Trastuzumab Plus Chemotherapy vs Chemotherapy Alone in First-line HER2 Positive Advanced Biliary Tract Cancer Patients

NCT ID: NCT07062263

Last Updated: 2025-07-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

220 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-21

Study Completion Date

2029-07-31

Brief Summary

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This is a randomized, open-label, two-arm, Phase III clinical trial evaluating the efficacy and safety of trastuzumab plus chemotherapy versus chemotherapy alone as first-line treatment in patients with HER2-positive advanced or metastatic biliary tract cancers (BTC). HER2-positive BTCs represent a molecular subset of these rare cancers, associated with poor prognosis and limited treatment options.

Eligible patients with histologically confirmed HER2-positive (IHC 3+ or IHC 2+ with FISH amplification) unresectable or metastatic biliary tract adenocarcinoma-including gallbladder cancer, intrahepatic, and perihilar cholangiocarcinoma-will be randomized in a 1:1 ratio. Participants in the intervention arm (Arm A) will receive either gemcitabine and cisplatin with or without nab-paclitaxel plus trastuzumab, while those in the control arm (Arm B) will receive chemotherapy alone (gemcitabine + cisplatin with or without nab-paclitaxel). Treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, or death.

The primary endpoint is 6-month progression-free survival (PFS). Secondary endpoints include overall survival (OS), response rate (RR), quality of life (QOL), and adverse event (AE) profiles. The study aims to enroll 196 patients across a single center in India over a period of 5 years, with an additional 6-month follow-up. This trial builds on earlier Phase II findings suggesting improved outcomes with trastuzumab in HER2-positive BTC and aims to provide the first randomized evidence for the benefit of HER2-targeted therapy in this setting.

Detailed Description

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Title of study- Trastuzumab plus chemotherapy vs Chemotherapy alone in first-line HER2 positive advanced biliary tract cancer patients - a randomized non-blinded two-arm Phase III prospective clinical trial (TAB - 2 study)

Indication- HER2-positive advanced/metastatic biliary tract cancers fit for first-line chemotherapy with gemcitabine-based combination

Type of Study- Two arm open-label prospective Phase III parallel design randomized superiority clinical trial

Biliary tract cancers (BTC), including gallbladder cancer and cholangiocarcinomas, are uncommon but aggressive tumors with poor prognosis in the advanced or metastatic setting. The current standard first-line treatment is gemcitabine combined with a platinum agent, which offers limited benefit. Emerging molecular profiling has identified HER2 overexpression or amplification in a subset of BTCs, particularly in gallbladder cancer, and has opened new avenues for targeted therapy.

Trastuzumab, a monoclonal antibody targeting the HER2 receptor, has shown clinical activity in HER2-positive BTCs in early-phase studies. Preliminary evidence from a single-arm Phase II study (TAB trial) suggests that combining trastuzumab with chemotherapy may significantly improve progression-free survival in treatment-naïve HER2-positive patients.

The TAB-2 study is a prospective, randomized, open-label Phase III trial designed to evaluate whether adding trastuzumab to first-line chemotherapy improves clinical outcomes in patients with HER2-positive advanced BTC. Patients will be randomized to receive chemotherapy with or without trastuzumab. Chemotherapy regimens may include gemcitabine and cisplatin, with or without nab-paclitaxel, based on physician discretion. Trastuzumab will be administered every three weeks along with chemotherapy until disease progression, unacceptable toxicity, or withdrawal of consent.

The study will be conducted at a single academic center in India, with a planned enrollment of 196 participants over 5 years. This trial aims to provide definitive randomized evidence regarding the benefit of incorporating HER2-targeted therapy into the first-line treatment of advanced BTC and to inform future clinical practice for this rare molecular subtype.

Primary endpoint- To evaluate the difference in progression-free survival (PFS) at 6 months between Trastuzumab - chemotherapy combination and chemotherapy alone.

Treatment scheme

1. Arm A (Trastuzumab plus chemotherapy)-Gemcitabine plus Trastuzumab OR Gemcitabine Plus Cisplatin Plus Nab-paclitaxel on D1 and D8 Plus Trastuzumab D1 Start of next cycle on D 22
2. Arm B (Chemotherapy alone)- Gemcitabine Plus Cisplatin D1 and D8 q 21 days OR Gemcitabine Plus Cisplatin Plus Nab-paclitaxel D1 and D8) Start of next cycle on D 22 Treatment in both arms will be continued till unacceptable toxicity, death, or consent withdrawal.

