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Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia

Phase 2
Completed
Conditions
Iron Overload
Oxidative Stress
Thalassemia Major
Interventions
Drug: Nigella Sativa Oil
Procedure: blood transfusion session
Registration Number
NCT04292314
Lead Sponsor
Beni-Suef University
Brief Summary

The aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production (LIPID PEROXIDATION) and their effect on iron intoxication (IRON CHELATION) in pediatric major thalassemia.

Detailed Description

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots. Beta thalassemia is classified into two types depending on the severity of symptoms: thalassemia major (also known as Cooley's anemia) and thalassemia intermedia. Of the two types, thalassemia major is more severe.

Beta-thalassemia syndromes are a group of hereditary blood disorders. It is characterized by reduced beta globin chain synthesis, resulting in reduced Hb in red blood cells (RBC), decreased RBC production and anemia.

Homozygotes for beta-thalassemia may develop either thalassemia major or thalassemia intermedia. Individuals with thalassemia major usually come to medical attention within the first 2 years and require regular blood transfusion to survive.

Affected infants with thalassemia major fail to thrive and become progressively pale. Feeding problems, diarrhea, irritability, recurrent bouts of fever, and enlargement of the abdomen, caused by splenomegaly, may occur. If a regular transfusion program that maintains a minimum Hb concentration of 95-105 g/L is initiated, then growth and development are normal until the age of 10-11 years. After the age of 10-11 years, affected individuals are at risk of developing severe complications related to posttransfusional iron overload, depending on their compliance with chelation therapy.

Complications of iron overload include growth retardation and failure of sexual maturation and also those complications observed in adults with hemachromatosis -associated hereditary hemochromatosis (HH): involvement of the heart (dilated myocardiopathy and pericarditis), liver (chronic hepatitis, fibrosis, and cirrhosis), and endocrine glands (resulting in diabetes mellitus and insufficiency of the parathyroid, thyroid, pituitary, and, less commonly, adrenal glands).

The underlying basis of b-thalassemia pathology is the diminished b-globin synthesis leading to a-globin accumulation and premature apoptotic destruction of erythroblasts, causing oxidative stress-induced ineffective erythropoiesis.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
350
Inclusion Criteria
  1. Any case with full manifestation of β-THALASSEMIA major disease
  2. #Aged from 7-15 years old
  3. accompanied with ineffective erythropoiesis

  4. with low hemoglobin level

  5. with iron overload

Exclusion Criteria
  1. The presence of any other chronic illness.
  2. Patient age>15 years old or < 7 years old.
  3. The presence of concomitant myocardial infarction, stroke, acute chest syndrome.
  4. The patient suffers from any other type of anemia.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Omega-3 experimental groupblood transfusion session50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Nigella sativa experimental groupNigella Sativa Oil50 patients from each participating hospital that will receive Nigella sativa supplementation (1g black seed oil contain 1% thymoquinone) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Nigella sativa experimental groupblood transfusion session50 patients from each participating hospital that will receive Nigella sativa supplementation (1g black seed oil contain 1% thymoquinone) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Hydroxyurea experimental groupblood transfusion session50 patients from each participating hospital that will receive hydroxyurea medication (5 to 15mg/kg) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Natural honey experimental groupblood transfusion session50 patients from each participating hospital that will receive natural honey(2.5 mg/kg dissolved in 250 ml water) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Ordinary hospital treatment groupblood transfusion session50 patients from each participating hospital that will receive the ordinary treatment of iron chelator agent of deferoxamine or deferasirox (SubQ infusion: 20 to 40 mg/kg/day over 8 to 12 hours, 6 to 7 nights per week, maximum daily dose: 40 mg/kg/day)for 8 consecutive months up to 10 months. in addition to iron chelator agent, this group receive regular blood transfusion session.
Omega-3 experimental groupOmega 350 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Omega-3 experimental groupDeferoxamine50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Nigella sativa experimental groupDeferoxamine50 patients from each participating hospital that will receive Nigella sativa supplementation (1g black seed oil contain 1% thymoquinone) per day for 8 consecutive months up to 10 months. in addition to experimental treatment this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods till efficacy of experimental treatment proved.
Hydroxyurea experimental groupHydroxyurea50 patients from each participating hospital that will receive hydroxyurea medication (5 to 15mg/kg) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Hydroxyurea experimental groupDeferoxamine50 patients from each participating hospital that will receive hydroxyurea medication (5 to 15mg/kg) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Natural honey experimental groupHoney50 patients from each participating hospital that will receive natural honey(2.5 mg/kg dissolved in 250 ml water) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Natural honey experimental groupDeferoxamine50 patients from each participating hospital that will receive natural honey(2.5 mg/kg dissolved in 250 ml water) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of deferoxamine/deferasirox plus regular blood transfusion with dose de-escalation methods until the efficacy of experimental treatment proved.
Ordinary hospital treatment groupDeferoxamine50 patients from each participating hospital that will receive the ordinary treatment of iron chelator agent of deferoxamine or deferasirox (SubQ infusion: 20 to 40 mg/kg/day over 8 to 12 hours, 6 to 7 nights per week, maximum daily dose: 40 mg/kg/day)for 8 consecutive months up to 10 months. in addition to iron chelator agent, this group receive regular blood transfusion session.
Primary Outcome Measures
NameTimeMethod
Reticulocyte absolute count10 months

