Trial Outcomes & Findings for Acetazolamide Efficacy in Ataxia in PMM2-CDG (NCT NCT04679389)
NCT ID: NCT04679389
Last Updated: 2025-03-20
Results Overview
To achieve this goal, we compared the change of Mini-ICARS score from baseline to after six months of treatment between the placebo and active treatment groups. Minimal score is 0, maximum score is 100, higher score indicates greater impairment. Each subscale has an ordinal scale with a 0 indicating normal and the higher score indicating greater impairment or that the patient was unable to complete the task.
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
25 participants
Primary outcome timeframe
baseline-6 months
Results posted on
2025-03-20
Participant Flow
Participant milestones
| Measure |
Acetazolamide
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
12
|
|
Overall Study
COMPLETED
|
2
|
0
|
|
Overall Study
NOT COMPLETED
|
11
|
12
|
Reasons for withdrawal
| Measure |
Acetazolamide
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Protocol Violation
|
2
|
0
|
|
Overall Study
Physician Decision
|
3
|
5
|
|
Overall Study
Withdrawal by Subject
|
5
|
7
|
Baseline Characteristics
Acetazolamide Efficacy in Ataxia in PMM2-CDG
Baseline characteristics by cohort
| Measure |
Acetazolamide
n=13 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
13.62 years
STANDARD_DEVIATION 8.47 • n=5 Participants
|
10.88 years
STANDARD_DEVIATION 5.26 • n=7 Participants
|
12.30 years
STANDARD_DEVIATION 7.11 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
18 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
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
22 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
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=5 Participants
|
12 participants
n=7 Participants
|
25 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline-6 monthsPopulation: Data was not collected nor analyzed for two subjects in the Acetazolamide arm and one subject in the Placebo arm
To achieve this goal, we compared the change of Mini-ICARS score from baseline to after six months of treatment between the placebo and active treatment groups. Minimal score is 0, maximum score is 100, higher score indicates greater impairment. Each subscale has an ordinal scale with a 0 indicating normal and the higher score indicating greater impairment or that the patient was unable to complete the task.
Outcome measures
| Measure |
Acetazolamide
n=11 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=11 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Efficacy of Acetazolamide on Ataxia Measured Via Miniature International Cooperative Ataxia Rating Scale (Mini-ICARS)
|
1.64 score on a scale
Standard Deviation 3.20
|
1.36 score on a scale
Standard Deviation 3.23
|
SECONDARY outcome
Timeframe: through study completion, approximately 2 yearsBlood pH level was assessed through venous blood gas test. The number of participants who experience a drug related adverse event related to abnormal blood pH value.
Outcome measures
| Measure |
Acetazolamide
n=13 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Abnormal Blood pH Value
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: through study completion, approximately 2 yearsElectrolyte balance was assessed through combination testing on concentration of potassium, sodium, chloride, bicarbonate, magnesium, calcium, and phosphate. The number of participants who experience a drug related adverse event related to abnormal electrolyte balance.
Outcome measures
| Measure |
Acetazolamide
n=13 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Electrolyte Balance Testing
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: through study completion, approximately 2 yearsUrine calcium excretion is measured by mg excreted per day. The number of participants who experience a drug related adverse event related to abnormal excretion of calcium.
Outcome measures
| Measure |
Acetazolamide
n=13 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Urine Calcium Excretion Testing
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Data was not collected nor analyzed for this outcome measure. Data collection for this outcome never occurred. PMM2 biomarker carbohydrate deficient transferrin was not collected for any subjects. The test was never collected for any subjects.
Number of patients with abnormal ratio result will be recorded to understand the effect acetazolamide has on this biomarker
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline, 6 monthsPopulation: Data was not collected nor analyzed for seven subjects in the Acetazolamide arm and four subjects in the Placebo arm
PROMIS = Physical activity 10-items from 1(no days) to 5(6-7 days), Strength impact 12-item from 1(no days) to 5(6-7 days), Fatigue 23-item from 1(never) to 5(almost always), Mobility 23-item from 1(not able to do) to 5(with no trouble), Pain interference 13-item from 1(never) to 5(almost always), Upper extremity coordination 29-item from 1(not able to do) to 5(with no trouble), Global Health 9-item from 1(poor) to 5( excellent), Parent Proxy Mobility 8-item from 1(not able to do) to 5(with no trouble), Anxiety 8-item from 1(never) to 5(almost always), Depresson 8-item from 1(never) to 5(almost always), Parent Proxy Fatigue 8-item from 1(never) to 5(almost always), Peer relationships 8-item from 1(never) to 5(almost always), Parent proxy pain interference 8-item from 1(never) to 5(almost always), Pain intensity 1-item from 0(no pain) to 10(worst pain). Total scores range from 168 - 845. Lower scores indicate worse health, higher scores indicate better health
Outcome measures
| Measure |
Acetazolamide
n=6 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=8 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Change in Patient Reported Outcomes Measurement Information System (PROMIS) Score
|
3.02 score on a scale
Standard Deviation 4.45
|
0.75 score on a scale
Standard Deviation 4.09
|
SECONDARY outcome
Timeframe: baseline, 6 monthsPopulation: Data was not collected nor analyzed for four participants in the Acetazolamide arm and three participants in the Placebo arm
PATA test measures the number of times a patient can say the word "PATA" in a 10 second time period. Number of "PATA"s spoken in 10 seconds indicates level of dysarthria. The higher the score the less dysarthria, the lower the score more dysarthria.
