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Hydoxycarbamide and L-Carnitine Therapy in Sickle Cell Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05081349
Recruitment Status : Completed
First Posted : October 18, 2021
Last Update Posted : October 22, 2021
Information provided by (Responsible Party):
Safaa AA Khaled, Assiut University

Brief Summary:
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

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine Drug: Hydroxycarbamide 500 Mg Oral Capsule Drug: L-Carnitine, 250 Mg Oral Capsule Phase 4

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 91 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Non-randomized clinical trial
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment With Hydoxycarbamide and L-Carnitine in Adult Patients With Severe Forms of Sickle Cell Anemia: An Overview
Actual Study Start Date : January 10, 2017
Actual Primary Completion Date : June 10, 2020
Actual Study Completion Date : July 10, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Hydra+L-Carnitine
Hydroxycarbamide+ L-Carnitine+supportive treatment
Drug: Hydroxycarbamide 500 Mg Oral Capsule+L-Carnitine
Combination therapy
Other Name: Hydroxyurea+LC

Active Comparator: Hydra only
Hydroxycarbamide+ supportive treatment
Drug: Hydroxycarbamide 500 Mg Oral Capsule
Single agent
Other Name: Hydroxyurea

Active Comparator: L-Carnitine only
L-Carnitine+ supportive treatment
Drug: L-Carnitine, 250 Mg Oral Capsule
single agent
Other Name: L-Carnitine

No Intervention: Supportive measures
Supportive only

Primary Outcome Measures :
  1. Hematological(HR) [ Time Frame: 2-3 months ]
    Change of hemoglobin

  2. Hematological response [ Time Frame: 2-3 months ]
    Change of hematocrit

Secondary Outcome Measures :
  1. Frequency of painful episodes/ blood transfusions [ Time Frame: 1-year ]
    Change of frequency of painful episodes and blood transfusion

Other Outcome Measures:
  1. Delayed effects [ Time Frame: 12-18 months ]
    effect on long term complications as cardiovascular and cerebrovascular ones

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with sickle cell disease
  • Not welling for pregnancy in females or to father a baby in males
  • Frequent episodes
  • Non-compliance to transfusion

Exclusion Criteria:

  • <18 years
  • Hypersensitivity to hydroxycarbamide or L-Carnitine
  • Pregnancy
  • Other chronic infection or inflammation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05081349

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Safaa A A Khaled
Assiut, Egypt, 71515
Sponsors and Collaborators
Assiut University
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Study Director: Israa EM Ashry, Prof Assiut University- Faculty of Medicine
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Responsible Party: Safaa AA Khaled, Clinical Professor, Assiut University
ClinicalTrials.gov Identifier: NCT05081349    
Other Study ID Numbers: SKhaled2021
First Posted: October 18, 2021    Key Record Dates
Last Update Posted: October 22, 2021
Last Verified: December 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors