Trial Outcomes & Findings for Smell Changes & Efficacy of Nasal Theophylline (NCT NCT03990766)
NCT ID: NCT03990766
Last Updated: 2022-03-31
Results Overview
Self-reported change in smell after intervention on a 7-point Likert scale. Response options with corresponding point values: 7) Much better, 6) Somewhat better, 5) Slightly better, 4) Neither better nor worse, 3) Slightly worse, 2) Somewhat worse, and 1) Much worse. The maximum score is 7, and higher values indicate better outcomes. A score of 4 is considered neutral. Scores of 5 or higher indicated clinical improvement.
COMPLETED
PHASE2
27 participants
6 weeks
2022-03-31
Participant Flow
Participant milestones
| Measure |
Theophylline
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
13
|
|
Overall Study
COMPLETED
|
12
|
10
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Smell Changes & Efficacy of Nasal Theophylline
Baseline characteristics by cohort
| Measure |
Theophylline Saline Irrigation
n=12 Participants
Theophylline 12 mg capsule contents dissolved into a sinus rinse bottle containing distilled or boiled tap water and USP Grade Sodium Chloride \& Sodium Bicarbonate Mixture commercially prepared packets, delivered to the bilateral nasal cavities twice daily.
Theophylline: Theophylline delivered via high-volume, low-pressure nasal saline irrigation
|
Placebo Saline Irrigation
n=10 Participants
Identical-appearing lactose monohydrate capsule contents dissolved into a sinus rinse bottle containing distilled or boiled tap water and USP Grade Sodium Chloride \& Sodium Bicarbonate Mixture commercially prepared packets, delivered to the bilateral nasal cavities twice daily.
Saline Nasal: Lactose delivered via high-volume, low-pressure nasal saline irrigation
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
57.5 years
n=113 Participants
|
59.5 years
n=163 Participants
|
59.0 years
n=160 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=113 Participants
|
6 Participants
n=163 Participants
|
14 Participants
n=160 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=113 Participants
|
4 Participants
n=163 Participants
|
8 Participants
n=160 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
0 Participants
n=160 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
0 Participants
n=160 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
0 Participants
n=160 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=113 Participants
|
1 Participants
n=163 Participants
|
2 Participants
n=160 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=113 Participants
|
9 Participants
n=163 Participants
|
20 Participants
n=160 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
0 Participants
n=160 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
0 Participants
n=160 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=113 Participants
|
10 participants
n=163 Participants
|
22 participants
n=160 Participants
|
|
Body Mass Index
|
32.3 kg/m^2
n=113 Participants
|
33.0 kg/m^2
n=163 Participants
|
33.0 kg/m^2
n=160 Participants
|
|
Smoking history
|
0 Participants
n=113 Participants
|
2 Participants
n=163 Participants
|
2 Participants
n=160 Participants
|
|
Deviated septum
|
3 Participants
n=113 Participants
|
2 Participants
n=163 Participants
|
5 Participants
n=160 Participants
|
|
Diabetes mellitus
|
1 Participants
n=113 Participants
|
1 Participants
n=163 Participants
|
2 Participants
n=160 Participants
|
|
Obstructive sleep apnea on positive airway pressure
|
2 Participants
n=113 Participants
|
0 Participants
n=163 Participants
|
2 Participants
n=160 Participants
|
|
Hypothyroidism
|
3 Participants
n=113 Participants
|
1 Participants
n=163 Participants
|
4 Participants
n=160 Participants
|
|
Family history of dementia
|
2 Participants
n=113 Participants
|
1 Participants
n=163 Participants
|
3 Participants
n=160 Participants
|
|
Moderate/severe comorbidity (ACE-27 scores 2-3)
|
5 Participants
n=113 Participants
|
1 Participants
n=163 Participants
|
6 Participants
n=160 Participants
|
|
Prior oral steroids
|
8 Participants
n=113 Participants
|
6 Participants
n=163 Participants
|
14 Participants
n=160 Participants
|
|
Prior nasal steroids
|
9 Participants
n=113 Participants
|
7 Participants
n=163 Participants
|
16 Participants
n=160 Participants
|
|
Prior olfactory training
|
1 Participants
n=113 Participants
|
2 Participants
n=163 Participants
|
3 Participants
n=160 Participants
|
|
Time from upper respiratory infection to treatment
|
11.0 months
n=113 Participants
|
8.4 months
n=163 Participants
|
8.8 months
n=160 Participants
|
PRIMARY outcome
Timeframe: 6 weeksSelf-reported change in smell after intervention on a 7-point Likert scale. Response options with corresponding point values: 7) Much better, 6) Somewhat better, 5) Slightly better, 4) Neither better nor worse, 3) Slightly worse, 2) Somewhat worse, and 1) Much worse. The maximum score is 7, and higher values indicate better outcomes. A score of 4 is considered neutral. Scores of 5 or higher indicated clinical improvement.
