Trial Outcomes & Findings for Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) (NCT NCT02323100)
NCT ID: NCT02323100
Last Updated: 2026-04-17
Results Overview
Change in average measurement of nasal potential difference in millivolts (mV) between baseline (Visit 0) and Day 4 (Visit 2) or Day 7 (Visit 3). Note: the outcome is assessed as change in mV at each visit, and then comparisons between visits are made.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
16 participants
Primary outcome timeframe
7 days
Results posted on
2026-04-17
Participant Flow
Participant milestones
| Measure |
Ravicti Low Dose
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM 1.1 gm/ml
11 subjects started 9 subjects completed 2 subjects had a serious adverse event 0 subject deaths
|
Placebo
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM
5 subjects started 5 subjects completed 0 subjects had a serious adverse event 0 subject deaths
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
5
|
|
Overall Study
COMPLETED
|
9
|
5
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
Reasons for withdrawal
| Measure |
Ravicti Low Dose
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM 1.1 gm/ml
11 subjects started 9 subjects completed 2 subjects had a serious adverse event 0 subject deaths
|
Placebo
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM
5 subjects started 5 subjects completed 0 subjects had a serious adverse event 0 subject deaths
|
|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
Baseline Characteristics
Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
Baseline characteristics by cohort
| Measure |
Ravicti Low Dose
n=9 Participants
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM 1.1 gm/ml
9 subjects between 18-65
|
Placebo
n=5 Participants
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM
5 subjects between 18-65
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=130 Participants
|
5 Participants
n=132 Participants
|
14 Participants
n=130 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Age, Continuous
|
31.7 years
STANDARD_DEVIATION 11.9 • n=130 Participants
|
31.8 years
STANDARD_DEVIATION 9.36 • n=132 Participants
|
31.7142857 years
STANDARD_DEVIATION 10.3756155 • n=130 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=130 Participants
|
3 Participants
n=132 Participants
|
7 Participants
n=130 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=130 Participants
|
2 Participants
n=132 Participants
|
7 Participants
n=130 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
3 Participants
n=130 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=130 Participants
|
5 Participants
n=132 Participants
|
11 Participants
n=130 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=130 Participants
|
5 Participants
n=132 Participants
|
14 Participants
n=130 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=130 Participants
|
0 Participants
n=132 Participants
|
0 Participants
n=130 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=130 Participants
|
5 Participants
n=132 Participants
|
14 Participants
n=130 Participants
|
PRIMARY outcome
Timeframe: 7 daysChange in average measurement of nasal potential difference in millivolts (mV) between baseline (Visit 0) and Day 4 (Visit 2) or Day 7 (Visit 3). Note: the outcome is assessed as change in mV at each visit, and then comparisons between visits are made.
Outcome measures
| Measure |
Ravicti Low Dose
n=7 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Chloride and Sodium Transport in Nasal Epithelium
Day 4 minus Baseline
|
-6.20 mV
Standard Error 3.54
|
-1.56 mV
Standard Error 4.19
|
|
Change in Chloride and Sodium Transport in Nasal Epithelium
Day 7 minus Baseline
|
2.22 mV
Standard Error 3.54
|
-1.55 mV
Standard Error 4.53
|
SECONDARY outcome
Timeframe: 7 days of treatment as measured on Day 1 (Visit 1) and Day 7 (Visit 3)Change in average sweat chloride measurement between baseline Day 1 (Visit 1) and Day 7 (Visit 3).
Outcome measures
| Measure |
Ravicti Low Dose
n=8 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Average Sweat Chloride
|
-1.8 mmol/L
Standard Error 1.9
|
0.5 mmol/L
Standard Error 2.5
|
SECONDARY outcome
Timeframe: 4 days and 7 days of study drug treatmentChange in baseline nasal potential difference (NPD) measured before initiation of study drug between Day 1 (Visit 1) and Day 4 (Visit 2) and between Day 1 (Visit 1) and Day 7 (Visit 3).
