ALS experts share their experiences with RADICAVA^®

This video is sponsored by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The speaker is presenting on behalf of, and has received compensation from MTPA.

Radicava ORS^® Treatment Initiation Checklist

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The ALSFRS-R and ALS Progression

Learn about the ALS Functional Rating Scale-Revised (ALSFRS-R) for evaluating the decline in physical function over time.

THE DIFFERENCE IS EXPERIENCE

RADICAVA^® (edaravone) has been evaluated in over two decades of clinical research, including four phase 3 trials^2-5

REVIEW RADICAVA ORS^® CLINICAL HISTORY

The ALSFRS-R is considered the gold standard used to measure ALS disease progression and trial outcomes⁶

The ALSFRS-R is a validated, questionnaire-based tool for evaluating decline in physical function over time.^7,8

The ALSFRS-R consists of 12 items in 4 domains.
Each item is scored between 4 and 0, for a maximum score of 48.^9,10

Tap on each item below to see the point descriptions.

Bulbar

Speech

Salivation

Swallowing

SPEECH:

Normal
Detectable speech disturbance
Intelligible with repeating
Speech combined with nonvocal communication
Loss of useful speech

SALIVATION:

Normal
Slight but definite excess of saliva in mouth; may have nighttime drooling
Moderately excessive saliva; may have minimal drooling
Marked excess of saliva with some drooling
Marked drooling; requires constant tissue or handkerchief

SWALLOWING:

Normal
Early eating problems—occasional choking
Dietary consistency changes
Needs supplemental tube feeding
NPO (exclusively parenteral or enteral feeding)

Fine Motor

Handwriting

Cutting Food (Patients without gastrostomy)

Cutting Food (Patients with gastrostomy)

Dressing and Hygiene

Handwriting:

Normal
Slow or sloppy; all words are legible
Not all words are legible
Able to grip pen but unable to write
Unable to grip pen

CUTTING FOOD:
(without gastrostomy)

Normal
Somewhat slow and clumsy, but no help needed
Can cut most foods, although clumsy and slow; some help needed
Food must be cut by someone, but can still feed slowly
Needs to be fed

CUTTING FOOD:
(with gastrostomy)

Normal
Clumsy, but no help needed
Some help needed with closures and fasteners
Provides minimal assistance to caregiver
Unable to perform any aspect of task

Dressing and Hygiene:

Normal
Independent and complete self-care with effort or decreased efficiency
Intermittent assistance or substitute methods
Needs attendant for self-care
Total dependence

Gross Motor

Turning in Bed

Walking

Climbing Stairs

TURNING IN BED:

Normal
Somewhat slow and clumsy, but no help needed
Can turn alone or adjust sheets, but with great difficulty
Can initiate, but not turn or adjust sheets alone
Helpless

WALKING:

Normal
Early ambulation difficulties
Walks with assistance
Non-ambulatory functional movement only
No purposeful leg movement

CLIMBING STAIRS:

Normal
Slow
Mild unsteadiness or fatigue
Needs assistance
Cannot do

Respiratory

Dyspnea

Orthopnea

Respiratory Insufficiency

DYSPNEA:

Normal
Occurs when walking
Occurs with one or more of the following: eating, bathing, dressing (ADL)
Occurs at rest, difficulty breathing when either sitting or lying
Significant difficulty, considering using mechanical respiratory support

ORTHOPNEA:

Normal
Some difficulty sleeping at night due to shortness of breath. Does not routinely use more than two pillows
Needs extra pillow in order to sleep (more than two)
Can only sleep sitting up
Unable to sleep

Respiratory Insufficiency:

Normal
Intermittent use of BiPAP
Continuous use of BiPAP during the night
Continuous use of BiPAP during the night and day
Invasive mechanical ventilation by intubation or tracheostomy

The ALSFRS-R was used to assess the primary endpoint in the pivotal phase 3 clinical trial for RADICAVA^® IV.^{11,a (see footnote)}

ALSFRS-R=ALS Functional Rating Scale-Revised; BiPAP=bilevel positive airway pressure.

^aMean change in ALSFRS-R at 24 weeks.¹² Return to content

See how RADICAVA^® was shown to slow the loss of physical function in patients with ALS.

LEARN MORE

ALSFRS-R—An Important Tool for Assessing ALS Progression⁶

Based on data from the PRO-ACT database, the average rate of ALSFRS-R decline is 1.02 points/month.¹³

Due to the heterogeneity of ALS, individual decline on the ALSFRS-R may vary widely among patients, with some patients losing 0 to 1 point/month and others losing up to 6 points/month.¹⁴

A report on a survey of 65 members of Northeast ALS Consortium (NEALS) concluded that a treatment is clinically meaningful when it results in 20% to 25% or greater change in the slope of ALSFRS-R reflecting the slowing of functional decline.¹⁰

In the evaluation of physical decline, the higher the score, the greater the physical function.^8,9

FDA=Food and Drug Administration; IV=intravenous; PRO-ACT=Pooled Resource Open-Access ALS Clinical Trials.

References: 1. US Food and Drug Administration. Clinical superiority findings. Accessed April 11, 2024. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/clinical-superiority-findings 2. Yoshino H, Kimura A. Investigation of the therapeutic effects of edaravone, a free radical scavenger, on amyotrophic lateral sclerosis (phase II study). Amyotroph Lateral Scler. 2006;7(4):241-245. 3. ClinicalTrials.gov. Safety study of oral edaravone administered in subjects with ALS. Accessed April 11, 2024. https://clinicaltrials.gov/study/NCT04165824 4. Brooks BR, Heiman-Patterson T, Wiedau-Pazos M, et al. Edaravone efficacy in amyotrophic lateral sclerosis with reduced forced vital capacity: post-hoc analysis of Study 19 (MCI186-19) [clinical trial NCT01492686]. PLoS One. 2022;17(6):e0258614. 5. Takei K, Watanabe K, Yuki S, et al. Edaravone and its clinical development for amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18(suppl 1):5-10. 6. Eisen A. Motor neurone disease. In: Turner MR, Kiernan MC, eds. Landmark Papers in Neurology. Oxford University Press; 2015:257-288. 7. Leigh PN, Swash M, Iwasaki Y, et al. Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5(2):84-98. 8. Cedarbaum JM, Stambler N, Malta E, et al; BDNF ALS Study Group (Phase III). The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. J Neurol Sci. 1999;169(1-2):13-21. 9. The ALS C.A.R.E. Program. Center for Outcomes Research, University of Massachusetts Medical School. ALS functional rating scale. Accessed January 6, 2022. http://www.outcomes-umassmed.org/ALS/alsscale.aspx. 10. Castrillo-Viguera C, Grasso DL, Simpson E, et al. Clinical significance in the change of decline in ALSFRS-R. Amyotroph Lateral Scler. 2010;11(1-2):178-180. 11. Mitsumoto H, Brooks BR, Silani V. Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved? Lancet Neurol. 2014;13:1127-1138. 12. Su XW, Broach JR, Connor JR, et al. Genetic heterogeneity of amyotrophic lateral sclerosis: implications for clinical practice and research. Muscle Nerve. 2014;49:786-803. 13. Atassi N, Berry J, Shui A, et al. The PRO-ACT database: design, initial analyses, and predictive futures. Neurology. 2014;83:1719-1725. 14. Proudfoot M, Jones A, Talbot K, et al. The ALSFRS as an outcome measure in therapeutic trials and its relationship to symptom onset. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17(5-6):414-425.

The ALSFRS-R and ALS Progression

The ALSFRS-R is considered the gold standard used to measure ALS disease progression and trial outcomes⁶