Hydoxycarbamide and L-Carnitine Therapy in Sickle Cell Anemia
NCT ID: NCT05081349
Last Updated: 2021-10-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE4
91 participants
INTERVENTIONAL
2017-01-10
2021-07-10
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Hydra+L-Carnitine
Hydroxycarbamide+ L-Carnitine+supportive treatment
Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine
Combination therapy
Hydra only
Hydroxycarbamide+ supportive treatment
Hydroxycarbamide 500 Mg Oral Capsule
Single agent
L-Carnitine only
L-Carnitine+ supportive treatment
L-Carnitine, 250 Mg Oral Capsule
single agent
Supportive measures
Supportive only
No interventions assigned to this group
Interventions
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Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine
Combination therapy
Hydroxycarbamide 500 Mg Oral Capsule
Single agent
L-Carnitine, 250 Mg Oral Capsule
single agent
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Not welling for pregnancy in females or to father a baby in males
* Frequent episodes
* Non-compliance to transfusion
Exclusion Criteria
* Hypersensitivity to hydroxycarbamide or L-Carnitine
* Pregnancy
* Other chronic infection or inflammation
18 Years
ALL
No
Sponsors
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Assiut University
OTHER
Responsible Party
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Safaa AA Khaled
Clinical Professor
Principal Investigators
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Israa EM Ashry, Prof
Role: STUDY_DIRECTOR
Assiut University- Faculty of Medicine
Locations
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Safaa A A Khaled
Asyut, , Egypt
Countries
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References
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Bunn HF. Pathogenesis and treatment of sickle cell disease. N Engl J Med. 1997 Sep 11;337(11):762-9. doi: 10.1056/NEJM199709113371107. No abstract available.
Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.
Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, Klug PP. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44. doi: 10.1056/NEJM199406093302303.
Kauf TL, Coates TD, Huazhi L, Mody-Patel N, Hartzema AG. The cost of health care for children and adults with sickle cell disease. Am J Hematol. 2009 Jun;84(6):323-7. doi: 10.1002/ajh.21408.
Elmariah H, Garrett ME, De Castro LM, Jonassaint JC, Ataga KI, Eckman JR, Ashley-Koch AE, Telen MJ. Factors associated with survival in a contemporary adult sickle cell disease cohort. Am J Hematol. 2014 May;89(5):530-5. doi: 10.1002/ajh.23683. Epub 2014 Feb 21.
Other Identifiers
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SKhaled2021
Identifier Type: -
Identifier Source: org_study_id