A Phase 4 interventional study of Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine and Hydroxycarbamide 500 Mg Oral Capsule in Sickle Cell Disease, sponsored by Assiut University. Completed at 1 site in Egypt. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-10-22.
Sponsored by Assiut University · Phase 4, Interventional, and Treatment
The role of the combination therapy of hydroxyurea and L-Carnitine was studied in thalassemic patients. nevertheless its role in sickle cell anemia patients was not investigated
Sickle cell disease (SCD) is a common monogenic disorder affecting over 100,000 people in the United States alone, and millions more worldwide. This often devastating disease is characterized by red blood cell (RBC) sickling; chronic hemolytic anemia; episodic vaso-occlusion associated with severe pain and inflammation; acute and cumulative organ damage that manifests as stroke, acute chest syndrome, sickle lung disease, pulmonary hypertension nephropathy and end-stage renal disease; and other chronic morbidities.
Lives of patients with SCD are characterized by frequent episodes of severe pain (vaso-occlusive events or "crises"); acute organ dysfunction, including a pneumonia-like syndrome termed acute chest syndrome, and strokes starting in childhood; and progressive multi-organ damage. Not surprisingly, patients with SCD have very high health care utilization (over $1 billion/year in healthcare costs in the United States alone, and a median life-expectancy of only \~45-58 years, compared to the life expectancy of 78.2 years overall in the United States.
Although it is licensed in the United States for administration to sickle cell patients who have ≥ 3 crises a year in steady state, hydroxyurea (HU) remains unlicensed in most countries where it is regarded as an experimental drug In those areas, where HU is unlicensed for SCD, it is offered to patients who have ≥ 5 crises a year; or 3-4 crises a year with either neutrophil count ≥ 10 × 109/L or platelet count ≥ 500 × 109/L in steady state ; bearing in mind that the reference range for neutrophil count in black people is 1-3 × 109/L, and is 100-300 × 109/L for platelets .
Since high neutrophil count in steady state is a marker of severe SCD , these criteria usually identify individuals who have a clinical course sufficiently severe to ensure that the benefits of hydroxyurea therapy justify the potential risks. HU therapy is offered if the patient does not want to have (more) children, and is weighed against any severe impairment of liver or kidney function, or blood cytopenia. HU is unlicensed in most countries because the long-term adverse effects are unknown, not because the clinical efficacy is in doubt. In fact, after over 9 years of follow-up, HbSS subjects who received HU in the US placebo-controlled trial, had significantly less painful crises, acute chest syndrome, and mortality . Potential long-term toxic effects that reduce enthusiasm for HU include teratogenicity, carcinogenesis and, for young children, impaired cognitive development.
1,125 studies on the registry are indexed under Anemia, Sickle Cell; 239 are open to participants now.
This study's enrollment of 91 is above the median of 40 across 762 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Assiut University is the lead sponsor of 5,050 studies on the registry; 2,174 are open to participants now.
Of its 14 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
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Exclusion Criteria:
Hydroxycarbamide+ L-Carnitine+supportive treatment
Drug: Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine
Hydroxycarbamide+ supportive treatment
Drug: Hydroxycarbamide 500 Mg Oral Capsule
L-Carnitine+ supportive treatment
Drug: L-Carnitine, 250 Mg Oral Capsule
Supportive only
Drug page: Hydroxyurea
Combination therapy
Also known as: Hydroxyurea+LC
Single agent
Also known as: Hydroxyurea
single agent
Also known as: L-Carnitine
Hematological(HR)
Change of hemoglobin
Time frame: 2-3 months
Hematological response
Change of hematocrit
Time frame: 2-3 months
Frequency of painful episodes/ blood transfusions
Change of frequency of painful episodes and blood transfusion
Time frame: 1-year
Delayed effects
effect on long term complications as cardiovascular and cerebrovascular ones
Time frame: 12-18 months
Plan to share: No
Ran from Jan 2017 to primary completion in Jun 2020, 41 months.
Of 22 phase 4 interventional studies under Anemia, Sickle Cell, half reached primary completion within 20 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 41. This study's 41 months is above that.
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This study is completed, as verified in Dec 2016. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Hydroxyurea