Outcome measures
| Measure |
Ravicti Low Dose
n=7 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Baseline Nasal Potential Difference (NPD)
Baseline PD, V1 to V2
|
-1.12 mV
Standard Error 3.95
|
2.20 mV
Standard Error 4.68
|
|
Change in Baseline Nasal Potential Difference (NPD)
Baseline PD, V1 to V3
|
-0.53 mV
Standard Error 3.95
|
11.76 mV
Standard Error 5.05
|
SECONDARY outcome
Timeframe: 4 and 7 daysPopulation: Ravicti high dose was ultimately not tested in the clinical trial.
Change in the nasal potential difference response (in mV) with perfusion of a solution containing amiloride from baseline to Days 4 and 7.
Outcome measures
| Measure |
Ravicti Low Dose
n=7 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Nasal Potential Difference With Perfusion of a Solution Containing Amiloride
Amiloride, V0 to V2
|
-6.74 mV
Standard Error 3.74
|
-1.82 mV
Standard Error 4.43
|
|
Change in Nasal Potential Difference With Perfusion of a Solution Containing Amiloride
Amiloride, V0 to V3
|
-5.32 mV
Standard Error 3.74
|
-10.67 mV
Standard Error 4.79
|
SECONDARY outcome
Timeframe: 4 and 7 daysChange in nasal potential difference (in mV) with perfusion of a low chloride solution containing amiloride from baseline to Days 4 and 7.
Outcome measures
| Measure |
Ravicti Low Dose
n=7 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Nasal Potential Difference During Perfusion With a Low Chloride Solution Containing Amiloride
Low chloride, V0 to V2
|
-5.14 mV
Standard Error 2.74
|
-0.97 mV
Standard Error 3.24
|
|
Change in Nasal Potential Difference During Perfusion With a Low Chloride Solution Containing Amiloride
Low chloride, V0 to V3
|
0.20 mV
Standard Error 2.74
|
0.70 mV
Standard Error 3.50
|
SECONDARY outcome
Timeframe: 4 and 7 daysChange in Nasal Potential Difference (in mV} with perfusion of a low chloride solution (containing amiloride) followed by the low chloride solution containing amiloride and Isoproterenol from baseline to Days 4 and 7.
Outcome measures
| Measure |
Ravicti Low Dose
n=7 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in Nasal Potential Difference With a Low Chloride Solution Containing Amiloride Plus the Change With a Low Chloride Solution Containing Amiloride and Isoproterenol
Low chloride plus isoproterenol, V0 to V2
|
-1.06 mV
Standard Error 1.55
|
-0.59 mV
Standard Error 1.83
|
|
Change in Nasal Potential Difference With a Low Chloride Solution Containing Amiloride Plus the Change With a Low Chloride Solution Containing Amiloride and Isoproterenol
Low chloride plus isoproterenol, V0 to V3
|
2.01 mV
Standard Error 1.55
|
-2.25 mV
Standard Error 1.98
|
SECONDARY outcome
Timeframe: 7 daysChange in FEV1 from baseline to Days 4 and 7.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Change in FEV1
FEV1, V0 to V2
|
1.9 Liters
Standard Error 1.3
|
-1.0 Liters
Standard Error 1.7
|
|
Change in FEV1
FEV1, V0 to V3
|
1.7 Liters
Standard Error 1.4
|
-0.4 Liters
Standard Error 1.9
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Electrolytes are presented in meq/L. Mean and SD reported at Screening and V4.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Electrolytes (Meq/L)
Sodium (meq/l), Screening
|
136.67 meq/L
Standard Deviation 2.00
|
137.20 meq/L
Standard Deviation 1.79
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Sodium (meq/l), 14 days
|
136.50 meq/L
Standard Deviation 2.88
|
137.20 meq/L
Standard Deviation 0.84
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Potassium (meq/l), Screening
|
4.08 meq/L
Standard Deviation 0.40
|
4.10 meq/L
Standard Deviation 0.42
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Potassium (meq/l), 14 days
|
4.00 meq/L
Standard Deviation 0.27
|
4.18 meq/L
Standard Deviation 0.40
