Borderline Resectable CarcinomaPhase 2ESR-22-21719NCT06017297
Neoadjuvant Tremelimumab and Durvalumab With Gem/Cis in Intrahepatic Cholangiocarcinoma
A Phase 2 study testing Durvalumab, Tremelimumab and Gemcitabine in people with Borderline Resectable Carcinoma and Biliary Tract Cancer, run by Georgetown University.
Trial summary
The goal of this clinical trial is to test feasibility and safety of the combination of tremelimumab and durvalumab plus gemcitabine and cisplatin as a neoadjuvant treatment bridge patients to a curative resection in treatment naïve borderline resectable, or resectable with high risk for recurrence intrahepatic… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Borderline Resectable Carcinoma, Biliary Tract Cancer
- What is being tested?
- Durvalumab, Tremelimumab and Gemcitabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2024to Nov 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients of age 18 years and older at the time of study entry, both sexes, all ethnicities.
- Patients with histologically proven intrahepatic cholangiocarcinoma, untreated with systemic therapy.
- Patients with an absence of extrahepatic metastasis (outside of periportal lymph node enlargement) or peritoneal carcinomatosis as demonstrated by CT-scan.
- Patients with a performance status ECOG 0, 1.
- Patients with an estimated life expectancy > 6 months.
- Patients with disease that is not readily suitable for resection with curative intent, as validated by a multidisciplinary committee with at least one hepatobiliary surgeon, defined as stage II and stage III disease, surgical resectable if there is tumor shrinkage.
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- Patients with at least one measurable lesion according to RECIST 1.1 criteria. Tumor assessment by computed tomography (CT) scan or magnetic resonance imaging (MRI) must be performed within 28 days prior to start of study.
- Patients with platelets ≥ 75,000/mm3, polynuclear neutrophils ≥ 1500/mm3, hemoglobin ≥ 9g/dL.
- Patients with serum creatinine < 1.5 times institutional upper limit of normal (ULN), measured creatinine clearance > 40 mL/min or Calculated creatinine clearance > 40 mL/min by the Cockcroft-Gault formula (Cockcroft and Gault 1976) or by 24 hour urine collection for determination of creatinine clearance.
- Patients with serum bilirubin ≤ 1.5 times institutional upper limit of normal (ULN) (after biliary drainage if necessary).
- Absolute neutrophil count (ANC ≥ 1.0 × 109 /L)
- Aspartate aminotransferase (AST or SGOT) / alanine transaminase (ALT or SGPT) ≤ 2.5 x institutional upper limit of normal unless liver metastases are present, in which case it must be ≤5x ULN
- Patients with a reference CT Scan within 30 days preceding the 1st cycle of treatment.
- Patients with US health insurance coverage.
- Patients are capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act in the US, European Union [EU] Data Privacy Directive in the EU) obtained from the patient/legal representative prior to performing any protocol-related procedures, including screening evaluations.
- Patients are willing and able to comply with the protocol for the duration of the study including undergoing treatment and able to comply with the protocol for the duration of the study including undergoing treatment and scheduled visits and examinations including follow up.
- Body weight ≥30 kg
Who cannot join
- Patients with hilar or distal cholangiocarcinoma or those with hepatocholangiocarcinoma.
- Patients who are eligible for surgical resection or liver transplantation based on tumor characteristics.
- Patients who would not be surgical candidates due to reasons unrelated to their cholangiocarcinoma, e.g. Cirrhosis with portal hypertension
- Patients with extrahepatic metastases beyond periportal lymph node enlargement
- Patients with a contraindication or grade 3-4 allergy to durvalumab, tremelimumab, gemcitabine, cisplatin, or capecitabine.
- Any unresolved toxicity NCI CTCAE Grade ≥2 from previous anticancer therapy with the exception of alopecia, vitiligo, and the laboratory values defined in the inclusion criteria
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- Patients with Grade ≥2 neuropathy will be evaluated on a case-by-case basis after consultation with the Study Physician.
- Patients with irreversible toxicity not reasonably expected to be exacerbated by treatment with durvalumab or tremelimumab may be included only after consultation with the Study Physician.
