Trial Outcomes & Findings for The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia (NCT NCT01987219)
NCT ID: NCT01987219
Last Updated: 2016-06-02
Results Overview
FVC is a measure of the amount of air exhaled, and is measured in liters of air per second. The percentage in the change in the amount of air exhaled from baseline, measured in liters of air per second. Increase in the percentage of air exhaled from baseline indicates improvement in respiratory function.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
15 participants
Primary outcome timeframe
Pre and 30 minutes post study drug administration
Results posted on
2016-06-02
Participant Flow
Fifteen patients with Familial Dysautonomia were enrolled into the study between March 2013 and December 2015. The study was conducted at the NYU Dsyautonomia Center and Israeli FD center.
Participant milestones
| Measure |
Ipratropium; Alubterol; Placebo
Participants in this group received ipratropium followed by albuterol followed by placebo
|
Albuterol; Placebo; Ipratropium
Participants in this group received albuterol followed by placebo followed by ipratropium
|
Albuterol; Ipratropium; Placebo
Participants in this group received albuterol followed by ipratropium followed by placebo
|
Placebo; Ipratropium; Albuterol
Participants in this group received placebo followed by ipratropium followed by albuterol
|
Placebo; Albuterol; Ipratropium
Participants in this group received placebo followed by albuterol followed by ipratropium
|
Ipratropium; Placebo; Albuterol
Participants in this group received ipratropium followed by placebo followed by albuterol
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
7
|
2
|
1
|
1
|
|
Overall Study
COMPLETED
|
2
|
2
|
6
|
2
|
1
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia
Baseline characteristics by cohort
| Measure |
Ipratropium; Alubterol; Placebo
n=2 Participants
|
Albuterol; Placebo; Ipratropium
n=2 Participants
|
Albuterol; Ipratropium; Placebo
n=7 Participants
|
Placebo; Ipratropium; Albuterol
n=2 Participants
|
Placebo; Albuterol; Ipratropium
n=1 Participants
|
Ipratropium; Placebo; Albuterol
n=1 Participants
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
2 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
13 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
7 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
8 Participants
n=8 Participants
|
|
Race/Ethnicity, Customized
White, not of Hispanic-American Origin
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
7 participants
n=5 Participants
|
2 participants
n=4 Participants
|
1 participants
n=21 Participants
|
1 participants
n=8 Participants
|
15 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Pre and 30 minutes post study drug administrationPopulation: Per protocol
FVC is a measure of the amount of air exhaled, and is measured in liters of air per second. The percentage in the change in the amount of air exhaled from baseline, measured in liters of air per second. Increase in the percentage of air exhaled from baseline indicates improvement in respiratory function.
Outcome measures
| Measure |
Albuterol-sulphate
n=15 Participants
Albuterol-sulphate (Proventil ®) Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
Albuterol-sulphate: Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
|
Ipratropium-bromide (Atrovent ®)
n=15 Participants
IpratroAnti-cholinergic(Atrovent ®) 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.pium-bromide
Ipratropium-bromide: Anti-cholinergic 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.
|
Placebo
n=14 Participants
Placebo Saline solution 3 cc NA
placebo: Saline solution 3 cc NA
|
|---|---|---|---|
|
Change From Baseline of Forced Vital Capacity
|
5.5 percentage change from baseline
Standard Deviation 13.6
|
4.7 percentage change from baseline
Standard Deviation 10.9
|
0.9 percentage change from baseline
Standard Deviation 10.7
|
PRIMARY outcome
Timeframe: Pre and 30 minutes post study drug administrationThe percentage change in respiratory function from baseline is measured in percentage change in Resistance, kPa/(L/s).
Outcome measures
| Measure |
Albuterol-sulphate
n=15 Participants
Albuterol-sulphate (Proventil ®) Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
Albuterol-sulphate: Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
|
Ipratropium-bromide (Atrovent ®)
n=15 Participants
IpratroAnti-cholinergic(Atrovent ®) 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.pium-bromide
Ipratropium-bromide: Anti-cholinergic 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.
|
Placebo
n=14 Participants
Placebo Saline solution 3 cc NA
placebo: Saline solution 3 cc NA
|
|---|---|---|---|
|
Change in Respiratory Function (Airway Resistance at 5 Hz) From Baseline
|
-22.7 percentage change from baseline
Standard Deviation 18.6
|
-19.5 percentage change from baseline
Standard Deviation 15.5
|
-1.1 percentage change from baseline
Standard Deviation 12.5
|
SECONDARY outcome
Timeframe: Pre and 30 post study drug admistrationForced expiratory volume is measured in liters of air per second. FEV was measured during the first second of exhalation.
Outcome measures
| Measure |
Albuterol-sulphate
n=15 Participants
Albuterol-sulphate (Proventil ®) Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
Albuterol-sulphate: Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
|
Ipratropium-bromide (Atrovent ®)
n=15 Participants
IpratroAnti-cholinergic(Atrovent ®) 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.pium-bromide
Ipratropium-bromide: Anti-cholinergic 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.
|
Placebo
n=14 Participants
Placebo Saline solution 3 cc NA
placebo: Saline solution 3 cc NA
|
|---|---|---|---|
|
Change in Forced Expiratory Volume (FEV) From Baseline
|
7.69 percentage change from baseline
Standard Deviation 12.06
|
4.81 percentage change from baseline
Standard Deviation 9.9
|
-0.9 percentage change from baseline
Standard Deviation 7.6
|
SECONDARY outcome
Timeframe: pre and 30 minutes post interventionFEF25-75 is measured in liters of air per second at 25-75%
Outcome measures
| Measure |
Albuterol-sulphate
n=15 Participants
Albuterol-sulphate (Proventil ®) Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
Albuterol-sulphate: Beta-2-adrenergic agonist 2.5 mg 3 cc inhalation Peak effect 15 - 30 mins. Mean duration of effect 3 hours
|
Ipratropium-bromide (Atrovent ®)
n=15 Participants
IpratroAnti-cholinergic(Atrovent ®) 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.pium-bromide
Ipratropium-bromide: Anti-cholinergic 500 mcg 3 cc inhalation Peak effect 30 - 90 mins. Duration of effect 2 - 4 hours.
|
Placebo
n=14 Participants
Placebo Saline solution 3 cc NA
placebo: Saline solution 3 cc NA
|
|---|---|---|---|
|
Change in Forced Expiratory Flow Between 25-75% (FEF25-75)
|
14.7 percentage change from baseline
Standard Deviation 30.6
|
11.5 percentage change from baseline
Standard Deviation 36.1
|
-2.99 percentage change from baseline
Standard Deviation 23.3
|
Adverse Events
Albuterol-sulphate
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Ipratropium-bromide (Atrovent ®)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place