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Chloride (meq/l), Screening
|
103.78 meq/L
Standard Deviation 1.72
|
103.80 meq/L
Standard Deviation 1.92
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Chloride (meq/l), 14 days
|
104.00 meq/L
Standard Deviation 2.73
|
104.80 meq/L
Standard Deviation 1.30
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Bicarbonate/CO2 (meq/l), Screening
|
23.78 meq/L
Standard Deviation 2.49
|
25.60 meq/L
Standard Deviation 1.95
|
|
Safety and Tolerability - Electrolytes (Meq/L)
Bicarbonate/CO2 (meq/l), 14 days
|
24.50 meq/L
Standard Deviation 4.07
|
25.20 meq/L
Standard Deviation 1.10
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Total Bilirubin on a comprehensive metabolic panel obtained from a serum sample and reported in mg/dl.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability-Liver Function-Total Bilirubin
Total Bilirubin (mg/dl), Screening
|
0.60 mg/dl
Standard Deviation 0.32
|
0.44 mg/dl
Standard Deviation 0.11
|
|
Safety and Tolerability-Liver Function-Total Bilirubin
Total Bilirubin (mg/dl), 14 days
|
0.55 mg/dl
Standard Deviation 0.26
|
0.36 mg/dl
Standard Deviation 0.05
|
SECONDARY outcome
Timeframe: 14 daysPopulation: Two participants, one from Drug and one from Placebo groups failed to return on day 14 and those data are missing.
Hepatic enzymes obtained from a serum sample -AST and ALT in units/L.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Liver Function-Liver Enzymes
AST (units/L), Screening
|
20.56 units/L
Standard Deviation 6.33
|
19.40 units/L
Standard Deviation 8.02
|
|
Safety and Tolerability - Liver Function-Liver Enzymes
AST (units/L), 14 days
|
20.88 units/L
Standard Deviation 10.32
|
20.00 units/L
Standard Deviation 9.14
|
|
Safety and Tolerability - Liver Function-Liver Enzymes
ALT (units/L), Screening
|
24.33 units/L
Standard Deviation 12.82
|
20.40 units/L
Standard Deviation 7.60
|
|
Safety and Tolerability - Liver Function-Liver Enzymes
ALT (units/L), 14 days
|
25.13 units/L
Standard Deviation 17.79
|
20.80 units/L
Standard Deviation 9.91
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Alkaline Phosphatase obtained on a Comprehensive Metabolic Panel (IU/L).
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Liver Function-Alkaline Phosphatase (IU/L)
Alkaline Phosphatase (IU/L), Screening
|
106.11 IU/L
Standard Deviation 37.30
|
72.20 IU/L
Standard Deviation 27.43
|
|
Safety and Tolerability - Liver Function-Alkaline Phosphatase (IU/L)
Alkaline Phosphatase (IU/L), 14 days
|
89.38 IU/L
Standard Deviation 24.25
|
75.60 IU/L
Standard Deviation 34.11
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Safety and Tolerability - serum protein biomarkers in g/dl.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Total Protein (g/dl), Screening
|
7.12 g/dl
Standard Deviation 0.49
|
6.76 g/dl
Standard Deviation 0.25
|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Total Protein (g/dl), 14 days
|
6.95 g/dl
Standard Deviation 0.4
|
6.64 g/dl
Standard Deviation 0.36
|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Hemoglobin (g/dl), Screening
|
14.29 g/dl
Standard Deviation 0.93
|
13.94 g/dl
Standard Deviation 1.21
|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Hemoglobin (g/dl), 14 days
|
14.03 g/dl
Standard Deviation 1.32
|
13.78 g/dl
Standard Deviation 1.40
|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Albumin (g/dl), Screening
|
4.32 g/dl
Standard Deviation 0.25
|
4.28 g/dl
Standard Deviation 0.30
|
|
Safety and Tolerability - Blood Protein Biomarkers (g/dl)
Albumin (g/dl), 14 days
|
3.98 g/dl
Standard Deviation 1.18
|
4.24 g/dl
Standard Deviation 0.44
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
White blood cells, red blood cells, and platelet cell counts from a complete blood cell count panel (K/ul).
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
White blood cell count (K/μL), Screening
|
7.73 K/uL
Standard Deviation 2.01
|
6.66 K/uL
Standard Deviation 2.38
|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
White blood cell count (K/μL), 14 days
|
7.03 K/uL
Standard Deviation 1.41
|
6.12 K/uL
Standard Deviation 1.67
|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
Red blood cell count (K/μL), Screening
|
4.86 K/uL
Standard Deviation 0.44
|
4.68 K/uL
Standard Deviation 0.38
|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
Red blood cell count (K/μL), 14 days
|
4.79 K/uL
Standard Deviation 0.43
|
4.60 K/uL
Standard Deviation 0.37
|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
Platelet count (ct K/ul), Screening
|
283.33 K/uL
Standard Deviation 54.94
|
269.00 K/uL
Standard Deviation 49.51
|
|
Safety and Tolerability - Blood Cell Counts (K/ul)
Platelet count (ct K/uL), 14 days
|
267.38 K/uL
Standard Deviation 77.32
|
264.0 K/uL
Standard Deviation 58.03
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
White blood cells are fractionated into different types of white blood cells and presented as the fraction of total white blood cells counted.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Blood Cell Counts (%)
Eosinophils (% white blood cells counted), 14 days
|
2.06 % of blood cells counted
Standard Deviation 1.38
|
2.62 % of blood cells counted
Standard Deviation 0.61
|
|
Safety and Tolerability - Blood Cell Counts (%)
Basophils (% white blood cells counted), Screening
|
0.47 % of blood cells counted
Standard Deviation 0.21
|
0.48 % of blood cells counted
Standard Deviation 0.22
|
|
Safety and Tolerability - Blood Cell Counts (%)
Basophils (%) white blood cells counted, 14 days
|
0.49 % of blood cells counted
Standard Deviation 0.32
|
0.54 % of blood cells counted
Standard Deviation 0.09
|
|
Safety and Tolerability - Blood Cell Counts (%)
Neutrophils (% white blood cells counted), Screening
|
63.58 % of blood cells counted
Standard Deviation 10.35
|
55.92 % of blood cells counted
Standard Deviation 12.33
|
|
Safety and Tolerability - Blood Cell Counts (%)
Neutrophils (% white blood cells counted), 14 days
|
60.33 % of blood cells counted
Standard Deviation 11.67
|
61.04 % of blood cells counted
Standard Deviation 6.37
|
|
Safety and Tolerability - Blood Cell Counts (%)
Lymphocytes (% white blood cells counted), Screening
|
26.60 % of blood cells counted
Standard Deviation 9.68
|
32.96 % of blood cells counted
Standard Deviation 10.64
|
|
Safety and Tolerability - Blood Cell Counts (%)
Lymphocytes (% white blood cell counted), 14 days
|
29.63 % of blood cells counted
Standard Deviation 10.04
|
27.84 % of blood cells counted
Standard Deviation 6.17
|
|
Safety and Tolerability - Blood Cell Counts (%)
Monocytes (% white blood cells counted), Screening
|
7.24 % of blood cells counted
Standard Deviation 1.54
|
8.32 % of blood cells counted
Standard Deviation 2.22
|
|
Safety and Tolerability - Blood Cell Counts (%)
Monocytes (% white blood cells counted), 14 days
|
7.51 % of blood cells counted
Standard Deviation 1.58
|
7.94 % of blood cells counted
Standard Deviation 1.94
|
|
Safety and Tolerability - Blood Cell Counts (%)
Eosinophils (% white blood cells counted), Screening
|
2.10 % of blood cells counted
Standard Deviation 2.19
|
2.32 % of blood cells counted
Standard Deviation 0.67
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant in the Drug group failed to have an ESR on screening, and one participant Drug failed to return on day 14 and those data are missing.
