4
Indication details
- Control Arm
- Placebo
- FDA Therapeutic Indication
- Nirogacestat for adult patients with progressing desmoid tumours who require systemic treatment.
- Tumour Type
-
Non-cancerous soft-tissue tumour
- Tumour Sub-type
- Desmoid (fibrous tissue)
- Tumour Stage
- Progressing not amenable to surgery
- Trial Name
- DeFi
- NCT Number
- NCT03785964
- Trial Phase
- Phase III
Approval details
- FDA Approval
- FDA approval November 2023
Primary Outcome(s)
- Primary Outcome(s)
- PFS
- Evaluated Outcome
- PFS
- Form(s)
- Form 2b
Outcome Data
- PFS Control
- 15.1 months
- PFS Gain
- 37.0 months (Calculated estimate of gain based on the PE HR 0.29)
- PFS HR
- 0.29 (0.15 - 0.55)
Adjustments
- QoL Comment
-
Improved QoL
- Toxicity Comment
-
20% vs 1% discontinued due to adverse effects
Score (after adjustments)
- Preliminary non-curative score
-
3
- QoL adjustment
- +1
- Non-curative score
-
4
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 417
- Scorecard version
- 1
- Issue date
- 16.01.2024
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.