Anemia, Sickle Cell
Conditions
Keywords
Antioxidant, Inflammation, Sickle Cell Disease, Oxidative Stress, Cytokines, anti-inflammatory, Acetylcarnitine
Brief summary
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the frequency of vaso-occlusive pain episodes and improve their quality of life.
Detailed description
People with sickle cell disease have more inflammation (a response of body tissues to injury or irritation) than people without sickle cell disease. This inflammation can be measured in the blood by checking the level of a protein called C reactive protein as well as other changes we see in blood due to inflammation (such as changes in platelets and other cells). There is even more inflammation during sickle-related complications (like pain or acute chest syndrome). We want to test if inflammation in people with sickle cell disease can be reduced by the use of antioxidant compounds. Antioxidants are nutrients (certain vitamins, minerals and enzymes) that can counteract the effects of oxidative stress arising from free radicals in our cells. The formation of free radicals is a normal cell process, but uncontrolled oxidative stress can cause problems for us. One such harmful problem is inflammation. We know from other research studies that antioxidants help with some conditions related to inflammation. In this study the antioxidant being tested is a combination of alpha-lipoic acid and acetyl-L-carnitine, both of which are natural parts of many of the foods we eat and are needed by our cells to make energy from food.
Interventions
DRUGalpha-lipoic acid and acetyl-L-carnitine
none to report
DRUGControl
none to report
Sponsors
National Center for Complementary and Integrative Health (NCCIH)
UCSF Benioff Children's Hospital Oakland
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Proven diagnosis of sickle cell disease, either homozygous sickle disease or Hb S Beta zero thalassemia genotype * Age at entry at least 14 years. Younger children will not be included since the combination alpha-lipoic acid and acetyl-L-carnitine tablets are not available in a smaller dose at this time.
Exclusion criteria
* More than 3 packed red blood transfusions in the past 12 months * Coexisting illness that could contribute to inflammation. These include chronic hepatitis, lupus, arthritis, inflammatory bowel disease, chronic osteomyelitis, and other similar conditions. * Acute sickle cell disease related symptoms requiring a hospital visit in the past 4 weeks * Women who are pregnant, attempting to get pregnant, or breast feeding * Active participation in other investigational drug or device studies * Participants who start hydroxyurea or regular transfusion therapy during the course of the study on the recommendation of their primary hematologist will be ineligible for further participation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| C-Reactive Protein | 6 months | C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states. |
Countries
United States
Participant flow
Recruitment details
First subject was enrolled 08/10/09. Length of subject involvement was 7 months. Each subject was seen once a month at Children's Hospital & Research Center Oakland
Pre-assignment details
Exclusions included those with 3 packed red blood transfusions in the past year, illness that could contribute to inflammation, acute sickle cell disease related symptoms requiring a hospital visit, those pregnant or breast feeding, or in other investigational drug or device studies, participants on hydroxyurea or a regular transfusion therapy.
Participants by arm
| Arm | Count |
|---|---|
| Alpha-lipoic Acid and Acetyl-L-carnitine alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months.
alpha-lipoic acid and acetyl-L-carnitine: none to report | 17 |
| Placebo 1400 mg placebo tablet twice a day for 6 months.
Placebo: none to report | 20 |
| Total | 37 |
Baseline characteristics
| Characteristic | Alpha-lipoic Acid and Acetyl-L-carnitine | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 1 Participants | 4 Participants | 5 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 16 Participants | 16 Participants | 32 Participants |
| Sex: Female, Male Female | 7 Participants | 8 Participants | 15 Participants |
| Sex: Female, Male Male | 10 Participants | 12 Participants | 22 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 17 | 0 / 20 |
| other Total, other adverse events | 6 / 17 | 12 / 20 |
| serious Total, serious adverse events | 8 / 17 | 9 / 20 |
Outcome results
Primary
C-Reactive Protein
C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states.
Time frame: 6 months
Population: Reason for discontinuation: started transfusions (2), alternative treatment (2), adverse events (4), subject preference (3).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alpha-lipoic Acid and Acetyl-L-carnitine | C-Reactive Protein | 11.4 mg/l | Standard Deviation 20.9 |
| Placebo | C-Reactive Protein | 6.8 mg/l | Standard Deviation 10.3 |