A
Indication details
- Control Arm
- Popensity score matched external control from ANBL0032. Matched cohort 3:1
- Therapeutic Indication
- Eflornithine to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. HRNB postimmunotherapy maintenance
- Tumour Type
-
Brain Tumours
- Tumour Sub-type
- Neuroblastoma
- Tumour Stage
- Adjuvant
- Trial Name
- Study 3b, NMTRC003B - ANBL0032
- NCT Number
- NCT02395666
- Trial Phase
- Phase II comparative study
Approval details
- FDA Approval
- FDA approval December 2023
Primary Outcome(s)
- Primary Outcome(s)
- EFS, as secondary endpoint OS
- Evaluated Outcome
- EFS
- Form(s)
- Form 1
Outcome Data
- OS Control
- 4 year OS 84%
- OS Gain
- 4 year OS gain 12%
- OS HR
- 0.32 (0.15-0-70)
- DFS Control
- 4 year EFS 73%
- DFS Gain
- 4 year EFS gain 11%
- DFS HR
- 0.48 (0.27- 0.85)
Adjustments
- QoL Comment
-
Not qualified for an ESMO-MCBS credit
Score (after adjustments)
- Curative score
-
A
- Comment
-
Scored on the same basis as a comparative study, using the propensity matched external control as if it were a randomized control arm.
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 424
- Scorecard version
- 1
- Issue date
- 01.02.2024
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.