frame AUC calculated from time zero to C5-D8 (3 months from the first PCI treatment)

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants

Secondary

Maximum Observed Concentration (Cmax) Was Performed for Patients in Arm A.

A non-compartmental analysis (NCA) was applied on the data.

Maximum observed concentration (Cmax) was performed for patients in arm A.

Posted

Number

ng/ml

Timepoints for pharmacokinetic (PK) sampling: Day -4 (before, 30m and 4hrs after Amphinex), C1-D1, C1-D8, C2-D8, C3-D8, C4-D8, C4-D18 (before, 30m and 4hrs after Amphinex), C5-D1, and C5-D8

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants	OG000

Secondary

Time to Cmax (Tmax) Was Performed for Patients in Arm A.

A non-compartmental analysis (NCA) was applied on the data as described by Gabrielsson & Weiner (Methods in molecular biology, 929:161-180, 2012).

A standard non-compartmental PK analysis (NCA) of bioanalytical data from 13 patients dosed with fimaporfin was performed for the RELEASE study.

Posted

Number

hours

Timepoints for PK sampling: Day -4 (before, 30 min and 4 hours after Amphinex), C1-D1, C1-D8, C2-D8, C3-D8, C4-D8, C4-D18 (before, 30 min and 4 hours after Amphinex) , C5-D1, and C5-D8

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts
Participants

Secondary

Health-related Quality of Life (QoL)

QoL assessment. Patients select one of four answers to 22 questions ranging from 1 (not at all) to 4 (very much). Lower total scores indicate a more favorable QoL perception than a higher score.

The RELEASE study did not collect QoL data.

Posted

Up to 18 months

ID	Title	Description
OG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.
OG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Units	Counts

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	PCI Treatment in Conjunction With Standard of Care (SoC)	Arm A: Fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by gemcitabine/cisplatin chemotherapy Fimaporfin and Gemcitabine: PCI treatment consists of IV administration of Amphinex solution for injection (investigational product) at 0.22 mg/kg dose of fimaporfin, followed 4 days later by a standard dose of gemcitabine infusion (1000 mg/m²) and intraluminal laser light application. Up to 2 PCI treatments will be given. Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.	7	19	12	18	18	18
EG001	Standard of Care (SoC)	Arm B: Gemcitabine/cisplatin chemotherapy Gemcitabine/Cisplatin chemotherapy: Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.	2	15	8	16	16	16

