3
Indication details
- Control Arm
- Placebo
- Treatment setting
- For adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy.
- Tumour Type
-
Gastrointestinal Cancers
- Tumour Sub-type
- Cholangiocarcinoma
- Tumour Stage
- Locally advanced or metastatic
- Tumour Sub-Group
- IDH1
- Trial Name
- ClarIDHy
- NCT Number
- NCT02989857
- Trial Phase
- Phase III
Approval details
- FDA Approval
- FDA approval August 2021
- EMA Approval
- EMA (CHMP) February 2023 EC decision May 2023
Primary Outcome(s)
- Primary Outcome(s)
- PFS
- Evaluated Outcome
- PFS
- Form(s)
- Form 2b
Outcome Data
- PFS Control
- 1.4 months
- PFS Gain
- 1.3 months, >10% plateau in the tail of the PFS curve
- PFS HR
- 0.37 (0.25-0.54)
Adjustments
- QoL Comment
-
No QoL benefit
Final Score (after adjustments)
- Preliminary non-curative score
-
2
- Long-term plateau in the PFS curve
- 1+
- Final non-curative score
-
3
- Comment
-
If mature non-significant OS is subject to major crossover, it is not taken into account for any adjustment. The study incorporated undesirable crossover undermining the veracity of the overall survival benefit evaluation.
EMA (CHMP) February 2023 EC decision May 2023
FDA approval August 2021
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 297
- Scorecard version
- 1
- Issue date
- 23.09.2021
- Last update
- 12.05.2023
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.