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?

This trial tests the safety and potential benefits of a treatment called TJ-68 for people with ALS, a disease affecting nerve cells in the brain and spinal cord. Researchers aim to determine if TJ-68 can reduce muscle cramps, a common ALS symptom. Participants will alternate between taking TJ-68 and a placebo over several weeks to assess the treatment's effectiveness. The trial seeks individuals diagnosed with ALS who experience daily muscle cramps and meet specific health criteria. As a Phase 1, Phase 2 trial, this research focuses on understanding how TJ-68 works in people and measuring its effectiveness in an initial, smaller group.

Is there any evidence suggesting that TJ-68 is likely to be safe for humans?

Research has shown that TJ-68 is generally safe for use. In a previous study, participants tried both the treatment and a placebo (a harmless pill with no effect) at different times. TJ-68 was well-tolerated, with most participants experiencing no serious side effects. These studies primarily aim to determine if the treatment is safe and can help reduce muscle cramps in people with ALS, a disease affecting nerve cells in the brain and spinal cord. Although detailed information about side effects is limited, its use in other studies suggests it is safe enough for further testing. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Researchers are excited about TJ-68 for ALS because it offers a unique approach compared to existing treatments. Unlike the standard medications that primarily focus on slowing disease progression or managing symptoms, TJ-68 is derived from traditional Japanese herbal medicine, which may provide a novel way to target ALS. This treatment uses natural compounds that could work differently from current drugs, potentially offering new hope for patients. Additionally, the crossover design of the trial allows each participant to serve as their own control, which might lead to more personalized insights into its effects.

What evidence suggests that TJ-68 might be an effective treatment for ALS?

Research shows that TJ-68 might help reduce muscle cramps in people with ALS, a disease affecting nerve cells in the brain and spinal cord. In this trial, participants will undergo different treatment sequences involving TJ-68 and a placebo. Studies have found that TJ-68 is safe and may work well in small, personalized trials. Specifically, participants in those trials experienced fewer muscle cramps with TJ-68 compared to a placebo. This suggests that TJ-68 could ease muscle cramps, providing some relief for those with ALS. ^{1 2 4 6 7}

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+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40545904/

Placebo-Controlled, Randomized Double-Blind N-Of-1 ... The study revealed that N-of-1 trial design can detect changes in a small sample size, and TJ-68 appeared to be safe.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37430314/

study protocol for a TJ-68 trial We are conducting a randomized clinical trial to test the safety and efficacy of TJ-68 in participants with ALS reporting frequent muscle cramps.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04998305

TJ-68 Clinical Trial in Patients With Amyotrophic Lateral ... 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 ...

4. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1002/mus.28459?af=R

Placebo‐Controlled, Randomized Double‐Blind N‐Of‐1 ... Randomized double-blind personalized N-of-1 clinical trial to test the safety and potential efficacy of TJ-68 for treating muscle cramps in ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04998305?cond=multiple+sclerosis

Study Details | TJ-68 Clinical Trial in Patients With Amyotrophic ... An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of ...

6. als.net als.net/als-trial-navigator/600/

TJ-68 Clinical Trial in Patients With Amyotrophic... 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.

7. mayo.edu mayo.edu/research/clinical-trials/cls-20531309

TJ-68 Clinical Trial in Patients with Amyotrophic Lateral ... The purpose of this study is to demonstrate the safety and potential effectiveness of TJ-68 for improving muscle cramps in participants with ...