Outcome measures
| Measure |
Theophylline
n=12 Participants
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
n=10 Participants
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
Number of Patients With Improvement in Global Rating of Smell Change
|
4 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 6 weeksWithin- and between-subject changes in UPSIT total scores measured at baseline and at 6 weeks after therapy. This test is a validated 40-question forced-choice odor identification test where microencapsulated odorants on a strip are released by scratching. Each question corresponds to one point, and there are no subscales. The minimum score is 0 while the maximum score is 40, and higher values indicate better outcomes.
Outcome measures
| Measure |
Theophylline
n=12 Participants
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
n=10 Participants
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
University of Pennsylvania Smell Identification Test (UPSIT) Total Score Change
|
1 score on a scale
Interval -4.0 to 12.0
|
0 score on a scale
Interval -8.0 to 5.0
|
SECONDARY outcome
Timeframe: 6 weeksWithin- and between-subject changes in QOD-NS scores measured at baseline and at 6 weeks after therapy, which is a validated 17-item questionnaire about quality of life and impairments related to olfactory dysfunction. Each question is scored from 0 to 3 points, and there are no subscales. The minimum score is 0 while the maximum score is 51, and higher values indicate worse quality of life or higher degree of impairment.
Outcome measures
| Measure |
Theophylline
n=12 Participants
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
n=10 Participants
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
Questionnaire of Olfactory Disorders-Negative Statements (QOD-NS) Score Change
|
-6.5 score on a scale
Interval -15.0 to 4.0
|
1.5 score on a scale
Interval -4.0 to 19.0
|
SECONDARY outcome
Timeframe: 6 weeksWithin- and between-subject changes measured at baseline and at 6 weeks after therapy. This test is a new disease-specific 28-item questionnaire that assesses for physical, functional, and emotional limitations in patients with olfactory dysfunction of any etiology. Each question is scored from 0 to 4. The minimum score is 0 while the maximum score is 112, and higher values indicate higher degree of limitations and worse outcomes. There are no subscales within the current questionnaire.
Outcome measures
| Measure |
Theophylline
n=12 Participants
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
n=10 Participants
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
Olfactory Dysfunction Outcomes Ratings (ODOR) Score Change
|
-1.5 score on a scale
Interval -18.0 to 6.0
|
-7.5 score on a scale
Interval -29.0 to 6.0
|
Adverse Events
Theophylline
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Theophylline
n=14 participants at risk
12 mg of theophylline dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
Placebo
n=13 participants at risk
Lactose capsule contents dissolved in 240 mL of saline, administered to the bilateral nasal cavities twice a day for 6 weeks
|
|---|---|---|
|
Blood and lymphatic system disorders
Detectable serum theophylline
|
0.00%
0/5 • Adverse event data were collected over 6 weeks of treatment
The first 10 participants regardless of intervention underwent theophylline level measurements after 1 week of treatment. If a measurable amount of theophylline was identified, the test would be immediately repeated. If theophylline was detected in the second sample, the plan was to continue measuring in another 10 subjects. In addition, all participants were queried biweekly regarding any adverse effects.
|
0.00%
0/5 • Adverse event data were collected over 6 weeks of treatment
The first 10 participants regardless of intervention underwent theophylline level measurements after 1 week of treatment. If a measurable amount of theophylline was identified, the test would be immediately repeated. If theophylline was detected in the second sample, the plan was to continue measuring in another 10 subjects. In addition, all participants were queried biweekly regarding any adverse effects.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place