Trial Outcomes & Findings for Effect of Hypertonic Saline on Mucus Clearance in Children Ages 5-12 With Cystic Fibrosis (NCT NCT01031706)

Last Updated: 2017-03-03

Results Overview

Average radio tracer clearance through 90 minutes (MCC90) is primary index of mucociliary clearance at each study. Primary study outcome: is absolute change in MCC90 between baseline and at end of treatment (where MCC measured 8-12 hours after final dose of study drug) - reflects sustained impact on MCC

Recruitment status

COMPLETED

Study phase

Target enrollment

23 participants

Primary outcome timeframe

Baseline versus after completion of 4 week treatment period

Results posted on

2017-03-03

Participant Flow

Participant milestones

Participant milestones
Measure	Hypertonic Saline 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days
Overall Study STARTED	14	9
Overall Study COMPLETED	12	8
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Hypertonic Saline 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days
Overall Study Adverse Event	1	0
Overall Study Withdrawal by Subject	1	1

Baseline Characteristics

Effect of Hypertonic Saline on Mucus Clearance in Children Ages 5-12 With Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hypertonic Saline n=14 Participants 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo n=9 Participants Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days	Total n=23 Participants Total of all reporting groups
Age, Continuous	11.9 years STANDARD_DEVIATION 3.3 • n=5 Participants	9.8 years STANDARD_DEVIATION 3.6 • n=7 Participants	11.1 years STANDARD_DEVIATION 3.5 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	4 Participants n=7 Participants	12 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline versus after completion of 4 week treatment period

Population: All subjects with available data analyzed

Outcome measures

Outcome measures
Measure	Hypertonic Saline n=12 Participants 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo n=8 Participants Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days
Change in Mucociliary Clearance Rate	2.77 percent clearance Standard Error .98	-2.35 percent clearance Standard Error 1.82

SECONDARY outcome

Timeframe: Baseline and after 4 weeks of treatment

Population: All available data included. Carry-forward of last FEV1 value (after 2 weeks of treatment) performed when data at 4 week time point not available

Absolute change in % predicted FEV1 between baseline and after 4 weeks of treatment calculated

Outcome measures

Outcome measures
Measure	Hypertonic Saline n=13 Participants 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo n=8 Participants Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days
FEV1 (Spirometry) Change	3.38 Percentage of predicted FEV1 Standard Error 2.01	1.09 Percentage of predicted FEV1 Standard Error 3.19

Adverse Events

Hypertonic Saline

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Dr. Scott H. Donaldson

University of North Carolina at Chapel Hill

Phone: 919-966-9198

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Hypertonic Saline n=14 participants at risk 6% NaCl, 4 ml TID via eFlow Hypertonic Saline: inhaled HS (6% NaCl, 4mL) three times a day for 28 days	Placebo n=9 participants at risk Placebo: 4 ml 0.12% NaCl inhaled three times a day x 28 days
Respiratory, thoracic and mediastinal disorders Cough during study drug inhalation	42.9% 6/14 • Number of events 6 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	22.2% 2/9 • Number of events 2 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Increased Cough	50.0% 7/14 • Number of events 10 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	66.7% 6/9 • Number of events 7 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Increased sputum production	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders wheezing	0.00% 0/14 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Cystic Fibrosis exacerbation	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Hemoptysis	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Dyspnea	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders New Pulmonary Pathogen	14.3% 2/14 • Number of events 2 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Gastrointestinal disorders Emesis	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Gastrointestinal disorders Abdominal pain	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Gastrointestinal disorders Anorexia	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Gastrointestinal disorders worsened steatorhea	14.3% 2/14 • Number of events 3 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Gastrointestinal disorders nausea	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
General disorders fever	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Musculoskeletal and connective tissue disorders Knee pain	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
General disorders Fatigue	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	22.2% 2/9 • Number of events 3 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Nervous system disorders headache	0.00% 0/14 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
General disorders Insomnia	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
General disorders Hyperactivity	0.00% 0/14 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders Pharynx irritation	14.3% 2/14 • Number of events 2 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Ear and labyrinth disorders Otalgia	7.1% 1/14 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	0.00% 0/9 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders nasal/sinus congestions	14.3% 2/14 • Number of events 2 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	33.3% 3/9 • Number of events 3 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.
Respiratory, thoracic and mediastinal disorders sinus pain	0.00% 0/14 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.	11.1% 1/9 • Number of events 1 • 1 month All events between screening and end of treatment recorded. At start of each visit, subject is asked whether they have experienced any new symptoms or problems since the prior visit.