- Radiotherapy treatment to more than 30% of the bone marrow or with a wide field of radiation within 4 weeks of the first dose of study drug
- Major surgical procedure (as defined by the Investigator) within 28 days prior to the first dose of Investigational Product (IP). Note: Local surgery of isolated lesions for palliative intent is acceptable
- Active or prior documented autoimmune or inflammatory disorders (including inflammatory bowel disease [e.g., colitis or Crohn's disease], diverticulitis [with the exception of diverticulosis], systemic lupus erythematosus, Sarcoidosis syndrome, or Wegener syndrome [granulomatosis with polyangiitis, Graves' disease, rheumatoid arthritis, hypophysitis, uveitis, etc.]). The following are exceptions to this criterion:
- Patients with vitiligo or alopecia
- Patients with hypothyroidism (e.g., following Hashimoto syndrome) stable on hormone replacement
- Any chronic skin condition that does not require systemic therapy
- Patients without active disease in the last 5 years may be included but only after consultation with the study physician
- Patients with celiac disease controlled by diet alone
- History of leptomeningeal carcinomatosis
- Mean QT interval corrected for heart rate using Fridericia's formula (QTcF) ≥470 ms calculated from 3 ECGs (within 15 minutes at 5 minutes apart)
- Prior randomization or treatment in a previous durvalumab and/or tremelimumab clinical study regardless of treatment arm assignment.
- Patients with an active autoimmune disease that has required systemic treatment in the past 2 years with the use of disease-modifying agents, corticosteroids, or immunosuppressive drugs. Exceptions to this criterion include intranasal, inhaled, topical steroids or local steroid injections, systemic corticosteroids at physiologic doses not to exceed 10mg/day or prednisone or its equivalent, or steroids as premedication for hypersensitivity reactions.
- Patients with a history of allogenic organ transplantation.
- Patients with a history of non-infectious pneumonitis that required steroids or has current pneumonitis.
- Patients who are recipients of a live attenuated vaccine within 30 days prior to the dose of durvalumab.
- Patients with poorly controlled diarrhea (grade ≥ 2).
- Coinfection of hepatitis B virus (HBV) and hepatitis C virus (HCV) as determined by viral load. HBV infection is allowed only if patient is on HBV treatment per institutional guideline. HCV infection is allowed.
- Patients known to have tested positive for human immunodeficiency virus (HIV) (positive HIV 1/2 antibodies) or active tuberculosis infection (clinical evaluation that may include clinical history, physical examination and radiographic findings, or tuberculosis testing in line with local practice)
- Patients with a serious, non-stabilized disease, active uncontrolled infection, or other serious underlying disorders that would likely prevent the patient from receiving therapy.
- Patients who are pregnant, breast-feeding, or of child-bearing age with a refusal to use effective contraception.
- Patients with another cancer, active within the 5 years preceding or at the time of inclusion in this trial. Note: Patients with early-stage cancer that has been resected/ablated/radiated, without evidence of disease recurrence or progression, within 2 years may be considered.
- Patients with legal incapacity.
- Patients who are deprived of civil liberty.
- Patients for whom it is impossible to sign the informed consent document or to adhere to medical follow-up of the trial for geographical, social, or psychological reasons.
- Patients who have participation in another clinical study with an investigational product during the last 4 weeks.
- Patients who are concurrently enrolled in another clinical study, unless it is an observational (non-interventional) clinical study or during the follow-up period of an interventional study.
- Patients who are judged by the investigator that the patient is unsuitable to participate in the study and the patient is unlikely to comply with the study procedures, restrictions and requirements.
About this study
Official title: Phase II Study of Neoadjuvant Durvalumab (MEDI4736) and Tremelimumab in Combination With Gemcitabine and Cisplatin in Patients With Intrahepatic Cholangiocarcinoma That is Borderline Resectable/Resectable But With High Risk for Recurrence.
The goal of this clinical trial is to test feasibility and safety of the combination of tremelimumab and durvalumab plus gemcitabine and cisplatin as a neoadjuvant treatment bridge patients to a curative resection in treatment naïve borderline resectable, or resectable with high risk for recurrence intrahepatic cholangiocarcinoma patients. The main question[s] it aims to answer are:
- What is the rate of conversion of unresectable tumor to resectable cancer?
- What are the side effects of this treatment combination?
Participants will undergo an initial tumor biopsy, imaging and laboratory studies prior to starting treatment with durvalumab, tremelimumab, gemcitabine and cisplatin. Participants will continue for 4 cycles and if the tumor is found to be resectable then they will undergo surgical resection. If the tumor is unresectable (can't be surgically removed) after 4 cycles, then participants will receive 4 more cycles and repeated imaging. If the tumor remains unresectable then the participant will be treated with capecitabine for up to 8 cycles and durvalumab for up to 12 months.
- Being tested
- Durvalumab, Tremelimumab, Gemcitabine, Cisplatin, Surgical Resection
- Medical subject headings
- Biliary Tract Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DurvalumabDrug
Durvalumab 1500 mg via intravenous (IV) infusion every 3 weeks for up to 8 cycles
TremelimumabDrug
A single dose of tremelimumab at 300mg IV is given on C1.
