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The Effect of Eltrombopag on the Expression of Platelet Collagen Receptor GPVI in Pediatric ITP.

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ClinicalTrials.gov Identifier: NCT03412188
Recruitment Status : Recruiting
First Posted : January 26, 2018
Last Update Posted : August 14, 2018
Sponsor:
Information provided by (Responsible Party):
Nayera Hazaa Elsherif, Ain Shams University

Study Description
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Brief Summary:

Using eltronmobag as an alternative pathway, which depend on stimulation of thrombocyte synthesis, in chronic and persistent immune thrombocytopenia may be more promising treatment than the classic type, not only by increasing platelets count but also through enhancing of the platelets activation and upregulation of GPVI expression on platelet surface.

This study will include 40 pediatric patients with chronic or persistent ITP, recruited from the Hematology clinic of pediatric hospital Ain Shams University, aiming to investigate the efficacy, & the safety and tolerability of eltrombopag therapy for children as well as the ability of eltrombopag to enhance the platelet activation through the up-regulation of glycoprotein VI (GPVI) receptor expression in comparison with other lines of treatment.

Patients will be divided into 2 groups : Group 1 on eltrombopag ; Group 2 : receiving other lines of therapy .

All Patients will be subjected to :

  1. bleeding score assessment ( baseline &every month ) and Health related quality of life based on Kids' ITP Tools (KIT) questionnaires( baseline and week 24 )
  2. Baseline and at week 24 bone marrow examination with reticulin stain
  3. clinical examination every 2 weeks and complete blood counts.
  4. Assessment of soluble form of glycoprotein VI using sandwich enzyme-linked immunosorbent assay (ELISA) as well as assessment of platelet activation by GPVI using flowcytometry (Gardiner, etal.,2010 ) at baseline and at the end of 6 months treatment period

Condition or disease Intervention/treatment Phase
Idiopathic Thrombocytopenic Purpura Drug: Eltrombopag Drug: conventional Not Applicable

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Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

Patients will be divided into 2 groups:

Group 1 (eltrombopag arm n=20 patients): Patients who showed no response (platelet count ≤ 20x109/L) initially for 3 months or relapse after 6 months after at least one prior ITP therapy. patients will receive a total daily dose of eltrombopag of (25-50mg/d). Dose adjustments may be made based on platelets count.

Group 2 (n=20 patients) Patients who are currently receiving other lines of treatment (steroids, IVIG, azathioprine, and rituximab).
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of the Effect of Eltrombopag on the Expression of Platelet Collagen Receptor Glycoprotein VI (GPVI) in Pediatric Patients With Chronic and Persistent Immune Thrombocytopenia.
Actual Study Start Date : August 1, 2018
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : November 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arms and Interventions
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Arm Intervention/treatment
Experimental: group 1 Eltrombopag arm

Group 1 (eltrombopag arm n=20 patients): Patients who showed no response (platelet count ≤ 20x109/L) initially for 3 months or relapse after 6 months after at least one prior ITP therapy. patients will receive a total daily dose of eltrombopag of (25-50mg/d). Dose adjustments may be made based on platelets count with an increment of 25mg once per day at 2 weeks intervals (Maximum dose: 75 mg orally once a day).

Patients, who responded poorly to eltrombopag in 6 months or developed adverse effects, were asked to discontinue the medication. Those who responded were followed for further 6 month period.

 Drug: Eltrombopag
Patients who showed no response (platelet count ≤ 20x109/L) initially for 3 months or relapse after 6 months after at least one prior ITP therapy will receive a total daily dose of eltrombopag of (25-50mg/d)
Other Name: Revolade
Active Comparator: group 2 conventional Treatment

Group 2 (n=20 patients) Patients who are currently receiving other lines of treatment (steroids, IVIG, azathioprine, and rituximab).

patients will continue on the conventional line of treatment

 Drug: conventional
Patients who are currently receiving conventional lines of treatment (steroids, IVIG, azathioprine, and rituximab, Mycophenolate mofetil) will continue on the same line of treatment
Other Names:
  • solumedrol
  • imuran
  • cellcept
  • mabthera
  • gammaglobulin


