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NCT Number: NCT04301336

Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia

The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease. In addition, investigators apply the Cost-effectiveness analysis (CEA) as a form of economic analysis that compares the relative costs and outcomes (effects) for different treatment regimens on vaso-occlusive painful crisis.

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Key information

Conditions

Vaso-occlusive Crisis Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Sickle Cell Anemia in Children Sickle Cell Disease Vaso-Occlusive Crises

Age range

5 year–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Faculty of Pharmacy, Beni-Suef university, Banī Suwayf, Egypt

Loading trial locations.

About this study

"Sickle cell disease is an inherited blood disorder characterized by defective hemoglobin (a protein in red blood cells that carries oxygen to the tissues of the body).

Sickle cell disease involves the red blood cells, or hemoglobin, and their ability to carry oxygen. Normal hemoglobin cells are smooth, round, and flexible, like the letter "O," so they can move through the vessels in our bodies easily. Sickle cell hemoglobin cells are stiff and sticky and form into the shape of a sickle, or the letter "C," when they lose their oxygen. These sickle cells tend to cluster together and cannot easily move through the blood vessels. The cluster causes a blockage in small arteries or capillaries and stops the movement of healthy, normal oxygen-carrying blood. This blockage is what causes the painful and damaging complications of sickle cell disease".

"Acute vaso-occlusive crisis (VOC) is a hallmark of sickle cell disease (SCD). Multiple complex pathophysiological processes can result in pain during a VOC. Despite significant improvements in the understanding and management of SCD, little progress has been made in the management of pain in SCD, although new treatments are being explored".

The Painful Episodes:

"The day-to-day management of sickle cell disease often equates with the management of acute and chronic pain. Patients manage many painful events at home so that hospital visits underestimate the frequency of pain

Acute painful episodes are the most commonly encountered vaso-occlusive events in patients of all ages. Presumed to be caused by sickle vaso-occlusion, pain often starts in young children as the hand-foot syndrome or dactylitis, a painful swelling of hands and feet due to inflammation of the metacarpal and metatarsal periosteum. Painful episodes, which last from hours to many days, usually occur with little warning and a clear precipitating event is not often found.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Any case with the full manifestation of sickle cell disease accompanied by acute painful crisis aged from 5-15 years old.

Exclusion criteria

  • The presence of any other chronic illness.
  • Patient age>18 years old or < 3 years old.
  • Patients with hepatic diseases including cholestasis hepatic encephalopathy and jaundice.
  • Patients with renal impairment
  • Diabetic patients

Treatment and study plan

OMega 3

Drug

Omega-3 supplementation (300-400mg EPA & 200-300mg DHA) per day for 8 consecutive months up to 10 months

Other names: omega-3 supplementation capsules

Vit D

Drug

50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months.

in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.

Other names: Vit-D medication oral drops

zinc sulfate

Drug

50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months.

in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.

Other names: Zinc tablet medication

Statins (Cardiovascular Agents)

Drug

50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months.

in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.

Other names: Simvastatin 20mg

Hydroxy Urea

Drug

50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.

Other names: Hydroxy Urea tablet medication 20mg/kg/day

Folic Acid Supplementation

Drug

Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response

Other names: Folic Acid tablet medication 1mg/day

Morphine sulfate

Drug

Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.

Other names: Morphine Sulfate intra venous medication

blood transfusion session

Procedure

Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.

Primary outcomes

  1. C-reactive protein mg/L

    Time frame: 10 months

    C-reactive protein milligrams per deciliter

  2. Hematocrit %

    Time frame: 10 months

    Hematocrit level in percentage value

  3. Fibrinogen mg/dl

    Time frame: 10 months

    Fibrinogen concentration in milligrams per deciliter

  4. Total cholesterol Mg/dl

    Time frame: 10 months

    Total cholesterol milligrams per deciliter

  5. HDL cholesterol Mg/dl

    Time frame: 10 months

    HDL cholesterol milligrams per deciliter

  6. LDL cholesterol Mg/dl

    Time frame: 10 months

    LDL cholesterol milligrams per deciliter

  7. Triglycerides Mg/dl

    Time frame: 10 months

    Triglycerides milligrams per deciliter

  8. leukocytes count μl

    Time frame: 10 months

    leukocytes in microliter

  9. hemoglobin (Hbg) g/dL

    Time frame: 10 months

    hemoglobin (Hbg) gram/deciliter

  10. White blood cells count

    Time frame: 10 months

    White blood cells count in a cubic milliliter of blood

  11. Lactic acid dehydrogenase U/L

    Time frame: 10 months

    Lactic acid dehydrogenase unit per litter

  12. Reticulocyte count %

    Time frame: 10 months

    Reticulocyte count percentage

  13. Red blood cell (erythrocyte ) sedimentation rate mm/hr

    Time frame: 10 months

    erythrocyte sedimentation rate in millimeters (mm) per one hour(hr)

  14. lymphocyte count µL

    Time frame: 10 months

    lymphocyte count in 1 microliter (µL) of blood

  15. Granulocyte absolute count cells/microliter

    Time frame: 10 months

    Granulocyte cells numbers in microliter

  16. Granulocytes,percentage (GR, pct)

    Time frame: 10 months

    percentage of white blood cells with granules in percentage

Sponsors and collaborators

Lead sponsor

Beni-Suef University

Other

Collaborators

  • Benisuef university hospital
  • Maternity and Children Hospital, Makkah
  • University of Arizona

Registry information

Official study title

Comparative Effectiveness of the Different Treatment Modalities for Management of Vaso-occlusive Painful Crisis in Pediatric Sickle Cell Disease

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Mar 10, 2020
Registry last updated
Jan 27, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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