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Duodenal ulcer	Gastrointestinal disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Pancreatitis acute	Gastrointestinal disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Pyrexia	General disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0011 events1 affected16 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Transaminases increased	Investigations	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Cholangitis	Hepatobiliary disorders	MedDRA 23.1	Systematic Assessment	EG0009 events6 affected18 at risk EG0017 events3 affected16 at risk
Cholangitis acute	Hepatobiliary disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0012 events2 affected16 at risk
Biliary obstruction	Hepatobiliary disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Cholecystitis	Hepatobiliary disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0011 events1 affected16 at risk
Biliary tract infection	Infections and infestations	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0011 events1 affected16 at risk
COVID-19	Infections and infestations	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Cholangitis infective	Infections and infestations	MedDRA 23.1	Systematic Assessment	EG0002 events1 affected18 at risk EG0010 events0 affected16 at risk
Sepsis	Infections and infestations	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Stent malfunction	Product Issues	MedDRA 23.1	Systematic Assessment	EG0004 events2 affected18 at risk EG0012 events1 affected16 at risk
Device occlusion	Product Issues	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Aortic stenosis	Vascular disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Aortic thrombosis	Vascular disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Tumour necrosis	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Toxic encephalopathy	Nervous system disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Pneumonia aspiration	Respiratory, thoracic and mediastinal disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Pulmonary oedema	Respiratory, thoracic and mediastinal disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Gastrointestinal disorders	Gastrointestinal disorders	MedDRA 23.1	Systematic Assessment	EG00030 events10 affected18 at risk EG00135 events12 affected16 at risk
General disorders and administration site conditions	General disorders	MedDRA 23.1	Systematic Assessment	EG00013 events9 affected18 at risk EG00113 events8 affected16 at risk
Investigations	Investigations	MedDRA 23.1	Systematic Assessment	EG00045 events6 affected18 at risk EG00110 events1 affected16 at risk
Blood and lymphatic system disorders	Blood and lymphatic system disorders	MedDRA 23.1	Systematic Assessment	EG0008 events1 affected18 at risk EG00112 events3 affected16 at risk
Hepatobiliary disorders	Hepatobiliary disorders	MedDRA 23.1	Systematic Assessment	EG0008 events4 affected18 at risk EG0016 events3 affected16 at risk
Skin and subcutaneous tissue disorders	Skin and subcutaneous tissue disorders	MedDRA 23.1	Systematic Assessment	EG00016 events8 affected18 at risk EG0016 events3 affected16 at risk
Metabolism and nutrition disorders	Metabolism and nutrition disorders	MedDRA 23.1	Systematic Assessment	EG0007 events3 affected18 at risk EG0017 events6 affected16 at risk
Infections and infestations	Infections and infestations	MedDRA 23.1	Systematic Assessment	EG0008 events5 affected18 at risk EG0011 events1 affected16 at risk
Nervous system disorders	Nervous system disorders	MedDRA 23.1	Systematic Assessment	EG0009 events6 affected18 at risk EG0010 events0 affected16 at risk
Injury, poisoning and procedural complications	Injury, poisoning and procedural complications	MedDRA 23.1	Systematic Assessment	EG0009 events4 affected18 at risk EG0010 events0 affected16 at risk
Musculoskeletal and connective tissue disorders	Musculoskeletal and connective tissue disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0015 events4 affected16 at risk
Product issues	Product Issues	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Renal and urinary disorders	Renal and urinary disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0013 events3 affected16 at risk
Vascular disorders	Vascular disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0011 events1 affected16 at risk
Eye disorders	Eye disorders	MedDRA 23.1	Systematic Assessment	EG0002 events2 affected18 at risk EG0011 events1 affected16 at risk
Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0011 events1 affected16 at risk
Psychiatric disorders	Psychiatric disorders	MedDRA 23.1	Systematic Assessment	EG0002 events2 affected18 at risk EG0011 events1 affected16 at risk
Respiratory, thoracic and mediastinal disorders	Respiratory, thoracic and mediastinal disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0012 events2 affected16 at risk
Cardiac disorders	Cardiac disorders	MedDRA 23.1	Systematic Assessment	EG0001 events1 affected18 at risk EG0010 events0 affected16 at risk
Ear and labyrinth disorders	Ear and labyrinth disorders	MedDRA 23.1	Systematic Assessment	EG0000 events0 affected18 at risk EG0011 events1 affected16 at risk

Due to the early termination of this study, no definitive conclusions can be drawn since an insufficient number of participants was recruited. A reduced statistical analysis was conducted.

Certain Agreements

First MCP Posted Date

ID	Term
D018281	Cholangiocarcinoma
D001650	Bile Duct Neoplasms

Ancestor Terms

ID	Term
D000230	Adenocarcinoma
D002277	Carcinoma
D009375	Neoplasms, Glandular and Epithelial
D009370	Neoplasms by Histologic Type
D009369	Neoplasms
D001661	Biliary Tract Neoplasms
D004067	Digestive System Neoplasms
D009371	Neoplasms by Site
D001649	Bile Duct Diseases
D001660	Biliary Tract Diseases
D004066	Digestive System Diseases

ID	Term
D000093542	Gemcitabine
D059039	Standard of Care

Ancestor Terms

ID	Term
D006571	Heterocyclic Compounds
D003841	Deoxycytidine
D003562	Cytidine
D011741	Pyrimidine Nucleosides
D011743	Pyrimidines
D006573	Heterocyclic Compounds, 1-Ring
D019984	Quality Indicators, Health Care
D011787	Quality of Health Care
D006298	Health Services Administration
D017530	Health Care Quality, Access, and Evaluation

Units	Counts
Participants	OG00019 OG00115

Units	Counts
Participants	OG00019 OG00115