Outcome measures
| Measure |
Acetazolamide
n=9 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=9 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Change in Dysarthria as Measured by the PATA Score
|
2.30 words/10 sec
Standard Deviation 3.71
|
0.59 words/10 sec
Standard Deviation 3.11
|
SECONDARY outcome
Timeframe: baseline, 6 monthsPopulation: Data was not collected nor analyzed for three participants in the Acetazolamide arm and one participant in the Placebo arm
The NPCRS is a scale that evaluates the patient's current function, system specific involvement, and current clinical assessment. Total scores range from 0 - 82. A mild score is 0-14, moderate score is 15-25, and severe is a score \>26.
Outcome measures
| Measure |
Acetazolamide
n=10 Participants
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=11 Participants
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Change in Nijmegen Pediatric CDG Rating Scale (NPCRS)
|
1.20 score on a scale
Standard Deviation 1.99
|
0.27 score on a scale
Standard Deviation 1.79
|
Adverse Events
Acetazolamide
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Acetazolamide
n=13 participants at risk
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 participants at risk
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Metabolism and nutrition disorders
Dehydration
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Fever
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Infections and infestations
Sepsis
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Vascular disorders
Thromboembolic Event
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Renal and urinary disorders
Acute Kidney Injury
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
Other adverse events
| Measure |
Acetazolamide
n=13 participants at risk
Acetazolamide was administered via capsule or liquid suspension. Capsule was 250 mg oral capsules encapsulated by gelatin capsule and filled with lactose to match placebo. Liquid suspension was 25 mg/mL oral suspension but adding 125 mg Acetazolamide tablets to suspending agent Ora-blend
Acetazolamide: administered orally or enterally
|
Placebo
n=12 participants at risk
Placebo was administered via capsule or liquid suspension. Capsule was a gelatin capsule filled with lactose powder to match Acetazolamide. Liquid suspension was a Ora-blend.
Placebo: administered orally or enterally
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Metabolism and nutrition disorders
Acidosis
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Ataxia
|
7.7%
1/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Bloating
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Blood and lymphatic system disorders
Blood and Lymphatic System Disorders - Other
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Metabolism and nutrition disorders
Anorexia
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
23.1%
3/13 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Dizziness
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Ear and labyrinth disorders
Ear and Labyrinth Disorders - Other
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Eye disorders
Eye Disorders - Other
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Eye disorders
Eye Pain
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Injury, poisoning and procedural complications
Fall
|
15.4%
2/13 • Number of events 4 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Fatigue
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
25.0%
3/12 • Number of events 5 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Fever
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Musculoskeletal and connective tissue disorders
Flank Pain
|
7.7%
1/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Gastritis
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Gastrointestinal Disorders - Other
|
30.8%
4/13 • Number of events 8 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 5 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
General Disorders and Administration Site Conditions - Other
|
15.4%
2/13 • Number of events 12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
25.0%
3/12 • Number of events 6 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Headache
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Hypersomnia
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Immune system disorders
Immune System Disorders - Other
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Infections and infestations
Infections and Infestations - Other
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
41.7%
5/12 • Number of events 6 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Investigations
Investigations - Other
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 4 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Lethargy
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Malaise
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Metabolism and nutrition disorders
Metabolism and Nutrition Disorders - Other
|
15.4%
2/13 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and Connective Tissue Disorder - Other
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Nausea
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Nervous System Disorders - Other
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
16.7%
2/12 • Number of events 5 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Ear and labyrinth disorders
Otitis Media
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Infections and infestations
Pharyngitis
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Skin and subcutaneous tissue disorders
Rash Acneiform
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Renal and urinary disorders
Renal and Urinary Disorders - Other
|
23.1%
3/13 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, Thoracic and Mediastinal Disorders - Other
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Seizure
|
7.7%
1/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Infections and infestations
Sinusitis
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Skin and subcutaneous tissue disorders
Skin and Subcutaneous Tissue Disorders - Other
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Somnolence
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Stomach Pain
|
15.4%
2/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Nervous system disorders
Tremor
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Renal and urinary disorders
Urinary Frequency
|
30.8%
4/13 • Number of events 4 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Renal and urinary disorders
Urinary Incontinence
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Gastrointestinal disorders
Vomiting
|
7.7%
1/13 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
33.3%
4/12 • Number of events 6 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Edema Limbs
|
0.00%
0/13 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
8.3%
1/12 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
Infections and infestations
Rhinitis Infective
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
|
General disorders
Non-Cardiac Chest Pain
|
7.7%
1/13 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
0.00%
0/12 • Adverse Events were collected from baseline to end of study, approximately 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place