Trial Outcomes & Findings for Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III (NCT NCT00415519)
NCT ID: NCT00415519
Last Updated: 2017-12-20
Results Overview
No primary endpoint was used, because various exploratory analyses were performed. 0=worst; 48=best
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
25 participants
Primary outcome timeframe
baseline and 24 weeks
Results posted on
2017-12-20
Participant Flow
Participant milestones
| Measure |
MCI-186
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
12
|
|
Overall Study
COMPLETED
|
9
|
12
|
|
Overall Study
NOT COMPLETED
|
4
|
0
|
Reasons for withdrawal
| Measure |
MCI-186
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Patient's convenience
|
1
|
0
|
Baseline Characteristics
Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III
Baseline characteristics by cohort
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline and 24 weeksNo primary endpoint was used, because various exploratory analyses were performed. 0=worst; 48=best
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Change From Baseline in Revised ALS Functional Rating Scale (ALSFRS-R) Score in Full Analysis Set (FAS) Population at 24 Weeks
|
-6.52 units on a scale
Standard Error 1.78
|
-6 units on a scale
Standard Error 1.83
|
PRIMARY outcome
Timeframe: 24 weeksNo primary endpoint was used, because various exploratory analyses were performed. Any of "death, disability of independent ambulation, loss of upper arm function, tracheotomy, use of respirator, and use of tube feeding" was defined as an event.
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Death or a Specified State of Disease Progression
death
|
1 events
|
0 events
|
|
Death or a Specified State of Disease Progression
disability of independent ambulation
|
4 events
|
2 events
|
|
Death or a Specified State of Disease Progression
loss of upper arm function
|
1 events
|
2 events
|
|
Death or a Specified State of Disease Progression
tracheotomy
|
0 events
|
0 events
|
|
Death or a Specified State of Disease Progression
use of respirator
|
0 events
|
0 events
|
|
Death or a Specified State of Disease Progression
use of tube feeding
|
1 events
|
0 events
|
PRIMARY outcome
Timeframe: baseline and 24 weeksPopulation: "1 patient with missing value at baseline" was excluded from the FAS in the MCI-186 group.
No primary endpoint was used, because various exploratory analyses were performed.
Outcome measures
| Measure |
MCI-186
n=12 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Change From Baseline in % Forced Vital Capacity (%FVC) in Full Analysis Set (FAS) Population at 24 Weeks
|
-18.75 percentage of FVC
Standard Error 4.58
|
-15.69 percentage of FVC
Standard Error 4.58
|
PRIMARY outcome
Timeframe: 24 weeksNo primary endpoint was used, because various exploratory analyses were performed.
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Percentage of Participants With Adverse Events
|
92.3 percentage of participants
|
100 percentage of participants
|
PRIMARY outcome
Timeframe: 24 weeksNo primary endpoint was used, because various exploratory analyses were performed.
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Percentage of Participants With Adverse Drug Reactions
|
23.1 percentage of participants
|
8.3 percentage of participants
|
PRIMARY outcome
Timeframe: 24 weeksNo primary endpoint was used, because various exploratory analyses were performed.
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
The Percentage of Participants With an Abnormal Change in Laboratory Tests That Occurred in More Than Two Patients
White blood cell count (WBC)
|
23.1 percentage of participants
|
8.3 percentage of participants
|
|
The Percentage of Participants With an Abnormal Change in Laboratory Tests That Occurred in More Than Two Patients
Other laboratory tests except for WBC
|
0 percentage of participants
|
0 percentage of participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: A note: Each three patients of both groups did not have data to Staggering due to data missing. Therefore, concerning staggering, the number of participants analysed are 10 in the MCI-186 group and 9 in the placebo of MCI-186 group.
No primary endpoint was used, because various exploratory analyses were performed.
