3
Indication details
- Control Arm
- Single arm (Phase I/II)
- FDA Therapeutic Indication
- Adult and paediatric patients with solid tumours that display an NTRK gene fusion, who have disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options
- Tumour Type
-
Tumour agnostic
- Tumour Stage
- Locally advanced or metastatic
- Tumour Sub-Group
- NTRK gene fusion
- Trial Name
- SCOUT and NAVIGATE
- NCT Number
- NCT02122913, NCT02637687 NCT02576431
- Trial Phase
- Phase I/II
Approval details
- FDA Approval
- FDA approval November 2018
- EMA Approval
- EMA (CHMP) July 2019 EC decision September 2019
Primary Outcome(s)
- Primary Outcome(s)
- ORR
- Evaluated Outcome
- ORR Overall population (pooled analysis)
- Form(s)
- Form 3
Outcome Data
- PFS Control
- 28.3 months
- RR Gain
- 79%
- DoR
- 35.2 months
Adjustments
- QoL Comment
-
QoL was not a prespecified endpoint
Score (after adjustments)
- Preliminary non-curative score
-
3
- Non-curative score
-
3
- Comment
-
EMA/FDA approvals were based on an initial cohort of 55 patients (with median DoR/PFS not reached). The presented results refer to an extended cohort of 159 patients (with available medians for DoR/PFS). For both cohorts, ORR was >60% (initial: 75%; extended: 79%), resulting in an MCBS score of 3, according to Form 3 (irrespective of median DoR/PFS).
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 143
- Scorecard version
- 1
- Issue date
- 20.12.2019
- Last update
- 22.10.2021
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.