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

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ClinicalTrials.gov Identifier: NCT04301336
Recruitment Status : Recruiting
First Posted : March 10, 2020
Last Update Posted : March 11, 2020
Sponsor:
Collaborators:
Department of clinical pharmacy, faculty of pharmacy, Beni-suef univeristy
Benisuef university hospital
University of Arizona
College of Pharmacy,Department of Pharmacy Practice,University of Arizona
Maternity and Children Hospital, Makkah
Information provided by (Responsible Party):
SHAIMAA MAHMOUD NASHAT SHAYED ABDELHALIM, Beni-Suef University

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Vaso-occlusive Crisis Sickle Cell Disease Sickle Cell Anemia in Children Drug: Omega 3 Drug: Vit D Drug: Zinc sulfate Drug: Statins (Cardiovascular Agents) Drug: Hydroxy Urea Drug: Folic Acid Supplementation Drug: Morphine Sulfate Procedure: blood transfusion session Phase 2 Phase 3

Detailed Description:

"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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 500 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Four experimental groups, one control group
Masking: Double (Participant, Care Provider)
Primary Purpose: Treatment
Official Title: Comparative Effectiveness of the Different Treatment Modalities for Management of Vaso-occlusive Painful Crisis in Pediatric Sickle Cell Disease
Actual Study Start Date : November 1, 2019
Estimated Primary Completion Date : October 2020
Estimated Study Completion Date : November 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Omega-3 experimental group

50 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 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.

Drug: Omega 3
Omega-3 supplementation (300-400mg EPA & 200-300mg DHA) per day for 8 consecutive months up to 10 months
Other Name: omega-3 supplementation capsules

Drug: Hydroxy Urea
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 Name: Hydroxy Urea tablet medication 20mg/kg/day

Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Name: Folic Acid tablet medication 1mg/day

Drug: Morphine Sulfate
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 Name: Morphine Sulfate intra venous medication

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

Experimental: Vit-D experimental group

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.

Drug: Vit D

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 Name: Vit-D medication oral drops

Drug: Hydroxy Urea
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 Name: Hydroxy Urea tablet medication 20mg/kg/day

Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Name: Folic Acid tablet medication 1mg/day

Drug: Morphine Sulfate
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 Name: Morphine Sulfate intra venous medication

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

Experimental: Zinc supplements experimental group

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.

Drug: Zinc sulfate

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 Name: Zinc tablet medication

Drug: Hydroxy Urea
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 Name: Hydroxy Urea tablet medication 20mg/kg/day

Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Name: Folic Acid tablet medication 1mg/day

Drug: Morphine Sulfate
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 Name: Morphine Sulfate intra venous medication

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

Experimental: Statin experimental group

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.

Drug: Statins (Cardiovascular Agents)

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 Name: Simvastatin 20mg

Drug: Hydroxy Urea
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 Name: Hydroxy Urea tablet medication 20mg/kg/day

Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Name: Folic Acid tablet medication 1mg/day

Drug: Morphine Sulfate
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 Name: Morphine Sulfate intra venous medication

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

Active Comparator: Ordinary hospital treatment group

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.

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

This group received regular blood transfusion session.

Drug: Hydroxy Urea
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 Name: Hydroxy Urea tablet medication 20mg/kg/day

Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Name: Folic Acid tablet medication 1mg/day

Drug: Morphine Sulfate
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 Name: Morphine Sulfate intra venous medication

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




Primary Outcome Measures :
  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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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:

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

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): NCT04301336


Contacts
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Contact: SHAIMAA M NASHAT SAYED ABDELHALIM, Ph.D Student 00201110177667 drshimaa.nashat@gmail.com

Locations
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Egypt
Faculty of medicine, Beni-suef univeristy - Beni-Seuf university hospital Recruiting
Banī Suwayf, Egypt
Contact: MOHAMED H MEABAD, M.D    00201001310287    M1hmeabed2@yahoo.com   
Contact: AHMED A ALBERRY, M.D    00201000425922    berry_ahmed@yahoo.com   
Faculty of Pharmacy, Beni-Suef university Recruiting
Banī Suwayf, Egypt
Contact: Shaimaa M Nashat Sayed Abdelhalim, Ph.D.student    00201110177667    drshimaa.nashat@gmail.com   
Contact: RAGHDA R SAYED, Ph.D.    00201010647666    smartraro@yahoo.com   
Saudi Arabia
Maternity and Children hospital Recruiting
Mecca, Saudi Arabia
Contact: Shaimaa M Nashat Sayed Abdelhalim, PhD student    00201110177667    drshimaa.nashat@gmail.com   
Contact: HASSAN Masmali [consultant of Pediatrics], M.D    00966555352442    masmaly@gmail.com   
Sponsors and Collaborators
Beni-Suef University
Department of clinical pharmacy, faculty of pharmacy, Beni-suef univeristy
Benisuef university hospital
University of Arizona
College of Pharmacy,Department of Pharmacy Practice,University of Arizona
Maternity and Children Hospital, Makkah
Investigators
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Study Director: John E. Murphy [Professor of Pharmacy Practice and Science], PharmD University of Arizona, College of Pharmacy
Study Director: Mohamed H Meabad [Prof of Pediatrics], M.D Beni-Suef University, Faculty of medicine
Study Director: AHMED A ALBERRY [Assistant prof of clinical pharmacology], M.D Beni-Suef University, Faculty of medicine
Study Director: RAGHDA R SAYED [Lecturer of Clinical Pharmacy, Ph.D. Beni-Suef University, Faculty of Pharmacy
Principal Investigator: Shaimaa M Nashat Sayed Abdelhalim, Ph.D Student Beni-Suef University, Faculty of Pharmacy

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Responsible Party: SHAIMAA MAHMOUD NASHAT SHAYED ABDELHALIM, Dr.Shaimaa Mahmoud Nashat Sayed Abdelhalim, Ph.D. Researcher and Principal Investigator, Beni-Suef University
ClinicalTrials.gov Identifier: NCT04301336    
Other Study ID Numbers: Novel TTT of pedia VOC/SCA
First Posted: March 10, 2020    Key Record Dates
Last Update Posted: March 11, 2020
Last Verified: March 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by SHAIMAA MAHMOUD NASHAT SHAYED ABDELHALIM, Beni-Suef University:
omega3
Vaso-occlusive painful crisis
Pediatric sickle cell anemia
comparative effective analysis in VOC
Anti-inflammatory effect of Vit-D
Anti-hemolytic effect of Vit-D
Anti-hyperlipidemia of Vit-D
effect of omega-3 on blood rheology
effect of omega-3 on blood viscosity
Antiaggregation effect of omega-3
Anti-inflammatory effect of statins
Anti-inflammatory effect of zinc supplements
effect of zinc supplements on blood viscosity
effect of zinc supplements on blood rheology
Antiaggregation effect of zinc supplements
pediatric sickle cell disease
Additional relevant MeSH terms:
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Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Vitamin D
Zinc
Folic Acid
Vitamin B Complex
Hydroxyurea
Morphine
Zinc Sulfate
Simvastatin
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Analgesics
Analgesics, Opioid
Narcotics
Central Nervous System Depressants
Physiological Effects of Drugs
Sensory System Agents
Peripheral Nervous System Agents
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Enzyme Inhibitors
Trace Elements