Beta Thalassemia
Conditions
Keywords
NAC, ALA
Brief summary
The current study is to investigate the potential roles of N-acetyl cysteine and Alpha-lipoic acid in patients with beta-thalassemia.
Detailed description
Beta-thalassemia (β-thalassemia) is a hereditary blood disorder, which is characterized by a genetic disorder in the production of β-globin chains. β-thalassemia is inherited mainly by an autosomal recessive manner resulting in reduced synthesis or absence of β-globin chains, leading to ineffective erythropoiesis and chronic hemolytic anemia. It is classified according to the severity into major, intermedia and minor. This is a randomized, parallel, clinical study that will be conducted on sixty-six patients with beta-thalassemia. The study duration will be 12 weeks. Patients will be divided into three groups as follows: Group I (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) only. Group II (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) plus NAC (600 mg orally once daily) for three months. Group III (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) plus ALA (600 mg orally once daily) for three months. The study will be approved by the Research Ethical Committee at Faculty of Pharmacy, Tanta University. All participants will be informed about benefits and risks of the study. The privacy of all participants will be respected and the data of enrolled participants will be confidential. All participants will sign their written informed consent.
Interventions
JADENU is indicated for the treatment of chronic iron overload due to blood transfusions.
N-acetyl cysteine (NAC) 600 mg will be administered orally once daily for 12 weeks.
Alpha lipoic acid (ALA) 600 mg will be administered orally once daily for 12 weeks.
Sponsors
Tanta University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Intervention model description
This is a randomized, parallel, clinical study that will be conducted on 66 patients with beta-thalassemia who fulfill the selection criteria and will be classified randomly into 3 groups as follows: Group I (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) only. Group II (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) plus NAC (600 mg orally once daily) for three months. Group III (n = 22): This group will include twenty-two patients with beta-thalassemia who will receive conventional thalassemia management (iron chelating agent) plus ALA (600 mg orally once daily) for three months.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Patients with beta-thalassemia who will receive conventional thalassemia management. * Both genders. * Age ≥ 18 years old.
Exclusion criteria
* Patients with familial hypercholesterolemia or history of premature atherosclerosis. * Patients with a prior history of significant cardiovascular diseases, such as coronary artery disease, myocardial infarction, or stroke. * Patients with severe renal dysfunction. * Patients with severe hepatic dysfunction. * Patients with diabetes. * Patients who will be non-compliant with the prescribed therapy. * Patients with other hemoglobinopathies. * Pregnant women. * Obese patients. * Patients who will receive antioxidant or anti-inflammatory medications. * Patients with inflammatory diseases, such as Systemic lupus erythematous, rheumatoid arthritis and inflammatory bowel disease. * Patients with oxidative stress related diseases, such as Alzheimer, Parkinson, COPD and cancer.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The change from baseline in carotid intima media thickness (CIMT) | 3 months | Non-invasive imaging technique, such as carotid ultrasonography will be used to measure carotid intima media thickness |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Malondialdehyde (MDA) | 3 months | Malondialdehyde (MDA) level will be assessed using commercially available kit |
| Change in high sensitivity C-reactive protein (hs-CRP) | 3 months | High sensitivity C-reactive protein (hs-CRP) level will be assessed using commercially available kit. |
| Change in asymmetric dimethyl arginine (ADMA) | 3 months | Asymmetric dimethyl arginine (ADMA) level will be assessed using commercially available kit. |
| Change in lipid profile | 3 months | HDL, total cholesterol and triglycerides will be assessed using commercially available kits. |
Countries
Egypt
Contacts
Primary ContactMahmoud M Elkholy, Master
Backup ContactBasma A Mansour, PhD
Outcome results
None listed