Outcome measures
| Measure |
MCI-186
n=13 Participants
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 Participants
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Percentage of Participants With Abnormal Changes in Sensory Examinations
Numbness
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Abnormal Changes in Sensory Examinations
Staggering
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Abnormal Changes in Sensory Examinations
Vibratory sensation
|
0 percentage of participants
|
0 percentage of participants
|
Adverse Events
MCI-186
Serious events: 3 serious events
Other events: 12 other events
Deaths: 0 deaths
Placebo of MCI-186
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
MCI-186
n=13 participants at risk
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 participants at risk
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Gastrointestinal disorders
Dysphagia
|
15.4%
2/13
|
0.00%
0/12
|
|
General disorders
Gait disturbance
|
7.7%
1/13
|
0.00%
0/12
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal disorder
|
7.7%
1/13
|
8.3%
1/12
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
7.7%
1/13
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
7.7%
1/13
|
0.00%
0/12
|
|
Vascular disorders
Pelvic venous thrombosis
|
0.00%
0/13
|
8.3%
1/12
|
Other adverse events
| Measure |
MCI-186
n=13 participants at risk
MCI-186 Injection 30 mg, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
Placebo of MCI-186
n=12 participants at risk
MCI-186 Injection Placebo, 2 ampoules per treatment, once daily, intravenously infused over 60 min
|
|---|---|---|
|
Ear and labyrinth disorders
Vertigo positional
|
7.7%
1/13
|
0.00%
0/12
|
|
Eye disorders
Abnormal sensation in eye
|
0.00%
0/13
|
8.3%
1/12
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13
|
0.00%
0/12
|
|
Gastrointestinal disorders
Diarrhoea
|
23.1%
3/13
|
8.3%
1/12
|
|
Gastrointestinal disorders
Dyschezia
|
0.00%
0/13
|
25.0%
3/12
|
|
Gastrointestinal disorders
Gingivitis
|
7.7%
1/13
|
0.00%
0/12
|
|
Gastrointestinal disorders
Proctalgia
|
0.00%
0/13
|
8.3%
1/12
|
|
Gastrointestinal disorders
Stomach discomfort
|
0.00%
0/13
|
8.3%
1/12
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/13
|
25.0%
3/12
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/13
|
8.3%
1/12
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/13
|
8.3%
1/12
|
|
General disorders
Abasia
|
0.00%
0/13
|
8.3%
1/12
|
|
General disorders
Feeling cold
|
7.7%
1/13
|
0.00%
0/12
|
|
General disorders
Gait disturbance
|
23.1%
3/13
|
8.3%
1/12
|
|
General disorders
Impaired healing
|
7.7%
1/13
|
0.00%
0/12
|
|
General disorders
Thirst
|
0.00%
0/13
|
8.3%
1/12
|
|
Infections and infestations
Acarodermatitis
|
7.7%
1/13
|
0.00%
0/12
|
|
Infections and infestations
Cystitis
|
0.00%
0/13
|
8.3%
1/12
|
|
Infections and infestations
Nasopharyngitis
|
15.4%
2/13
|
16.7%
2/12
|
|
Infections and infestations
Oral herpes
|
7.7%
1/13
|
0.00%
0/12
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/13
|
8.3%
1/12
|
|
Infections and infestations
Sinusitis
|
0.00%
0/13
|
8.3%
1/12
|
|
Infections and infestations
Tinea pedis
|
7.7%
1/13
|
0.00%
0/12
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.00%
0/13
|
8.3%
1/12
|
|
Injury, poisoning and procedural complications
Contusion
|
7.7%
1/13
|
8.3%
1/12
|
|
Injury, poisoning and procedural complications
Joint sprain
|
0.00%
0/13
|
16.7%
2/12
|
|
Investigations
Blood urine present
|
0.00%
0/13
|
8.3%
1/12
|
|
Investigations
Glucose urine present
|
7.7%
1/13
|
0.00%
0/12
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/13
|
8.3%
1/12
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
7.7%
1/13
|
0.00%
0/12
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal disorder
|
0.00%
0/13
|
8.3%
1/12
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/13
|
8.3%
1/12
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/13
|
16.7%
2/12
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/13
|
8.3%
1/12
|
|
Nervous system disorders
Headache
|
23.1%
3/13
|
16.7%
2/12
|
|
Nervous system disorders
Post herpetic neuralgia
|
0.00%
0/13
|
8.3%
1/12
|
|
Nervous system disorders
Tension headache
|
0.00%
0/13
|
8.3%
1/12
|
|
Psychiatric disorders
Anxiety disorder
|
0.00%
0/13
|
8.3%
1/12
|
|
Psychiatric disorders
Depression
|
7.7%
1/13
|
0.00%
0/12
|
|
Psychiatric disorders
Insomnia
|
7.7%
1/13
|
8.3%
1/12
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
7.7%
1/13
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/13
|
8.3%
1/12
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/13
|
8.3%
1/12
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/13
|
8.3%
1/12
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
7.7%
1/13
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Sputum retention
|
7.7%
1/13
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
23.1%
3/13
|
8.3%
1/12
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/13
|
8.3%
1/12
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.7%
1/13
|
0.00%
0/12
|
|
Skin and subcutaneous tissue disorders
Haemorrhage subcutaneous
|
0.00%
0/13
|
8.3%
1/12
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
15.4%
2/13
|
8.3%
1/12
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.4%
2/13
|
0.00%
0/12
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
|
0.00%
0/13
|
8.3%
1/12
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER