Trial Outcomes & Findings for Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation (NCT NCT00116558)
NCT ID: NCT00116558
Last Updated: 2019-07-31
Results Overview
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
153 participants
Primary outcome timeframe
6 weeks
Results posted on
2019-07-31
Participant Flow
Participant milestones
| Measure |
Standard of Care NIPPV and Nutritional Monitoring
Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
|
Standard of Care NIPPV
Participants with 50-74% predicted forced vital capacity (FVC).
|
Early NIPPV
Participants with \>80% predicted forced vital capacity (FVC).
|
|---|---|---|---|
|
Overall Study
STARTED
|
80
|
26
|
47
|
|
Overall Study
COMPLETED
|
49
|
11
|
14
|
|
Overall Study
NOT COMPLETED
|
31
|
15
|
33
|
Reasons for withdrawal
| Measure |
Standard of Care NIPPV and Nutritional Monitoring
Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
|
Standard of Care NIPPV
Participants with 50-74% predicted forced vital capacity (FVC).
|
Early NIPPV
Participants with \>80% predicted forced vital capacity (FVC).
|
|---|---|---|---|
|
Overall Study
Death
|
11
|
5
|
3
|
|
Overall Study
Withdrawal by Subject
|
17
|
3
|
12
|
|
Overall Study
disease progression
|
3
|
7
|
18
|
Baseline Characteristics
Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
Baseline characteristics by cohort
| Measure |
Standard of Care NIPPV and Nutritional Monitoring
n=80 Participants
Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
|
Standard of Care NIPPV
n=26 Participants
Participants with 50-74% predicted forced vital capacity (FVC).
|
Early NIPPV Intervention
n=47 Participants
Participants with \>80% predicted forced vital capacity (FVC).
|
Total
n=153 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
59.0 years
STANDARD_DEVIATION 11.7 • n=5 Participants
|
60.8 years
STANDARD_DEVIATION 11.4 • n=7 Participants
|
56.2 years
STANDARD_DEVIATION 11.2 • n=5 Participants
|
58.6 years
STANDARD_DEVIATION 11.6 • n=4 Participants
|
|
Sex: Female, Male
Female
|
28 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
55 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
52 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
98 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
80 participants
n=5 Participants
|
26 participants
n=7 Participants
|
47 participants
n=5 Participants
|
153 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 6 weeksPopulation: Data were not obtained from the "Standard of Care NIPPV and Nutritional Monitoring" arm.
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Outcome measures
| Measure |
Standard of Care NIPPV and Nutritional Monitoring
Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
|
Standard of Care NIPPV
n=26 Participants
Participants with 50-74% predicted forced vital capacity (FVC).
|
Early NIPPV Intervention
n=47 Participants
Participants with \>80% predicted forced vital capacity (FVC).
|
|---|---|---|---|
|
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment.
|
0 Participants
|
23 Participants
|
35 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: one yearDuration of patient survival
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: one monthNumber of hours of NIPPV use per month
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Duration of study (approximately 1 year)Total daily energy expenditure (TDEE) with be measured using the dual labeled water (DLW) method
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: one monthPopulation: No data were collected for the "Standard of Care NIIPPV and Nutritional Monitoring" arm.
Percent of patients achieving tolerance (\>4hr/night NIPPV after 2-3 week titration period) determined by actual usage data.
Outcome measures
| Measure |
Standard of Care NIPPV and Nutritional Monitoring
Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
|
Standard of Care NIPPV
n=23 Participants
Participants with 50-74% predicted forced vital capacity (FVC).
|
Early NIPPV Intervention
n=35 Participants
Participants with \>80% predicted forced vital capacity (FVC).
|
|---|---|---|---|
|
Tolerance of NIPPV Treatment
|
0 Participants
|
11 Participants
|
14 Participants
|
Adverse Events
Combined Arms
Serious events: 42 serious events
Other events: 62 other events
Deaths: 19 deaths
Serious adverse events
| Measure |
Combined Arms
n=153 participants at risk
Sincere efforts were made to locate per arm data, but these data are no longer available. All adverse events are reported as one arm/group.
|
|---|---|
|
Nervous system disorders
ALS Progression
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration Pneumonia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Renal and urinary disorders
Bladder malignancy/UTI
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Blood Clot
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Cardiac disorders
Cardiac Arrest
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Colonic Bleed
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Nervous system disorders
Concussion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
General disorders
Death
|
9.2%
14/153 • Number of events 14 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Difficulty Breathing
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Dislodged PEG Tube
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Distended Abdomen
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Dysphagia
|
2.0%
3/153 • Number of events 3 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Metabolism and nutrition disorders
Failure to Thrive
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Hip Fracture
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Metabolism and nutrition disorders
Hypokalemia, hypomagnesia, and antythmia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Infections and infestations
Infection
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Cardiac disorders
Irregular Heartbeat
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Laceration
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Liver Cancer Diagnosis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Loculated right pleural effusion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Cardiac disorders
Myocardial Infarction
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
PEG Procedure Complications
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Surgical and medical procedures
Peg Replacement
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
2.6%
4/153 • Number of events 4 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Emboli and pneumonia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Complications
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Gastrointestinal disorders
Sepsis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Subarachroid Hemmorrhage and Contusion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Subdural Hematoma
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Transitional Cell Carcinoma
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
Other adverse events
| Measure |
Combined Arms
n=153 participants at risk
Sincere efforts were made to locate per arm data, but these data are no longer available. All adverse events are reported as one arm/group.
|
|---|---|
|
Gastrointestinal disorders
Abdominal Distension
|
2.0%
3/153 • Number of events 3 • 3 years, 4 months
|
|
Metabolism and nutrition disorders
Abnormal Lab
|
5.2%
8/153 • Number of events 8 • 3 years, 4 months
|
|
Immune system disorders
Allergy
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Ankle Sprain
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
2.6%
4/153 • Number of events 4 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Blister
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Blood Clot
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
2.6%
4/153 • Number of events 4 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Bruise
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
General disorders
Burning Sensation
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
General disorders
Cold
|
4.6%
7/153 • Number of events 8 • 3 years, 4 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colon Polyp
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Nervous system disorders
Concussion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Immune system disorders
Congestion
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Infections and infestations
Conjunctivitis
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Gastrointestinal disorders
Constipation
|
5.2%
8/153 • Number of events 8 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Contusion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Decreased Rectal Tone
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Dehydration
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Dental
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Diarrhea
|
2.6%
4/153 • Number of events 6 • 3 years, 4 months
|
|
Gastrointestinal disorders
Diverticulitis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Nervous system disorders
Dizziness
|
2.0%
3/153 • Number of events 4 • 3 years, 4 months
|
|
Eye disorders
Dry Eye
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Dysphagia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Infections and infestations
Ear Infection
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Blood and lymphatic system disorders
Edema
|
4.6%
7/153 • Number of events 7 • 3 years, 4 months
|
|
General disorders
Fall
|
5.2%
8/153 • Number of events 9 • 3 years, 4 months
|
|
General disorders
Fatigue
|
3.3%
5/153 • Number of events 5 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Fracture
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Gastritis
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Nervous system disorders
Hallucination
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Nervous system disorders
Headache
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Metabolism and nutrition disorders
Hypercarbia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Cardiac disorders
Hypertension
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Cardiac disorders
Hypotension
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Infections and infestations
Infection
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Knee Sprain
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Laceration
|
1.3%
2/153 • Number of events 3 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Lesion
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Nosebleed
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
PEG
|
2.0%
3/153 • Number of events 3 • 3 years, 4 months
|
|
General disorders
Pain
|
2.6%
4/153 • Number of events 6 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Immune system disorders
Post Nasal Drip
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Pressure Sores
|
0.65%
1/153 • Number of events 2 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
RADS
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.6%
4/153 • Number of events 4 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
1.3%
2/153 • Number of events 2 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Shoulder Pain
|
2.0%
3/153 • Number of events 3 • 3 years, 4 months
|
|
Gastrointestinal disorders
Sialorrhea
|
0.65%
1/153 • Number of events 3 • 3 years, 4 months
|
|
Infections and infestations
Sinus Infection
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
General disorders
Sore Throat
|
0.65%
1/153 • Number of events 2 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Spasm (Respiratory)
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Stomach Upset
|
2.0%
3/153 • Number of events 3 • 3 years, 4 months
|
|
Infections and infestations
UTI
|
2.6%
4/153 • Number of events 5 • 3 years, 4 months
|
|
Gastrointestinal disorders
Urgent Bowel Movements
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Renal and urinary disorders
Urinary Frequency
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Gastrointestinal disorders
Vomiting
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Weakness
|
2.0%
3/153 • Number of events 4 • 3 years, 4 months
|
|
General disorders
Weight Loss
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.65%
1/153 • Number of events 1 • 3 years, 4 months
|
Additional Information
Edward J. Kasarskis, M.D., Ph.D.
University of Kentucky
Phone: 859-218-5061
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place