In both arms Immunotherapy is allowed as per the discretion of the treating physician

Sample Size calculation- Based published phase 2 single-arm TAB trial, the median PFS in HER2 positive biliary tract cancer with Trastuzumab plus chemotherapy was approximately 8 months (6-month PFS was approximately 75 - 80%), while the median PFS with chemotherapy alone ranges from 5-6 months (6-month PFS approximately 55%-65%). Assuming the Trastuzumab combination will increase the 6-month PFS to 75% in comparison to 55% with chemotherapy alone, with a power of 80% and alpha of 0.05 and margin of risk difference of 3 %, a Phase III randomized study will require a total of 196 patients (98 per arm), assuming an attrition rate of 5 % per arm with a study accrual period of 5 years. Follow up the duration of the study will be 6 months post-accrual of the last patient.

Conditions

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Biliary Tract Cancer HER2-positive Cancer Advanced Cancer Unresectable Biliary Tract Carcinoma Metastatic Biliary Tract Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Phase III open-label parallel design randomized clinical trial
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm A: Trastuzumab plus Chemotherapy

Trastuzumab + Gemcitabine + cisplatin

* Gemcitabine 1000 mg/m2 IV over 30 mins on day 1 and day 8 q 3 weekly
* Cisplatin 25 mg/m2 IV over 60 minutes on day 1 and day 8 q 3 weekly
* Trastuzumab 8mg/kg IV over 90 mins as first dose and subsequent doses at 6mg/kg IV over 30-60 minutes q 3 weekly (=1 cycle)

OR

* Trastuzumab with Gemcitabine-cisplatin-Nab-Paclitaxel
* Gemcitabine 800 mg/m2 IV over 30 mins on day 1 and day 8 q 3 weekly
* Nab-paclitaxel 100 mg/m2 IV over 1-2 hours on day 1 and day 8 q 3 weekly
* Cisplatin 25 mg/m2 IV over 60 minutes on day 1 and day 8 q 3 weekly
* Trastuzumab 8mg/kg IV over 90 mins as first dose and subsequent doses at 6mg/kg IV over 30-60 minutes q 3 weekly (=1 cycle) Start of next cycle on D22 Immunotherapy is allowed as per the discretion of the treating physician

Group Type EXPERIMENTAL

Trastuzumab

Intervention Type DRUG

Inj Trastuzumab given in a dose of 8mg/kg intravenously over 90 mins as first dose and subsequent doses at a dose of 6mg/kg intravenously over 30-60 minutes in 3 weekly cycle along with standard of care chemotherapy

Chemotherapy

Intervention Type DRUG

Gemcitabine + cisplatin OR Gemcitabine-cisplatin-Nab-Paclitaxel given as a standard of care, chemotherapy as per the institutional guidlines

Arm B: Chemotherapy alone

Gemcitabine + cisplatin

* Gemcitabine 1000 mg/m2 IV over 30 mins on day 1 and day 8 q 3 weekly
* Cisplatin 25 mg/m2 IV over 60 minutes on day 1 and day 8 q 3 weekly (=1 cycle)

OR

* Gemcitabine-cisplatin-Nab-Paclitaxel
* Gemcitabine 800 mg/m2 IV over 30 mins on day 1 and day 8 q 3 weekly
* Nab-paclitaxel 100 mg/m2 IV over 1-2 hours on day 1 and day 8 q 3 weekly =Cisplatin 25 mg/m2 IV over 60 minutes on day 1 and day 8 q 3 weekly (=1 cycle) Start of next cycle on D22 Immunotherapy is allowed as per the discretion of the treating physician

Group Type ACTIVE_COMPARATOR

Chemotherapy

Intervention Type DRUG

Gemcitabine + cisplatin OR Gemcitabine-cisplatin-Nab-Paclitaxel given as a standard of care, chemotherapy as per the institutional guidlines

Interventions

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Trastuzumab

Inj Trastuzumab given in a dose of 8mg/kg intravenously over 90 mins as first dose and subsequent doses at a dose of 6mg/kg intravenously over 30-60 minutes in 3 weekly cycle along with standard of care chemotherapy

Intervention Type DRUG

Chemotherapy

Gemcitabine + cisplatin OR Gemcitabine-cisplatin-Nab-Paclitaxel given as a standard of care, chemotherapy as per the institutional guidlines

Intervention Type DRUG

Other Intervention Names

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Transtuzumab with standand of chemotherapy Standard of chemotherapy Gemcitabine cisplatin Nab-Paclitaxel

Eligibility Criteria

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Inclusion Criteria

Histologically confirmed adenocarcinoma of the biliary tract, with the following specifications -

1. Biliary tract cancers include gallbladder cancer, intrahepatic cholangiocarcinoma, and perihilar cholangiocarcinoma.
2. HER2-positive by IHC or FISH
3. Age \>=18 years.
4. ECOG performance status 0 - 2.
5. Unresectable or metastatic cancer.
6. Patient does not have any contraindications to receive chemotherapy or trastuzumab.
7. Adequate hematological, hepatic, and renal function parameters- Hematological- Hb\> 80 g/L, ANC ≥ 1.5 x 109/L, platelets ≥ 100 x 109/L. Liver functions- bilirubin ≤ 2 x upper limit normal (ULN), AST/ALT ≤ 5 x ULN, alkaline phosphatase ≤ 6 x upper limit normal (ULN) S. albumin ≥ 30 g/L.

Renal function- Creatinine ≤ 1.5 ULN, Creatinine clearance \>= 30 mL/min.
8. Normal cardiac ejection fraction and cardiac function, as assessed by echocardiography, ejection fraction (EF) \>=50% or above the lower limit of normal. ECG with no clinically relevant abnormalities.
9. Women of childbearing age should have a negative pregnancy test at the time of randomization and should be willing to use adequate contraception during the treatment phase of the trial.
10. Subjects must provide written informed consent prior to the performance of study-specific procedures or assessments, and must be willing to comply with treatment and follow-up assessments and procedures.
11. Subjects who have received adjuvant chemotherapy will be considered eligible provided that therapy is completed more than 12 months before study enrollment. Patients who have received radiation therapy and surgery will also be eligible provided the interventions have been completed 3 and 2 weeks, respectively, before enrolment in the study.
12. Negative serum pregnancy test (if applicable) and willing for adequate contraception.
13. At least one measurable disease according to RECIST criteria.
14. Life expectancy of at least 12 weeks.

Exclusion Criteria

1. Distal cholangiocarcinoma
2. Known hypersensitivity or contraindications against gemcitabine, cisplatin, Nab- paclitaxel, or trastuzumab.
3. Clinically significant active coronary heart disease, cardiomyopathy, or congestive heart failure, NYHA III-IV.
4. Clinically significant valvular defect.
5. Past or current history of other malignancies not curatively treated and without evidence of disease for more than 5 years, except for curatively treated basal cell carcinoma of the skin and in situ carcinoma of the cervix.
6. Severe dyspnea at rest due to complications of advanced malignancy or requiring supplementary oxygen therapy.
7. Baseline neuropathy \> NCI Grade I.
8. Subject pregnant or breastfeeding, or planning to become pregnant within 6 months after the end of treatment.
9. Received prior chemotherapy within 1 year.
10. Any active ILD/ history of lung illness requiring bronchodilator drugs.
11. Patients with prior chemotherapy for metastatic disease will be ineligible for enrollment in the study.
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tata Memorial Centre

OTHER

Sponsor Role lead

Responsible Party

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Dr. Vikas Ostwal

Professor Dept of Medical Oncology, Convener Gastro-Intestinal Disease Management Group

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Vikas Ostwal, DM

Role: PRINCIPAL_INVESTIGATOR

Tata Memorial Hospital, Parel, Mumbai, Maharashtra, India 400012

Locations

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Homi Bhabha Cancer Hospital and Research Centre, Muzaffarpur

Muzaffarpur, Bihar, India

Site Status NOT_YET_RECRUITING

Tata Memorial Hospital

Mumbai, Maharashtra, India

Site Status RECRUITING

Institute of Medical Sciences & SUM Hospital

Bhubaneswar, Odisha, India

Site Status NOT_YET_RECRUITING

Homi Bhabha Cancer Hospital and Research Centre

Mūllānpur, Punjab, India

Site Status RECRUITING

Mahamana Pandit Madan Mohan Malviya Cancer Centre (MPMMC) and Homi Bhabha Cancer Hospital (HBCH)

Varanasi, Uttar Pradesh, India

Site Status RECRUITING

MAX Super Speciality Hospital, SAKET

Delhi, , India

Site Status NOT_YET_RECRUITING

Countries

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India

Central Contacts

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Vikas S Ostwal, DM

Role: CONTACT

0222417000 ext. 6323

Anant Ramaswamy, DM

Role: CONTACT

0222417000 ext. 6323

Facility Contacts

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Dr Gaurav Kumar, MD

Role: primary

0621-2232032 ext. 122

Vikas Ostwal, DM

Role: primary

0222417000 ext. 6323

Anant Ramaswamy, DM

Role: backup

0222417000 ext. 6323

Soumya S Panda, DM

Role: primary

8895370579

Pragyat Thakur

Role: primary

+910160 281 0022

Dr Bal Krishna Mishra, MD

Role: primary

9415214254

Dr Atul Sharma, DM

Role: primary

9958048055

Other Identifiers

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4172

Identifier Type: -

Identifier Source: org_study_id

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