Reticulocyte absolute count in a cubic milliliter of blood

HDL cholesterol Mg/dl10 months

HDL cholesterol milligrams per deciliter

LDL cholesterol Mg/dl10 months

LDL cholesterol milligrams per deciliter

Total cholesterol Mg/dl10 months

Total cholesterol milligrams per deciliter

% Chelation activity Fe+++ - thymoquinone complex3 months

Chelation activity of Ferric - thymoquinone complex in percentage measured by high pressure liquid chromatography coupled with gaschromatography - mass spectroscopy analysis

% transferrin saturation10 months

transferrin saturation percentage

F 2 -isoprostanes pg/mL3 months

plasma F 2 -isoprostanes Picograms Per Millilitre measured by high pressure liquid chromatography assay

Serum total iron mcg/dL10 months

Serum total iron micrograms per decilitre

C-reactive protein mg/L10 months

C-reactive protein milligrams per deciliter

Serum Ferritin ng/ml10 months

Serum Ferritin Nanograms per milliliter

Total Iron Binding Capacity (TIBC) mcg/dL10 months

Total Iron Binding Capacity micrograms per decilitre

hemoglobin (Hbg) g/dL10 months

hemoglobin (Hbg) gram/deciliter

mean corpuscular hemoglobin (MCH) pg/ml10 months

mean corpuscular hemoglobin (MCH) Picograms Per Millilitre

leukocytes count μl10 months

leukocytes in microliter

% Chelation activity Fe++ - thymoquinone complex3 months

Chelation activity of Ferrous - thymoquinone complex in percentage measured by high pressure liquid chromatography coupled with gaschromatography- mass spectroscopy analysis

Lactic acid dehydrogenase U/L10 months

Lactic acid dehydrogenase unit per litter

Reticulocyte count %10 months

Reticulocyte count percentage

Hb-F level g/dL10 months

hemoglobin- F level in gram per deciliter

Triglycerides Mg/dl10 months

Triglycerides milligrams per deciliter

White blood cells count10 months

White blood cells count in a cubic milliliter of blood

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (4)

Maternity and Children hospital

🇸🇦

Mecca, Saudi Arabia

Health insurance hospital

🇪🇬

Banī Suwayf, Egypt

Faculty of medicine, Beni-suef univeristy - Beni-Suef university hospital

🇪🇬

Banī Suwayf, Egypt

Faculty of Pharmacy, Beni-Suef university

🇪🇬

Banī Suwayf, Egypt

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