Erythrocyte sedimentation rate is an indicator of inflammation that measures the rate at which red blood cells settle to the bottom of a test tube in mm/hr.
Outcome measures
| Measure |
Ravicti Low Dose
n=8 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Erythrocyte Sedimentation Rate (mm/hr)
ESR (mm/hr), Screening
|
11.13 mm/hr
Standard Deviation 10.93
|
10.80 mm/hr
Standard Deviation 10.85
|
|
Safety and Tolerability - Erythrocyte Sedimentation Rate (mm/hr)
ESR (mm/hr), 14 days
|
12.25 mm/hr
Standard Deviation 13.40
|
16.40 mm/hr
Standard Deviation 17.95
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Serum glucose was obtained in a comprehensive metabolic panel in mg/dl.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Serum Glucose (mg/dl)
Glucose (mg/dl), Screening
|
100.67 mg/dl
Standard Deviation 16.24
|
82.60 mg/dl
Standard Deviation 6.19
|
|
Safety and Tolerability - Serum Glucose (mg/dl)
Glucose (mg/dl), 14 days
|
125.88 mg/dl
Standard Deviation 111.43
|
87.80 mg/dl
Standard Deviation 14.38
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Blood urea nitrogen (BUN), serum creatinine, and serum uric acid were obtained in a comprehensive metabolic panel in mg/dl.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Urea Nitrogen (BUN) (mg/dl), Screening
|
16.67 mg/dl
Standard Deviation 5.96
|
16.20 mg/dl
Standard Deviation 2.49
|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Urea Nitrogen (mg/dl), 14 days
|
14.38 mg/dl
Standard Deviation 2.39
|
16.20 mg/dl
Standard Deviation 3.03
|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Creatinine (mg/dl), Screening
|
0.83 mg/dl
Standard Deviation 0.16
|
0.80 mg/dl
Standard Deviation 0.12
|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Creatiniine (mg/dl), 14 days
|
0.79 mg/dl
Standard Deviation 0.10
|
0.80 mg/dl
Standard Deviation 0.16
|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Serum Uric Acid (mg/dl), Screening
|
7.30 mg/dl
Standard Deviation 1.26
|
6.26 mg/dl
Standard Deviation 1.24
|
|
Safety and Tolerability - Blood Waste Metabolites (mg/dl)
Serum Uric Acid (mg/dl), 14 days
|
7.06 mg/dl
Standard Deviation 1.36
|
5.78 mg/dl
Standard Deviation 1.09
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
C reactive protein (cRP) is a systemic marker of inflammation and was measured in mg/dl.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Blood Biomarker of Inflammation (mg/dl)
C-reactive protein (mg/dl), Screening
|
0.42 mg/dl
Standard Deviation 0.49
|
0.35 mg/dl
Standard Deviation 0.34
|
|
Safety and Tolerability - Blood Biomarker of Inflammation (mg/dl)
C-reactive protein (mg/dl), 14 days
|
0.95 mg/dl
Standard Deviation 1.39
|
0.36 mg/dl
Standard Deviation 0.34
|
SECONDARY outcome
Timeframe: 14 daysPopulation: One participant from Drug failed to return on day 14 and those data are missing.
Hematocrit is resulted as the fraction of red blood cells in total volume of blood from which they are measured.
