Trial Outcomes & Findings for Effectiveness of Arginine as a Treatment for Sickle Cell Anemia (NCT NCT00513617)
NCT ID: NCT00513617
Last Updated: 2017-03-29
Results Overview
Gardos channel activity: a calcium (Ca2+)-activated K+ channel
COMPLETED
PHASE2
128 participants
12 weeks after randomization
2017-03-29
Participant Flow
Enrolled subjects at participating sites from May 2004 through July 2007. Sites consisted of sickle cell treatment centers from across the United States.
All subjects were to be without hydroxyurea, transfusion, and arginine for 90 days prior to enrollment. Prior to randomization, blood was drawn for baseline efficacy and safety measurements.
Participant milestones
| Measure |
Low Dose
0.05 g/kg/day of Arginine in capsule form
|
High Dose
0.10 g/kg/day of Arginine in capsule form
|
Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
36
|
35
|
38
|
|
Overall Study
COMPLETED
|
25
|
25
|
30
|
|
Overall Study
NOT COMPLETED
|
11
|
10
|
8
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Baseline characteristics by cohort
| Measure |
Low Dose
n=36 Participants
0.05 g/kg/day of Arginine in capsule form
|
High Dose
n=35 Participants
0.10 g/kg/day of Arginine in capsule form
|
Placebo
n=38 Participants
|
Total
n=109 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
14 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
50 Participants
n=157 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
22 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
59 Participants
n=157 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
|
Age, Continuous
|
24.5 years
STANDARD_DEVIATION 12.85 • n=99 Participants
|
20.0 years
STANDARD_DEVIATION 10.01 • n=107 Participants
|
21.0 years
STANDARD_DEVIATION 11.49 • n=206 Participants
|
23.2 years
STANDARD_DEVIATION 11.75 • n=157 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
60 Participants
n=157 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=99 Participants
|
16 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
49 Participants
n=157 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=99 Participants
|
35 participants
n=107 Participants
|
38 participants
n=206 Participants
|
109.0 participants
n=157 Participants
|
|
Genotype of SCD
Sickle cell S-Beta Thalassemia (SB0)
|
2 participants
n=99 Participants
|
3 participants
n=107 Participants
|
2 participants
n=206 Participants
|
7.0 participants
n=157 Participants
|
|
Genotype of SCD
Sickle cell Anemia (SS)
|
34 participants
n=99 Participants
|
32 participants
n=107 Participants
|
36 participants
n=206 Participants
|
102.0 participants
n=157 Participants
|
PRIMARY outcome
Timeframe: 12 weeks after randomizationPopulation: All subjects that were randomized and dosed - Intent to Treat (ITT) No imputation used.
Gardos channel activity: a calcium (Ca2+)-activated K+ channel
Outcome measures
| Measure |
Low Dose
n=12 Participants
0.05 g/kg/day of Arginine in capsule form
|
High Dose
n=11 Participants
0.10 g/kg/day of Arginine in capsule form
|
Placebo
n=14 Participants
|
|---|---|---|---|
|
Gardos Channel Activity
|
-0.0342 mmol/10^13 cells x min
Standard Deviation 0.2341
|
0.0043 mmol/10^13 cells x min
Standard Deviation 0.3028
|
0.1076 mmol/10^13 cells x min
Standard Deviation 0.2822
|
PRIMARY outcome
Timeframe: 12 weeks after randomizationPopulation: All subjects that were randomized and dosed - ITT No imputation used.
Nitric oxide from plasma amino acids
Outcome measures
| Measure |
Low Dose
n=12 Participants
0.05 g/kg/day of Arginine in capsule form
|
High Dose
n=11 Participants
0.10 g/kg/day of Arginine in capsule form
|
Placebo
n=14 Participants
|
|---|---|---|---|
|
Nitric Oxide
|
-3.8697 uM
Standard Deviation 15.7574
|
2.9250 uM
Standard Deviation 11.1646
|
-3.0265 uM
Standard Deviation 18.6887
|
PRIMARY outcome
Timeframe: 12 weeks after randomizationMean corpuscular hemoglobin concentration as measured by an Advia machine
Outcome measures
| Measure |
Low Dose
n=12 Participants
0.05 g/kg/day of Arginine in capsule form
|
High Dose
n=11 Participants
0.10 g/kg/day of Arginine in capsule form
|
Placebo
n=14 Participants
|
|---|---|---|---|
|
Mean Corpuscular Hemoglobin Concentration
|
-1.8485 g/dL
Standard Deviation 6.3092
|
0.7692 g/dL
Standard Deviation 2.2165
|
-0.0705 g/dL
Standard Deviation 1.4422
|
SECONDARY outcome
Timeframe: 12 weeks after randomizationSoluble vascular cell adhesion molecule (sVCAM) a vascular adhesion molecule
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeks after randomization8-iso-PGF2a is a measure of lipid peroxidation and oxidative damage in vivo measured by enzyme immunoassay kit from Cayman chemical
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeks after randomizationEndothelin-1 is a potent vasoconstrictor and pro-inflammatory agent which is elevated in SCD patients
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeks after randomizationFetal hemoglobin (HbF) as measured by the Advia machine
Outcome measures
Outcome data not reported
Adverse Events
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