GemcitabineDrug
Gemcitabine is dosed at 1000 mg/m2 IV on day (D)1 and D8 of each cycle.
CisplatinDrug
Cisplatin is dosed at 25mg/m2 on D1 and D8 of each cycle.
Surgical ResectionProcedure or surgery
If the tumor is evaluated to be resectable (as defined as successfully treated stage II (tumor shrink away from vessels), stage IIIA (tumor shrink away from visceral peritoneum), stage IIIB (N1 disease no longer pathologically enlarged) after C4 or C8, then the patient may proceed with surgical tumor resection.
| Study group | Role | What they receive |
|---|---|---|
| Durvalumab and Tremelimumab plus gemcitabine/cisplatin | Experimental | Durvalumab and tremelimumab plus gemcitabine/cisplatin combination therapy. If the tumor is evaluated to be resectable after Cycle 4 (C4), then the patient may proceed with surgical tumor resection. If the tumor is deemed unresectable after C4, then the patient will proceed with Cycle 5-8 followed by reevaluation for surgical resection. Receives: Drug: Durvalumab; Drug: Tremelimumab; Drug: Gemcitabine; Drug: Cisplatin; Procedure: Surgical Resection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Conversion from unresectable to resectable
Rate of conversion of unresectable tumor to resectable cancer after neoadjuvant durvalumab + tremelimumab + gemcitabine + cisplatin after 4 or 8 cycles. Surgical evaluation will be done in joint by institutional radiology and hepatobiliary surgery using clinical data (CT/MRI imaging, patient performance status, labs, etc.) If among the evaluable 24 patients, 9 or more (45%) patients undergo such conversion, the investigational treatment will be considered as promising/feasible. The resectable rate will be estimated with its 95% exact confidence interval.
Measured: 8 Cycles, 21 day cycles
Incidence of related treatment emergent adverse events
Number of participants with related treatment emergent adverse events
Measured: 36 months
Secondary outcomes (8)
Objective Response Rate (ORR)
ORR will be estimated with its 95% exact confidence interval based on RECIST v1.1
Measured: 36 months
Pathological complete response (pCR)
Measured: 36 months
Overall survival (OS)
Kaplan-Meier method is used to represent secondary outcome OS.
Measured: 36 months
Progression-free survival (PFS)
Kaplan-Meier method is used to represent secondary outcome of PFS
Measured: 36 months
Rate of R0 resection
R0 resection rate will be estimated with its 95% exact confidence interval
Measured: 8 Cycles, 21 day cycles
Relapse free survival (RFS)
Kaplan-Meier method will be used to analyze RFS
Measured: 36 months
Patient Reported outcomes (PRO) decline
As measured by qualify of life changes per EORTC QLQ-BIL-20 questionnaires. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.
Measured: 8 Cycles, 21 day cycles
Event Free Survival (EFS)
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2023
- Study startDec 2024 (estimated)
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (estimated)
Why it stopped: Sponsor decision and challenges in finding eligible participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Borderline Resectable CarcinomaStudies of the same condition, open and under way.
- More trials run by Georgetown UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Georgetown University
- Sponsor type
- University, hospital or other
- Collaborators
- AstraZeneca
- Responsible party
- Sponsor
- Investigators
- Aiwu R He, MD, Principal investigator, Georgetown University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06017297
- Sponsor's protocol ID
- STUDY00006462
- Acronym
- ESR-22-21719
- Status last verified
- Nov 2024
- First submitted
- 24 Aug 2023
- Last updated
- 12 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Neoadjuvant Durvalumab (MEDI4736) and Tremelimumab in Combination With Gemcitabine and Cisplatin in Patients With Intrahepatic Cholangiocarcinoma That is Borderline Resectable/Resectable But With High Risk for Recurrence.
- ClinicalTrials.gov ID
- NCT06017297
- Acronym / sponsor ID
- ESR-22-21719 / STUDY00006462
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor decision and challenges in finding eligible participants
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Georgetown UniversityUniversity, hospital or other
- Collaborators
- AstraZeneca
- Study start
- Dec 2024 (estimated)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 30 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to test feasibility and safety of the combination of tremelimumab and durvalumab plus gemcitabine and cisplatin as a neoadjuvant treatment bridge patients to a curative resection in treatment naïve borderline resectable, or resectable with high risk for recurrence intrahepatic cholangiocarcinoma patients. The main question[s] it aims to answer are:
- What is the rate of conversion of unresectable tumor to resectable cancer?
- What are the side effects of this treatment combination?
