A novel muscle cramp scale (MCS) in amyotrophic lateral sclerosis (ALS)

Mitsumoto, Hiroshi; Chiuzan, Codruța; Gilmore, Madison; Zhang, Yuan; Ibagon, Camila; McHale, Brittany; Hupf, Jonathan; Oskarsson, Björn

doi:10.1080/21678421.2019.1603310

Cited by 9 publications

(14 citation statements)

References 20 publications

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“…No PROMs met all of the predefined criteria (23)(24)(25)(26)(27)(28)(29)(30)(31). As listed in Table 1, a few PROMs had medium-to highpriority rankings (23)(24)(25)(26)(27)(28)(29)(30)(31), and there were several PROMs that contained questions with the potential to be adapted and evaluated for skeletal muscle cramping in dialysis.…”

Section: Resultsmentioning

confidence: 99%

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis

Richardson

Grandinetti²,

Hilliard-Boone

et al. 2022

CJASN

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Skeletal muscle cramping is a common and bothersome symptom for patients on maintenance dialysis therapy, regardless of modality, and it has not been prioritized for innovative assessments or treatments. Research to prevent or treat skeletal muscle cramping in patients receiving dialysis is hindered by poorly understood pathophysiology, lack of an accepted definition, and the absence of a standardized measurement method. The Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a multidisciplinary workgroup to define a set of patient-reported outcome measures for use in clinical trials to test the effect of new dialysis devices, new KRTs, lifestyle/behavioral modifications, and medications on skeletal muscle cramping. Upon determining that foundational work was necessary, the workgroup undertook a multistep process to elicit concepts central to developing the basis for demonstrating content validity of candidate patient-reported outcome measures for skeletal muscle cramping in patients on dialysis. The workgroup sought to (1) create an accepted, patient-endorsed definition for skeletal muscle cramping that applies to all dialysis modalities, (2) construct a conceptual model for developing and evaluating a skeletal muscle cramping–specific patient-reported outcome measure, and (3) identify potential questions from existing patient-reported outcome measures that could be modified or adapted and subsequently tested in the dialysis population. We report the results of the workgroup efforts, provide our recommendations, and issue a call to action to address the gaps in knowledge and research needs we identified. These action steps are urgently needed to quantify skeletal muscle cramping burden, assess the effect, and measure meaningful changes of new interventions to improve the experience of patients receiving dialysis and suffering from skeletal muscle cramping.

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Section: Resultsmentioning

confidence: 99%

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis

Richardson

Grandinetti²,

Hilliard-Boone

et al. 2022

CJASN

View full text Add to dashboard Cite

show abstract

“…Our research team at Columbia University developed the MCS (40). The Columbia MCS consists of 5 items and takes only a few minutes to complete and can be accurately and effectively administered in person or by telephone.…”

Section: Outcome Measures Columbia Muscle Cramp Scale (Mcs)mentioning

confidence: 99%

“…One major advantage of the MCS over the cramp diary is that it is administered by an evaluator, reducing recall bias and lack of insight, which can limit subjective assessments. Based on the FDA's recommendation to our Pre-IND inquiry (PIND 152896), we have decided to use #5 item of MCS Visual Analog Scale (VAS) (40) as the primary outcome in this trial.…”

Section: Outcome Measures Columbia Muscle Cramp Scale (Mcs)mentioning

confidence: 99%

“…Endpoints of the Study: 1) Primary Safety Endpoint: No clinically signi cant signs and symptoms, no laboratory ndings of hypokalemia, nor other AEs. 2) Primary E cacy Endpoint: response to MCS item #5 (40): "Muscle Cramps Affecting Overall Daily Activity" We assess the MCS VAS obtained during the second week of each treatment period for comparison with the MCS obtained during the second week of each placebo periods. The study has been powered to detect a one-point difference, on average, between the treatment period and the placebo period.…”

Section: Columbia-suicide Severity Rating Scalementioning

confidence: 99%

“…We determined the sample size required based on the Visual Analog Scale endpoint under a four-period crossover design. Speci cally, assuming a within-subject standard deviation of the scale at 1.4 based on our previous publication (40), we sized the study to achieve 85% power to detect a one-point shift on the scale at 5% signi cance (two-sided) and obtained a total sample size of 22. We plan to randomize the participants in a 1:1 ratio to two possible treatment sequences: ABBA or BAAB.…”

Section: Sample Size and Study Powermentioning

confidence: 99%

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Randomized Double-Blind Personalized N-of-1 Clinical Trial to Test the Safety and Potential Efficacy of TJ-68 for Treating Muscle Cramps in Amyotrophic Lateral Sclerosis (ALS): study protocol for a TJ-68 trial.

Mitsumoto

Cheung

Oskarsson

et al. 2023

Preprint

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Introduction/Aims: Muscle cramps are a common and often disabling symptom in Amyotrophic Lateral Sclerosis (ALS), a devastating and incurable neurodegenerative disorder. To date, there are no medications specifically approved for the treatment of muscle cramps. Ameliorating muscle cramps in this patient population may sustain quality of life longer. A traditional Japanese medicine, shakuyakukanzoto (TJ-68) is widely prescribed in Japan for managing muscle cramps in a variety of conditions. The Japanese ALS Management Guideline mentions TJ-68 for difficult muscle cramps in ALS. Therefore, this clinical trial is designed to test the safety and efficacy of TJ-68 in participants with ALS reporting serious muscle cramps using a personalized N-of-1 approach. If successful, we can introduce TJ-68 for muscle cramps in ALS to Western medicine. Methods: With FDA-IND approval, we are conducting a two-site, double-blind, randomized personalized N-of-1 early clinical trial with TJ-68. At least 22 participants with ALS and daily muscle cramps will receive drug or placebo for 2 weeks (one treatment period) followed by a 1-week washout in a four-period cross-over design. While the primary objective is to evaluate the safety of TJ-68, the study was sized to detect a one-point shift on the Columbia Muscle Cramp Scale (MCS) item #5 with 85% power under a four-period crossover design.The secondary assessment includes the full MCS score, a Cramp Diary, Clinical Global Impression of Changes, Goal Attainment Scale, quality of life scale and ALS functional rating scale-revised (ALSFRS-R). Discussion: The study is underway. A personalized N-of-1 trial design is an efficient approach to testing medications that alleviate MCS in rare disorders. If TJ-68 proves safe and efficacious, we will work to introduce this product to Western medicine for treatment of patients with ALS and disabling muscle cramps. Trial Registration: This clinical trial has been registered with ClinicalTrials.gov (NCT04998305).

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Sodium Phenylbutyrate and Tauroursodeoxycholic Acid: A Story of Hope Turned to Disappointment in Amyotrophic Lateral Sclerosis Treatment

Ketabforoush,

Faghihi,

Azedi

et al. 2024

Clin Drug Investig

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A novel muscle cramp scale (MCS) in amyotrophic lateral sclerosis (ALS)

Cited by 9 publications

References 20 publications

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis

Randomized Double-Blind Personalized N-of-1 Clinical Trial to Test the Safety and Potential Efficacy of TJ-68 for Treating Muscle Cramps in Amyotrophic Lateral Sclerosis (ALS): study protocol for a TJ-68 trial.

Sodium Phenylbutyrate and Tauroursodeoxycholic Acid: A Story of Hope Turned to Disappointment in Amyotrophic Lateral Sclerosis Treatment

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