PCI Treatment/Gemcitabine & Chemotherapy vs Chemotherapy Alone in Patients With Inoperable Extrahepatic Bile Duct Cancer

NCT04099888 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: PCIA 203/18
• Study Type: interventional
• Target: 41
• Locations: 14 countries
• Sites: 50

Brief Summary

This study will assess the safety and effectiveness of fimaporfin-induced photochemical internalisation (PCI) of gemcitabine complemented by systemic gemcitabine/cisplatin chemotherapy compared to gemcitabine/cisplatin alone, in patients with inoperable cholangiocarcinoma (CCA). Participants will be randomly assigned to one of the treatment groups and will receive study treatment for 6 months, followed by assessments every 3 months, as applicable.

Conditions

Cholangiocarcinoma

Keywords

Phase II; Safety; Tolerability; Efficacy; Amphinex-induced Photochemical Internalisation; PCI; Amphinex; Gemcitabine; Cisplatin; Inoperable; CCA; Cholangiocarcinoma; Bile duct cancer; Photodynamic therapy; Extrahepatic; Perihilar; Distal; Fimaporfin; Pivotal; RELEASE; PDT; FimaChem; Klatskin; First line treatment; Standard of care; SOC; Chemotherapy; Local treatment

Outcome Measures

Primary Outcomes (1)

• 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 (12)

• Overall Survival (OS)

  Up to 24 months

• Best Overall Response (BOR)

  Up to 18 months

• Objective Response Rate (ORR)

  Up to 18 months

• Duration of Response (DoR)

  Up to 24 months

• Overall Disease Control Rate (DCR)

  6 months and 12 months

Study Arms (2)

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

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.

Also known as: PCI treatment

PCI treatment in conjunction with Standard of Care (SoC)

Gemcitabine/Cisplatin chemotherapy DRUG

Up to 8 cycles of gemcitabine/cisplatin combination chemotherapy will be administered.

Also known as: Standard of care (SoC)

PCI treatment in conjunction with Standard of Care (SoC); Standard of Care (SoC)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• PCI Biotech AS: lead

Study Sites (50)

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Emory University Hospital, 1365C Clifton Road NE

Atlanta, Georgia, 30322, United States

Location

University of Chicago Medical Center, 5841 South Maryland Avenue

Chicago, Illinois, 60637, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

The Mayo Clinic Hospital - Saint Mary's Campus, 1216 Second Street Southwest

Rochester, Minnesota, 55902, United States

Location

Baylor College of Medicine

Houston, Texas, 77096, United States

Location

UZ Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Odense Universitetshospital

Odense, 5000, Denmark

Location

Tampereen yliopistollinen sairaala, Syöpätautien klinikka

Tampere, FI-33520, Finland

Location

Centre Hospitalier Universitaire Grenoble Alpes - Hopital Albert Michallon

Grenoble, Cedex 09, 38043, France

Location

CHU Angers

Angers, Cedex 9, 49933, France

Location

CHU Dupuytren, 2 Avenue Martin Luther King

Limoges, 87042, France

Location

Institut Gustave Roussy, Département de gastro-entérologie

Villejuif, 94805, France

Location

Klinikum rechts der Isar der Technischen Universität München

München, Bavaria, 81675, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt am Main, Hesse, 60590, Germany

Location

Universitätsklinikum Essen

Essen, North Rhine-Westphalia, 45122, Germany

Location

Universitätsklinikum Bonn

Bonn, 53105, Germany

Location

Universitätsklinikum Hamburg Eppendorf, I. Medizinische Klinik und Poliklinik (Gastroenterologie mit Sektionen Infektiologie und Tropenmediz)

Hamburg, 20246, Germany

Location

Klinikum Landshut

Landshut, 84034, Germany

Location

LAKUMED Kliniken

Landshut, 84036, Germany

Location

Universität Leipzig KöR

Leipzig, 04103, Germany

Location

Klinikum Mannheim Universitätsklinikum gGmbH

Mannheim, 68167, Germany

Location

Klinikum der Ludwig-Maximilians-Universität MünchenKlinik

München, 81377, Germany

Location

Klinikum Nürnberg Nord, Medizinische Klinik 6 - (Schwerpunkte Gastroenterologie, Hepatologie, Endokrinologie)

