Trial Outcomes & Findings for Aerosolized Hypertonic Xylitol Versus Hypertonic Saline in Cystic Fibrosis (CF) Subjects (NCT NCT00928135)

NCT ID: NCT00928135

Last Updated: 2021-06-25

Results Overview

Change in lung function (forced expiratory volume in 1 second) between baseline and Day 14

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 63 participants
• Primary outcome timeframe: 14 days
• Results posted on: 2021-06-25

Participant Flow

1. subject screen failed due to intolerance to hypertonic saline 2. subjects failed xylitol trial inhalation

Participant milestones

Measure	7% Hypertonic Saline 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Overall Study STARTED	30	30
Overall Study COMPLETED	29	30
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	7% Hypertonic Saline 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

Aerosolized Hypertonic Xylitol Versus Hypertonic Saline in Cystic Fibrosis (CF) Subjects

Baseline characteristics by cohort

Measure	Hypertonic Saline n=29 Participants 7% hypertonic saline given twice daily for 14 days	Xylitol n=30 Participants Xylitol given twice daily for 14 days	Total n=59 Participants Total of all reporting groups
Age, Continuous	30.4 years STANDARD_DEVIATION 12.8 • n=5 Participants	29.7 years STANDARD_DEVIATION 12.3 • n=7 Participants	30.1 years STANDARD_DEVIATION 12.4 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants	15 Participants n=7 Participants	27 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants	15 Participants n=7 Participants	32 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	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	28 Participants n=5 Participants	30 Participants n=7 Participants	58 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Forced Expiratory volume % predicted	53.3 Percentage predicted STANDARD_DEVIATION 18.8 • n=5 Participants	58.8 Percentage predicted STANDARD_DEVIATION 20.1 • n=7 Participants	56.1 Percentage predicted STANDARD_DEVIATION 19.5 • n=5 Participants

PRIMARY outcome

Timeframe: 14 days

Change in lung function (forced expiratory volume in 1 second) between baseline and Day 14

Outcome measures

Measure	7% Hypertonic Saline n=29 Participants 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol n=30 Participants 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Change in FEV1 Percentage Predicted From Baseline	11.8 percentage of predicted Interval 8.1 to 15.5	8.8 percentage of predicted Interval 4.7 to 12.9

SECONDARY outcome

Timeframe: 14 days

Population: Subjects who were able to produce sputum at baseline and day 14

Mean difference from baseline of Sputum density expressed as log colony forming units between baseline and Day 14

Outcome measures

Measure	7% Hypertonic Saline n=18 Participants 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol n=22 Participants 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Density of Colonization of Pseudomonas Aeruginosa Per Gram of Sputum	-1.4 log CFU/ml Interval -2.8 to 0.1	-0.9 log CFU/ml Interval -2.3 to 0.4

SECONDARY outcome

Timeframe: 6 months

numbers of subjects that experienced an exacerbation during the 6 months follow-up

Outcome measures

Measure	7% Hypertonic Saline n=29 Participants 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol n=30 Participants 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Exacerbations During 6 Months Follow-up	15 Participants	11 Participants

Adverse Events

7% Hypertonic Saline

Serious events: 2 serious events

Other events: 11 other events

Deaths: 0 deaths

Hypertonic Xylitol

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	7% Hypertonic Saline n=29 participants at risk 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol n=30 participants at risk 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Gastrointestinal disorders Distal intestinal obstruction syndrome	6.9% 2/29 • Number of events 2 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	0.00% 0/30 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days

Other adverse events

Measure	7% Hypertonic Saline n=29 participants at risk 5 ml of 7% saline twice daily Saline: 7% hypertonic saline solution for aerosol; Dosage: 5 ml twice a day (BID)	Hypertonic Xylitol n=30 participants at risk 5 ml of 15% xylitol twice daily Xylitol: 15% xylitol solution for aerosol; Dosage: 5 ml twice a day (BID)
Hepatobiliary disorders abnormal laboratory values	17.2% 5/29 • Number of events 5 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	13.3% 4/30 • Number of events 4 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days
Respiratory, thoracic and mediastinal disorders bronchospasm	0.00% 0/29 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	6.7% 2/30 • Number of events 2 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days
Gastrointestinal disorders Bowel obstruction	10.3% 3/29 • Number of events 3 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	0.00% 0/30 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days
Respiratory, thoracic and mediastinal disorders Hemoptysis	6.9% 2/29 • Number of events 2 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	3.3% 1/30 • Number of events 1 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days
Vascular disorders deep vein thrombosis	0.00% 0/29 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	3.3% 1/30 • Number of events 1 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days
Respiratory, thoracic and mediastinal disorders flu	3.4% 1/29 • Number of events 1 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days	0.00% 0/30 • For the duration of the study treatment (up tp 14 days) and Up to 7 days after last study drug inhalation for a total of up to 21 days

Additional Information

Lakshmi Durairaj

University of Iowa

Phone: 3193537968

Email: lakshmi-durairaj@uiowa.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place