Participants will undergo an initial tumor biopsy, imaging and laboratory studies prior to starting treatment with durvalumab, tremelimumab, gemcitabine and cisplatin. Participants will continue for 4 cycles and if the tumor is found to be resectable then they will undergo surgical resection. If the tumor is unresectable (can't be surgically removed) after 4 cycles, then participants will receive 4 more cycles and repeated imaging. If the tumor remains unresectable then the participant will be treated with capecitabine for up to 8 cycles and durvalumab for up to 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Durvalumab and Tremelimumab plus gemcitabine/cisplatin | Experimental | Drug: Durvalumab, Drug: Tremelimumab, Drug: Gemcitabine, Drug: Cisplatin, Procedure: Surgical ResectionDurvalumab and tremelimumab plus gemcitabine/cisplatin combination therapy. If the tumor is evaluated to be resectable after Cycle 4 (C4), then the patient may proceed with surgical tumor resection. If the tumor is deemed unresectable after C4, then the patient will proceed with Cycle 5-8 followed by reevaluation for surgical resection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Patients of age 18 years and older at the time of study entry, both sexes, all ethnicities.
- Patients with histologically proven intrahepatic cholangiocarcinoma, untreated with systemic therapy.
- Patients with an absence of extrahepatic metastasis (outside of periportal lymph node enlargement) or peritoneal carcinomatosis as demonstrated by CT-scan.
- Patients with a performance status ECOG 0, 1.
- Patients with an estimated life expectancy > 6 months.
Key exclusion criteria (35 listed)
- Patients with hilar or distal cholangiocarcinoma or those with hepatocholangiocarcinoma.
- Patients who are eligible for surgical resection or liver transplantation based on tumor characteristics.
- Patients who would not be surgical candidates due to reasons unrelated to their cholangiocarcinoma, e.g. Cirrhosis with portal hypertension
- Patients with extrahepatic metastases beyond periportal lymph node enlargement
- Patients with a contraindication or grade 3-4 allergy to durvalumab, tremelimumab, gemcitabine, cisplatin, or capecitabine.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DurvalumabDurvalumab 1500 mg via intravenous (IV) infusion every 3 weeks for up to 8 cycles | Drug | Durvalumab and Tremelimumab plus gemcitabine/cisplatin |
| TremelimumabA single dose of tremelimumab at 300mg IV is given on C1. | Drug | Durvalumab and Tremelimumab plus gemcitabine/cisplatin |
| GemcitabineGemcitabine is dosed at 1000 mg/m2 IV on day (D)1 and D8 of each cycle. | Drug | Durvalumab and Tremelimumab plus gemcitabine/cisplatin |
| CisplatinCisplatin is dosed at 25mg/m2 on D1 and D8 of each cycle. | Drug | Durvalumab and Tremelimumab plus gemcitabine/cisplatin |
| Surgical ResectionIf the tumor is evaluated to be resectable (as defined as successfully treated stage II (tumor shrink away from vessels), stage IIIA (tumor shrink away from visceral peritoneum), stage IIIB (N1 disease no longer pathologically enlarged) after C4 or C8, then the patient may proceed with surgical tumor resection. | Procedure or surgery | Durvalumab and Tremelimumab plus gemcitabine/cisplatin |
Primary outcome
Rate of Conversion from unresectable to resectable
Time frame: 8 Cycles, 21 day cycles
Rate of conversion of unresectable tumor to resectable cancer after neoadjuvant durvalumab + tremelimumab + gemcitabine + cisplatin after 4 or 8 cycles. Surgical evaluation will be done in joint by institutional radiology and hepatobiliary surgery using clinical data (CT/MRI imaging, patient performance status, labs, etc.) If among the evaluable 24 patients, 9 or more (45%) patients undergo such conversion, the investigational treatment will be considered as promising/feasible. The resectable rate will be estimated with its 95% exact confidence interval.
Incidence of related treatment emergent adverse events
Time frame: 36 months
Number of participants with related treatment emergent adverse events
8 secondary outcomes
Objective Response Rate (ORR)
Time frame: 36 months
Pathological complete response (pCR)
Time frame: 36 months
Overall survival (OS)
Time frame: 36 months
Progression-free survival (PFS)
Time frame: 36 months
Rate of R0 resection
Time frame: 8 Cycles, 21 day cycles
Relapse free survival (RFS)
Time frame: 36 months
Patient Reported outcomes (PRO) decline
Time frame: 8 Cycles, 21 day cycles
Event Free Survival (EFS)
Time frame: 36 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Nov 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aiwu R He, MD principal investigator
Georgetown University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Durvalumab, Tremelimumab and Gemcitabine in people with Borderline Resectable Carcinoma and Biliary Tract Cancer, run by Georgetown University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 35 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 12 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Conversion from unresectable to resectable (time frame: 8 Cycles, 21 day cycles). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2024.