TJ-68 for ALS

Recruiting at 2 trial locations

Overseen By Hiroshi Mistumoto, MD, Dsc.

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Hiroshi Mitsumoto

Must be taking: Riluzole, Edaravone, Relyvrio

Must not be taking: Corticosteroids, Diuretics

Disqualifiers: Allergies, Pseudoaldosteronism, Hypokalemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 11 weeks

11 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Who Is on the Research Team?

Hiroshi Mistumoto, MD, Dsc.

Principal Investigator

Columbia University

Are You a Good Fit for This Trial?

Adults aged 20-70 with ALS, PMA, or PLS who experience daily muscle cramps. They must be able to swallow liquids, have a caregiver if needed for communication, and can travel to the study site. Participants should be on stable doses of certain ALS medications and not taking specific OTC products or drugs that affect enzyme metabolism.

Inclusion Criteria

I am willing to stop using OTC products with peony root or Glycyrrhiza.

I understand and am willing to sign the informed consent.

My lung function is at least 45% of what is considered normal.

Exclusion Criteria

I do not have any active diseases needing treatment, other than ALS.

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

I am not taking any medication that increases the risk of low potassium or pseudoaldosteronism, except for potassium-sparing diuretics.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive TJ-68 and placebo in a multi-period crossover design, serving as their own controls

11 weeks

Weekly visits for assessment

Washout

1-week washout period between each 2-week treatment period

3 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• TJ-68

Trial Overview The trial is testing TJ-68 against a placebo to see if it helps with muscle cramps in ALS patients. It's a double-blind study where neither doctors nor participants know who gets the real treatment as they're randomly assigned.

How Is the Trial Designed?

Group I: Treatment sequence TJ-68-Placebo-Placebo-TJ-68 Experimental Treatment 2 Interventions

Employing an N-of-1, crossover design, each participant in the TJ-68 clinical trial will serve as his/her control. The participation will last for 11 weeks - four, 2-week treatment periods with 1-week washout (WO) period between each treatment period. Participants (n=13) will be randomized to the following treatment sequences: TJ-68, placebo, placebo, TJ-68 (1 week WO between each treatment period)

Group II: Treatment sequence Placebo-TJ-68-TJ-68-Placebo Experimental Treatment 2 Interventions

Employing an N-of-1, crossover design, each participant in the TJ-68 clinical trial will serve as his/her control. The participation will last for 11 weeks - four, 2-week treatment periods with 1-week washout (WO) period between each treatment period. Participants (n=13) will be randomized to the following treatment sequences: placebo, TJ-68, TJ-68, placebo (1 week WO between each treatment period)

Who Is Running the Clinical Trial?

Hiroshi Mitsumoto

Lead Sponsor

Trials

1

Recruited

10+

Tsumura & Co., Tokyo, Japan

Collaborator

Trials

1

Recruited

10+