Trial Outcomes & Findings for Cystic Fibrosis and Endothelial Function: At Rest and During Exercise (NCT NCT01772758)
NCT ID: NCT01772758
Last Updated: 2019-06-20
Results Overview
Brachial artery FMD induced by reactive hyperemia assessed vascular endothelial function at baseline and several hours after treatment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
64 participants
Primary outcome timeframe
pre-treatment Baseline and 2-3 hours post-treatment
Results posted on
2019-06-20
Participant Flow
Participant milestones
| Measure |
Protocol 1: AOC
measurements at baseline and 2 hours following the antioxidant cocktail that is comprised of over the counter vitamins (vitamin C 1000mg, vitamin E 600 IU, and alpha lipoic acid 600 mg) that will be given in two doses, 30 minutes apart.
Vitamin C, 1000mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Vitamin E, 600IU: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Alpha Lipoic Acid, 600mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
|
Protocol 2: BH4 (5mg)
measurements at baseline and 3 hours following the single dose of 5mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Protocol 2: BH4 (20mg)
measurements at baseline and 3 hours following the single dose of 20mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Healthy Controls
measurements were done with no intervention
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
18
|
17
|
12
|
17
|
|
Overall Study
COMPLETED
|
18
|
17
|
12
|
17
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Cystic Fibrosis and Endothelial Function: At Rest and During Exercise
Baseline characteristics by cohort
| Measure |
Protocol 1: AOC
n=18 Participants
measurements at baseline and 2 hours following the antioxidant cocktail that is comprised of over the counter vitamins (vitamin C 1000mg, vitamin E 600 IU, and alpha lipoic acid 600 mg) that will be given in two doses, 30 minutes apart.
Vitamin C, 1000mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Vitamin E, 600IU: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Alpha Lipoic Acid, 600mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
|
Protocol 2: BH4 (5mg)
n=17 Participants
measurements at baseline and 3 hours following the single dose of 5mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Protocol 2: BH4 (20mg)
n=12 Participants
measurements at baseline and 3 hours following the single dose of 20mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Healthy Controls
n=17 Participants
baseline measurements were done with no intervention
|
Total
n=64 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
17.9 years
STANDARD_DEVIATION 7.5 • n=5 Participants
|
17 years
STANDARD_DEVIATION 7 • n=7 Participants
|
19 years
STANDARD_DEVIATION 8 • n=5 Participants
|
15.7 years
STANDARD_DEVIATION 5.2 • n=4 Participants
|
17.3 years
STANDARD_DEVIATION 7.0 • n=21 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
35 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
29 Participants
n=21 Participants
|
|
Height
|
158.6 cm
STANDARD_DEVIATION 14.2 • n=5 Participants
|
158.1 cm
STANDARD_DEVIATION 14.1 • n=7 Participants
|
162.1 cm
STANDARD_DEVIATION 11.1 • n=5 Participants
|
162.9 cm
STANDARD_DEVIATION 14.7 • n=4 Participants
|
160.3 cm
STANDARD_DEVIATION 14.0 • n=21 Participants
|
|
Weight
|
53.1 kg
STANDARD_DEVIATION 14.7 • n=5 Participants
|
53.0 kg
STANDARD_DEVIATION 14.6 • n=7 Participants
|
57.8 kg
STANDARD_DEVIATION 14.7 • n=5 Participants
|
52.5 kg
STANDARD_DEVIATION 16.5 • n=4 Participants
|
53.8 kg
STANDARD_DEVIATION 15.3 • n=21 Participants
|
PRIMARY outcome
Timeframe: pre-treatment Baseline and 2-3 hours post-treatmentPopulation: Participants included patients diagnosed with cystic fibrosis and healthy age-matched controls.
Brachial artery FMD induced by reactive hyperemia assessed vascular endothelial function at baseline and several hours after treatment.
Outcome measures
| Measure |
Protocol 1: AOC
n=18 Participants
measurements at baseline and 2 hours following the antioxidant cocktail that is comprised of over the counter vitamins (vitamin C 1000mg, vitamin E 600 IU, and alpha lipoic acid 600 mg) that will be given in two doses, 30 minutes apart.
Vitamin C, 1000mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Vitamin E, 600IU: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
Alpha Lipoic Acid, 600mg: Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID
|
Protocol 2: BH4 (5mg)
n=17 Participants
measurements at baseline and 3 hours following the single dose of 5mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Protocol 2: BH4 (20mg)
n=12 Participants
measurements at baseline and 3 hours following the single dose of 20mg/kg Kuvan® or sapropterin dihydrochloride which is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).BH4 has been shown in past studies to increase NO bioavailability.
BH4: Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects will receive an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)
|
Healthy Controls
n=17 Participants
measurements were done with no intervention
|
|---|---|---|---|---|
|
Percentage Flow-Mediated Dilation (FMD)
Pre treatment
|
5.64 percentage of change in FMD
Standard Deviation 2.80
|
5.32 percentage of change in FMD
Standard Deviation 3.53
|
6.29 percentage of change in FMD
Standard Deviation 3.21
|
7.21 percentage of change in FMD
Standard Deviation 3.17
|
|
Percentage Flow-Mediated Dilation (FMD)
Post treatment
|
7.58 percentage of change in FMD
Standard Deviation 4.16
|
4.81 percentage of change in FMD
Standard Deviation 2.83
|
7.39 percentage of change in FMD
Standard Deviation 3.41
|
—
|
Adverse Events
Antioxidant Cocktail: CF Patients
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Antioxidant Cocktail: Healthy Controls
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Tetrahydrobiopterin (BH4): 5mg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Tetrahydrobiopterin (BH4): 20mg
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