2
Indication details
- Control Arm
- Placebo
- Therapeutic Indication
- 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
- PFS HR
- 0.37 (0.25-0.54)
- OS Control
- 7.5 months
- OS Gain
- 2.8 months
- OS HR
- 0.79 (0.56-1.12) OS NS (mature)
Adjustments
- QoL Comment
-
No QoL benefit
Score (after adjustments)
- Preliminary non-curative score
-
2
- Non-curative score
-
2
- Comment
-
Mature OS is subject to major crossover; therefore, it is not taken into account for any score adjustments. The study incorporated undesirable crossover undermining the veracity of the overall survival benefit evaluation. Crossover-adjusted estimated OS gain is 5.2 months, HR 0.49 (0.34-0.70).
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 297
- Scorecard version
- 1
- Issue date
- 23.09.2021
- 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.