Efficacy
Explore the pivotal clinical trial for
Study Design
The study design of the pivotal clinical trial evaluated RADICAVA® vs placebo over 24 weeks1
Pivotal Study Design1,2
Patients who completed the observation period were randomized to
In the pivotal clinical trial for
most patients in the
(n=63)
(n=62)
Inclusion criteria of the pivotal clinical trial
At study entry1,3 |
---|
Definite or Probable ALS (El Escorial revised and Airlie House diagnostic criteria) |
Scores ≥2 on all items of the ALSFRS-R |
Grade 1 or 2 Japan ALS severity classificationa |
Normal respiratory function (≥80% FVC) |
Disease duration ≤2 years |
Age 20-75 |
At randomization3 |
---|
Deterioration in ALSFRS-R score during 12-week pre-study observation period of 1 to 4 points |
The inclusion criteria4:
- Used a defined population based on an analysis of an earlier trial
- Identified patients who would be expected to have a sufficient and measurable deterioration over 24 weeks in order to see the effect of treatment
aJapan ALS severity classification grades, which were used for the pivotal clinical trial (J19), range from 1 to 5 as follows5:
- Able to work or perform housework
- Independent living but unable to work
- Requiring assistance for eating, excretion, or ambulation
- Presence of respiratory insufficiency, difficulty in coughing out sputum or dysphagia
- Using a tracheostomy tube, tube feeding, or tracheostomy positive-pressure ventilation
FVC=forced vital capacity.
The enrichment strategy for Study 19 resulted in patients with relatively good function at baseline and adequate disease progression on the ALSFRS-R scale.
Slowing Loss of Physical Function
In the pivotal clinical trial, RADICAVA® demonstrated 33% less change in ALSFRS-R scores from baseline vs placebo at 24 weeks1
A report concerning a survey of 65 members of the NEALS Consortium concluded that a treatment that resulted in a 20%-25% or greater change in the slope of the ALSFRS-R is clinically meaningful.6
Physical Function in Daily Activities at 24 Weeks1
Change from Baseline
LS Mean ± SE
In the pivotal clinical trial, there was
33%
less change in ALSFRS-R scores from baseline vs placebo at 24 weeks.1
In the pivotal clinical trial, RADICAVA® patients declined slower vs placebo patients2
Physical Function in Daily Activities at 24 Weeks2
Mean change in ALSFRS-R Score (LOCF)
LS Mean ± SE
In the pivotal clinical trial for
most patients in the
(n=63)
(n=62)