3
Indication details
- Control Arm
- Single arm (Phase I/II)
- FDA Therapeutic Indication
- EMA/FDA: Treatment for adult patients with locally advanced or metastatic solid tumors with a rearranged during transfection (RET) gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options (other than non-small cell lung cancer and thyroid cancer).
- Tumour Stage
- Locally advanced or metastatic
- Trial Name
- LIBRETTO-001
- NCT Number
- NCT03157128
- Trial Phase
- Phase I/II
Approval details
- FDA Approval
- FDA accelerated approval September 2022
- EMA Approval
- EMA (CHMP) March 2024. Pending EC decision (recommendations on extensions)
Primary Outcome(s)
- Primary Outcome(s)
- ORR
- Evaluated Outcome
- ORR Overall study population
- Form(s)
- Form 3
Outcome Data
- PFS Control
- 13.2 months
- ORR
- 43.9%
- DoR
- 24.5 months
Adjustments
- QoL Comment
-
QoL was not a prespecified endpoint
- Toxicity Comment
-
14/45 patients (31%) experience grade 3-5 AEs impacting on daily well being
Score (after adjustments)
- Preliminary non-curative score
-
3
- Non-curative score
-
3
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 357
- Scorecard version
- 1
- Issue date
- 14.11.2022
- Last update
- 26.03.2024
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.