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Safety and Tolerability - Hematocrit (%)
Hematocrit (% packed red blood cell volume), Screening
|
42.71 % of packed red blood cell volume
Standard Deviation 2.60
|
42.42 % of packed red blood cell volume
Standard Deviation 3.99
|
|
Safety and Tolerability - Hematocrit (%)
Hematocrit (% packed red blood cell volume), 14 days
|
41.56 % of packed red blood cell volume
Standard Deviation 3.34
|
41.72 % of packed red blood cell volume
Standard Deviation 3.82
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 4 daysConcentration of PBA and PAA in blood over time
Outcome measures
| Measure |
Ravicti Low Dose
n=9 Participants
Low dose Ravicti® oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Ravicti low dose: 8 am, 4pm and midnight
|
Placebo
n=5 Participants
Matching placebo taken at 8am, 4pm and midnight for 7 days.
Placebo: 8 am, 4pm and midnight
|
|---|---|---|
|
Pharmacokinetics (PK) Studies
PBA, Day 1 Hr 2 - Day 1 Baseline
|
42.6 micrograms/ml
Standard Error 8.5
|
NA micrograms/ml
Standard Error NA
All data below detectable limits
|
|
Pharmacokinetics (PK) Studies
PBA, Day 4 Hr 2 - Day 1 Baseline
|
43.7 micrograms/ml
Standard Error 9.8
|
NA micrograms/ml
Standard Error NA
All data below detectable limits
|
|
Pharmacokinetics (PK) Studies
PAA, Day 1 Hr 4 - Day 1 Baseline
|
26.1 micrograms/ml
Standard Error 4.3
|
NA micrograms/ml
Standard Error NA
All data below detectable limits
|
|
Pharmacokinetics (PK) Studies
PAA, Day 4 Hr 4 - Day 1 Baseline
|
57.6 micrograms/ml
Standard Error 10.3
|
NA micrograms/ml
Standard Error NA
All data below detectable limits
|
Adverse Events
Ravicti Low Dose
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Ravicti Low Dose
n=9 participants at risk
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM 1.1 gm/ml
9 subject were at risk and 3 subjects had an adverse event. Adverse events were headache, brain fog, nausea/vomiting/flatulence, and new sputum bacteria.
|
Placebo
n=5 participants at risk
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM
5 subject were at risk and 4 subjects had an adverse event. Adverse events were headache, brain fog, nausea/vomiting/flatulence, and new sputum bacteria.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Cystic Fibrosis Pulmonary Exacerbation
|
11.1%
1/9 • 14 days
|
0.00%
0/5 • 14 days
|
|
Infections and infestations
Covid-19
|
11.1%
1/9 • 14 days
|
0.00%
0/5 • 14 days
|
Other adverse events
| Measure |
Ravicti Low Dose
n=9 participants at risk
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM 1.1 gm/ml
9 subject were at risk and 3 subjects had an adverse event. Adverse events were headache, brain fog, nausea/vomiting/flatulence, and new sputum bacteria.
|
Placebo
n=5 participants at risk
6.0 ml po 8 AM, 6.05 ml 4 PM, 6.05 ml 12 AM
5 subject were at risk and 4 subjects had an adverse event. Adverse events were headache, brain fog, nausea/vomiting/flatulence, and new sputum bacteria.
|
|---|---|---|
|
Nervous system disorders
headache
|
11.1%
1/9 • 14 days
|
40.0%
2/5 • 14 days
|
|
Nervous system disorders
brain fog
|
0.00%
0/9 • 14 days
|
20.0%
1/5 • 14 days
|
|
Gastrointestinal disorders
nausea, vomiting, flatulence
|
11.1%
1/9 • 14 days
|
20.0%
1/5 • 14 days
|
|
Infections and infestations
new sputum bacteria
|
0.00%
0/9 • 14 days
|
40.0%
2/5 • 14 days
|
Additional Information
Pamela L. Zeitlin, Chair, Department of Pediatrics
National Jewish Health
Phone: 303-398-1196
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place