Nuremberg, 90419, Germany

Location

Azienda Ospedaliero Universitaria Di Modena Policlinico

Modena, Emilia-Romagna, 41100, Italy

Location

IRCCS Saverio de Bellis, Via Turi, 27

Castellana Grotte, 70013, Italy

Location

Oslo Universitetssykehus HF Radiumhospitalet

Oslo, Norway

Location

Zaklad Opieki Zdrowotnej MSW z Warminsko-Mazurskim Centrum Onkologii

Olsztyn, Warmian-Masurian Voivodeship, 10-228, Poland

Location

Med-Polonia Sp. z o.o.

Poznan, 60-569, Poland

Location

National Cancer Center, 323 Ilsan-ro, Ilsandong-gu

Goyang-si, Gyeonggido, 10408, South Korea

Location

Pusan National University Hospital, 179 Gudeok-ro, Seo-gu

Busan, 49241, South Korea

Location

Kyungpook National University Chilgok Hospital, 807 Hoguk-ro, Buk-gu

Daegu, 41404, South Korea

Location

Asan Medical Center, 88 Olympic-ro 43-gil, Songpa-gu

Seoul, 05505, South Korea

Location

Severance Hospital Yonsei University Health System, 50-1, Yonsei-Ro, Seodaemun-Gu

Soeul, 03722, South Korea

Location

The Catholic University of Korea, Seoul St. Mary's Hospital, 222 Banpo-Daero Seocho-gu

Soeul, 06591, South Korea

Location

Clinica Universidad Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital del Mar

Barcelona, 08033, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario HM Sanchinarro - CIOCC

Madrid, 28050, Spain

Location

Hospital Universitario Puerta de Hierro - Majadahonda

Majadahonda, 28222, Spain

Location

Corporacio Sanitaria Parc Tauli

Sabadell, 08208, Spain

Location

Karolinska Universitetssjukhuset Solna, P.O Bäckencancer, Karolinska Universitetssjukhuset

Stockholm, Stockholms Ian, SE-17176, Sweden

Location

Taichung Veterans General Hospital, No. 1650 Taiwan Boulevard, Sec. 4

Taichung, 40705, Taiwan

Location

Taipei Veterans General Hospital, No. 201, Sction 2, Shi-pai Road

Taipei, 11217, Taiwan

Location

Chang Gung Memorial Hospital, Linkou, Dept. of Medical Oncology, 5 Fuxing Street, Guishan

Taoyuan District, 33305, Taiwan

Location

MNPE of Kharkiv Regional Council Regional Clinical Specialized Dispensary of Radiation Protection

Kharkiv, Ukraine

Location

SI Institute for General and Urgent Surgery n.a. V.T. Zaitseva of NAMS of Uktraine

Kharkiv, Ukraine

Location

SI "National Institute of Surgery and Transplantology n.a. O.O. Shalimov " of NAMS of Ukraine

Kyiv, Ukraine

Location

Municipal Nonprofit Enterprise City Hospital #3 of Zaporizhzhia City Council

Zaporizhzhya, Ukraine

Location

MeSH Terms

Conditions

Cholangiocarcinoma; Bile Duct Neoplasms

Interventions

Gemcitabine; Standard of Care

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Biliary Tract Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Heterocyclic Compounds; Deoxycytidine; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Limitations and Caveats

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.

Results Point of Contact

• Title: Chief Executive Officer
• Organization: PCI Biotech AS

post@pcibiotech.no

(+47) 67 11 54 00

Study Officials

• PCI Biotech

  PCI Biotech

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2019
• First Posted: September 23, 2019
• Study Start: May 23, 2019
• Primary Completion: April 28, 2022
• Study Completion: May 6, 2022
• Last Updated: September 11, 2023
• Results First Posted: September 11, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

BE (2); DK (1); SE (1); UA (4); IT (2); TW (3); FI (1); DE (11); NO (1); US (6); PL (2); KR (6); FR (4); ES (6)