3
Indication details
- Control Arm
- Single arm
- Therapeutic Indication
- As monotherapy for the treatment of adult and adolescents ≥12 years of age with advanced RET-mutant medullary thyroid cancer (MTC)
- Tumour Type
-
Endocrine Tumours
- Tumour Sub-type
- Thyroid Cancer - medullary
- Tumour Stage
- Advanced or metastatic
- Tumour Sub-Group
- RET-mutant
- Trial Name
- LIBRETTO-001 (Cohort 4)
- NCT Number
- NCT03157128
- Trial Phase
- Phase I/II
Approval details
- FDA Approval
- FDA approval May 2020
- EMA Approval
- EMA (CHMP) December 2020 EC decision February 2021 EMA (CHMP) extension of indication July 2022 EC decision September 2022
Primary Outcome(s)
- Primary Outcome(s)
- ORR
- Evaluated Outcome
- ORR, DOR
- Form(s)
- Form 3
Outcome Data
- PFS Control
- 23.6 months
- ORR
- 73%
- DoR
- 22.0 months
Adjustments
- QoL Comment
-
QoL was an exploratory endpoint
Score (after adjustments)
- Preliminary non-curative score
-
3
- Non-curative score
-
3
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 227
- Scorecard version
- 1
- Issue date
- 4.11.2020
- Last update
- 21.07.2023
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.