Outcome Measures
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Primary Outcome Measures :
  1. Expression of glycoprotein VI collagen receptor [ Time Frame: at 24 weeks ]
    Percentages of patients achieving increased expression of glycoprotein VI collagen receptor


Secondary Outcome Measures :
  1. Complete response or response [ Time Frame: at 24 weeks ]
    Percentages of patients achieving Complete response" (CR) defined as any platelet count of 100x109/L at least once throughout the study period in the absence of rescue treatment and those achieving "Response" (R) is defined as any platelet count between 50 and 100x 109/L or doubling of the baseline count at least once throughout the study period

  2. Maintenance of response [ Time Frame: at 24 weeks ]
    The maximum duration for which a patient continuously maintained a platelet count between 50 and 100x 10*9/L in the absence of rescue treatment.

  3. Safety: Number of Patients who will have adverse events [ Time Frame: at 24 weeks ]
    Number of Patients who will have adverse events.


Eligibility Criteria
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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age ≥ 1 to ≤ 18 years at enrollment. Patient should be able to swallow tablet form. Weight should be ≥ 12kg.
  • Diagnosis of persistent (3-<12 months duration) or chronic cases (≥12 months duration) with day 1 platelet count ≤ 20x10^9.
  • Patients have a bleeding score grade 3 for skin and/or grade 2 or higher for mucosal domains and/or higher than grade 1 for organ domain at the baseline visit or the worst bleeding incident episode in the patient's medical reports in the last 3 months prior screening, using ITP-BAT (v1.0) score.
  • Normal Kidney function tests and liver function tests.

Exclusion Criteria:

  • Acute thrombocytopenic purpura patients or Other causes of thrombocytopenia. Patients with Evans syndrome
  • Hypertension, cardiovascular disease, diabetes, hepatitis C virus (HCV), HIV, hepatitis B surface antigen(HBsAg) seropositive status.
  • Baseline bone marrow biopsy with evident fibrosis (reticulin stain grade 2 or more)
  • Patients who have previously received eltrombopag.
Contacts and Locations
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT03412188


Contacts
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Contact: nayera elsherif, MD 01003322052 elsherif.nayera@gmail.com
Contact: fatma ebeid, MD 01095569596 dr.fatma_ebeid@yahoo.com

Locations
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Egypt
Ainshams University , Faculty of medicine , Pediatric Hematology&Oncology unit, children hospital. Recruiting
Cairo, Egypt, 11579
Contact: Micheal morcos, M.Sc       mekoo_828@yahoo.com   
Contact: Mahmoud Kenny, MD       moudykenny@yahoo.com   
Principal Investigator: Azza AG Tantawy, MD         
Sub-Investigator: Nayera HK El Sherif, MD         
Sub-Investigator: Fatma S Ebeid, MD         
Sub-Investigator: Eman AR Ismail, MD         
Sub-Investigator: Mahmoud A Kenny, MD         
Sub-Investigator: Michael BE morcos, M.Sc         
Sponsors and Collaborators
Nayera Hazaa Elsherif
Investigators
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Principal Investigator: Azza AG Tantawy, MD Ain shams university, Faculty of medicine, Pediatric Hematology &Oncology department
More Information
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Publications of Results:

Other Publications:

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Responsible Party: Nayera Hazaa Elsherif, assistant professor of pediatrics, Ain Shams University
ClinicalTrials.gov Identifier: NCT03412188    
Other Study ID Numbers: GPVI-ITP
First Posted: January 26, 2018    Key Record Dates
Last Update Posted: August 14, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Still investigators did not decide about sharing individual participants data

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Nayera Hazaa Elsherif, Ain Shams University:
Chronic ITP
persistent ITP
Eltrombopag
GPVI receptor
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
 Hemorrhagic Disorders
Autoimmune Diseases
Azathioprine
gamma-Globulins
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antimetabolites, Antineoplastic
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents