4
Indication details
- Combined Agent(s)
- Gemcitabine and cisplatin
- Control Arm
- Placebo + gemcitabine and cisplatin
- Therapeutic Indication
- In combination with gemcitabine and cisplatin is indicated for the first‑line treatment of adults with unresectable or metastatic biliary tract cancer (BTC).
- Tumour Type
-
Gastrointestinal Cancers
- Tumour Sub-type
- Biliary tract cancer
- Tumour Stage
- Unresectable or metastatic
- Trial Name
- TOPAZ-1
- NCT Number
- NCT03875235
- Trial Phase
- Phase III
Approval details
- FDA Approval
- FDA approval September 2022
- EMA Approval
- EMA (CHMP) approved November 2022 pending EC decision
- Comment
- FDA approval for first-line treatment of adults with unresectable or metastatic biliary tract cancer (BTC).
Primary Outcome(s)
- Primary Outcome(s)
- OS
- Evaluated Outcome
- OS
- Form(s)
- Form 2a
Outcome Data
- PFS Control
- 5.7 months
- PFS Gain
- 1.5 months
- PFS HR
- 0.75 (0.63-0.89)
- OS Control
- 11.5 months
- OS Gain
- 1.3 months (14.5% 2-year OS gain) The 2-year OS gain reported was based on 9 patients (2.6% of the experimental group) who were still alive at that time.
- OS HR
- 0.80 (0.66-0.97)
Adjustments
- QoL Comment
-
QoL data pending
Final Score (after adjustments)
- Preliminary non-curative score
-
4
- Final non-curative score
-
4
- Comment
-
EMA (CHMP) approved November 2022 pending EC decision
FDA approval September 2022 for first-line treatment of adults with unresectable or metastatic biliary tract cancer (BTC).
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 350
- Scorecard version
- 1
- Issue date
- 22.09.2022
- Last update
- 18.01.2024
Legend
Information about symbols, abbrevations and color codes
Red = negative
Green = positive
Grey = neutral
?
Grey + ? = neutral with pending data
- DFS
- Disease-Free Survival
- DoR
- Duration of Response
- EFS
- Event-Free Survival
- HR
- Hazard Ratio
- NEB
- No evaluable benefit
- NI
- Non-inferiority Study
- ORR
- Overall Response Rate
- OS
- Overall Surival
- pCR
- Pathological Complete Response/Remission
- PFS
- Progression-Free Survival
- QoL
- Quality of Life
- RFS
- Relapse-Free Survival
- RR
- Response Rate
QoL adjustments
Other adjustments*
Serious and disabling adverse effects
Red = negative / deterioration
Green = positive / improvement
QoL adjustments
Red = negative / deterioration
Green = positive / improvement
Other adjustments*
Red = negative / deterioration
Green = positive / improvement
Serious and disabling adverse effects
Red = negative / deterioration
Green = positive / improvement
* Other adjustments include:
- Long-term plateau in the survival curve
- Long-term plateau in the PFS curve
- Early stopping or crossover
- Only improved PFS mature data shows no OS advantage and no improved Qol.