TJ-68 Clinical Trial in Patients With Amyotrophic Lateral Sclerosis (ALS) and Muscle Cramps

• Status: Not yet recruiting
• Trial end date: 2023-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of the study is to demonstrate the safety and potential efficacy of TJ-68 for improving muscle cramps in participants with ALS based on a two-site, randomized, placebo-controlled double-blind multi-period crossover (N-of-1) study design.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hiroshi Mitsumoto

Criteria

Inclusion Criteria:

• Diagnosed with ALS, PMA or PLS based on the El Escorial ALS Diagnostic Criteria or based on more recently revised Gold Coast ALS diagnostic criteria

• Experiences at least one muscle cramp in any muscle per day

• Age 20 to 70 years old

• Forced vital capacity is 60% of normal or greater in a seated position

• Able to swallow liquid via the mouth or be given via a feeding tube

• Caregiver available to assist with speaking or writing on behalf of the participant if they are not able to speak or write due to the disease

• Able to comprehend and willing to give (sign) the informed consent

• Willing to commute to the study site for the frequent visits, including a screening visit (study visits at the end of week 2, 5, 8 and 11)

• Taking a stable dose of Riluzole (Rilutek), Edaravone (RADICAVA), or both for at least a month before randomization and not expected to require dose titration or initiation of these medications during the study period

• Willing to discontinue over-the-counter (OTC) products containing any peony root, Glycyrrhiza, or both

• Willing to discontinue Mexiletine, Quinine sulfate, or Ranolazine during the study period

• Willing to avoid food, beverages, and medications that may induce or inhibit metabolism of enzyme of transporters.

• Willing to refrain from initiation or dose adjustment of baclofen, gabapentin, pregabalin, and/or memantine during the study period (stable dosing of these medications is allowed).

• Willing to practice contraceptive measures for male and female patients.

Exclusion Criteria:

• History of allergic reactions to peony root, Glycyrrhiza, or FD&C Yellow No. 5 (tartrazine)

• Takes any medication known to increase the risk of pseudoaldosteronism or hypokalemia, including corticosteroids and diuretics (except potassium sparing diuretics, such as spironolactone or amiloride)

• History of pseudoaldosteronism or hypokalemia or current use of potassium supplementation

• Screening potassium level 3.4 mEq/L or less

• Screening diastolic blood pressure (DBP) more than 90 mmHg or systolic blood pressure (SBP) more than 150 mmHg after sufficient rest

• Screening albumin below normal laboratory level either at the Columbia or Mayo Clinic Jacksonville laboratory

• Screening bicarbonate or carbon dioxide level less than 19 mmol/L, suggesting metabolic alkalosis

• Screening sodium level greater than 145 mmol/L, suggesting hypernatremia

• Unstable or active medical or neurological (other than ALS) diseases which require treatment

• Failure of Capacity Assessment

• Not able and/or willing to comprehend and sign the informed consent

• Not able to speak or write English to complete the primary outcome measure, MCS

• Taking any experimental medication or unapproved medications

• Those who are pregnant or breast feeding

• Those who have renal or hepatic impairment