Trial Outcomes & Findings for TJ-68 Clinical Trial in Patients With Amyotrophic Lateral Sclerosis (ALS) and Muscle Cramps (NCT NCT04998305)

NCT ID: NCT04998305

Last Updated: 2025-05-01

Results Overview

This is designed to measure improvements in muscle cramps. MCS-VAS indicates the level to which muscle cramps affect overall daily activity. The score ranges from 0 to 10; 0 indicates no interference and 10 indicates severe interference with overall daily activity. MCS-VAS will be administered by a trained evaluator to reduce recall bias and lack of insight, which can limit subjective assessments. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported
• Results posted on: 2025-05-01

Participant Flow

Participant milestones

Measure	Treatment Sequence TJ-68-Placebo-Placebo-TJ-68 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 will be randomized to the following treatment sequences: TJ-68, placebo, placebo, TJ-68 (1 week WO between each treatment period)	Treatment Sequence Placebo-TJ-68-TJ-68-Placebo 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 will be randomized to the following treatment sequences: placebo, TJ-68, TJ-68, placebo (1 week WO between each treatment period)
Treatment (2 Weeks) STARTED	6	5
Treatment (2 Weeks) COMPLETED	6	5
Treatment (2 Weeks) NOT COMPLETED	0	0
Washout (1 Week) STARTED	6	5
Washout (1 Week) COMPLETED	6	5
Washout (1 Week) NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Treatment Sequence TJ-68-Placebo-Placebo-TJ-68 n=6 Participants 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 will be randomized to the following treatment sequences: TJ-68, placebo, placebo, TJ-68 (1 week WO between each treatment period)	Treatment Sequence Placebo-TJ-68-TJ-68-Placebo n=5 Participants 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 will be randomized to the following treatment sequences: placebo, TJ-68, TJ-68, placebo (1 week WO between each treatment period)	Total n=11 Participants Total of all reporting groups
Age, Continuous	63 years STANDARD_DEVIATION 8.15 • n=5 Participants	62.8 years STANDARD_DEVIATION 7.29 • n=7 Participants	62.9 years STANDARD_DEVIATION 7.38 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants	5 participants n=7 Participants	11 participants n=5 Participants

PRIMARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

This is designed to measure improvements in muscle cramps. MCS-VAS indicates the level to which muscle cramps affect overall daily activity. The score ranges from 0 to 10; 0 indicates no interference and 10 indicates severe interference with overall daily activity. MCS-VAS will be administered by a trained evaluator to reduce recall bias and lack of insight, which can limit subjective assessments. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
Visual Analog Scale (MCS-VAS) Score	2.07 score on a scale Standard Deviation 2.03	1.7 score on a scale Standard Deviation 1.91

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Changes in trigger, frequency, severity, and location of muscle cramps will be measured by administering all of MCS questions. Motor behaviors which trigger muscle cramps and muscle cramps' effects on sleep quality will also be measured. The score for each component of MCS -- trigger, frequency, severity, location, behavior, and effect on sleep quality -- will range from 1 to 5, with the severity increasing from 1 to 5. The total score range is 6 to 30. All of the MCS components will be administered by a trained evaluator and evaluated by the investigator. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
Overall Muscle Cramp Scale (MCS) Score	14.7 score on a scale Standard Deviation 5.07	13.1 score on a scale Standard Deviation 5.81

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Cramp pain will be measured on a scale of 0 to 10 with 0 indicating no pain and 10 indicating severe pain. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
Self-reported Cramp Pain Score	0.59 score on a scale Standard Deviation 0.41	0.49 score on a scale Standard Deviation 0.46

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Changes in functionality due to disease progression will be measured by administering ALSFRS-R to participants. ALSFRS-R includes 12 questions that can have a score of 0 to 4. A score of 0 on a question would indicate no function while a score of 4 would indicate full function. Total score range is 0 to 48. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
ALSFRS-R Score	34.1 score on a scale Standard Deviation 5.37	34.5 score on a scale Standard Deviation 5.39

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Changes in participant's feelings since the start of dosing will be measured by using a score of 1 to 7 with 1 indicating "very much improved" and 7 indicating "very much worse." To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
Clinical Global Impression of Changes (CGIC) Score	3.48 score on a scale Standard Deviation 1.24	2.95 score on a scale Standard Deviation 1.28

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Participants' motor functions and resulting quality of life will be measured by asking questions about their ability to perform certain tasks or feelings of hopelessness within the last two weeks. Participants can answer by saying never, rarely, sometimes, often, or always/cannot do at all. The ALSAQ-5 full score range is from 0 to 100, with 0 reflecting the best health state. To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
ALSAQ-5 (Quality of Life Questionnaire) Score	4.83 score on a scale Standard Deviation 2.39	5 score on a scale Standard Deviation 3.05

SECONDARY outcome

Timeframe: Assessed at Baseline, Week 2, Week 5, Week 8 and Week 11; Week 2 reported

Participant and the evaluator will collaborate and establish a goal. Progression of goal achievement will be measured over the course of participation and scored from -2 to +2 with -2 indicating "(achievement) much worse than expected" and +2 indicating "(achievement) much better than expected." To minimize carryover effects, results reflect the average of the scores taken at the second week of each treatment period.

Outcome measures

Measure	Placebo n=11 Participants Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 Participants Participants who were administered TJ-68 during the first 2-week treatment period.
Goal Attainment Scale (GAS) Score	0.50 score on a scale Standard Deviation 1.14	0.67 score on a scale Standard Deviation 1.11

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

TJ-68

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=11 participants at risk Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 participants at risk Participants who were administered TJ-68 during the first 2-week treatment period.
Infections and infestations Respiratory distress due to RSV	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
Gastrointestinal disorders Diverticulitis with pericecal abscess	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks

Other adverse events

Measure	Placebo n=11 participants at risk Participants who were administered the placebo during the first 2-week treatment period.	TJ-68 n=11 participants at risk Participants who were administered TJ-68 during the first 2-week treatment period.
General disorders Facial rash	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
Gastrointestinal disorders Abdominal bloating	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
Gastrointestinal disorders Stomachache	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
General disorders Elevated blood pressure	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
General disorders Flu-like symptoms	9.1% 1/11 • 11 weeks	9.1% 1/11 • 11 weeks
General disorders Joint pain	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
Eye disorders Scleral erythema	0.00% 0/11 • 11 weeks	9.1% 1/11 • 11 weeks
Infections and infestations COVID-19	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
General disorders Fatigue	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
General disorders Sinusitis	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
General disorders Hypertension	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
General disorders Fall	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
Blood and lymphatic system disorders Hypokalemia	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
Skin and subcutaneous tissue disorders Rash acneiform	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
Renal and urinary disorders Urinary frequency	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks
Respiratory, thoracic and mediastinal disorders Dyspnea	9.1% 1/11 • 11 weeks	0.00% 0/11 • 11 weeks

Additional Information

Hiroshi Mitsumoto, MD

Columbia University

Phone: 212-305-1319

Email: hm264@cumc.columbia.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place