4
Indication details
- Control Arm
- Cabozantinib or Vandetanib
- FDA Therapeutic Indication
- Selpercatinib for adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy.
- Tumour Type
-
Endocrine Tumours
- Tumour Sub-type
- Thyroid cancer - Medullary
- Tumour Stage
- Advanced or metastatic
- Tumour Sub-Group
- RET-mutant (M918T or other)
- Trial Name
- LIBRETTO-531
- NCT Number
- NCT04211337
- Trial Phase
- Phase III
Approval details
- FDA Approval
- FDA approval September 2024
Primary Outcome(s)
- Primary Outcome(s)
- PFS. OS as secondary endpoint (crossover allowed)
- Evaluated Outcome
- PFS
- Form(s)
- Form 2b
Outcome Data
- PFS Control
- 16.8 months
- PFS Gain
- 43.2 months (estimated)
- PFS HR
- 0.28 (0.16 - 0.48) P<0.001
- OS HR
- Immature
Adjustments
- QoL Comment
-
QoL data pending
Score (after adjustments)
- Preliminary non-curative score
-
3
- Long-term plateau in the PFS curve
- 1+
- Non-curative score
-
4
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 469
- Scorecard version
- 1
- Issue date
- 08.11.2024
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.