Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03896971
Recruitment Status : Recruiting
First Posted : April 1, 2019
Last Update Posted : June 17, 2019
Sponsor:
Collaborator:
Assiut University
Information provided by (Responsible Party):
Safaa AA Khaled, Assiut University

Brief Summary:

To the investigator's Knowledge this is the first study that will assess Treatment with thrombopoietin Mimetic plus immunosuppressiveTherapy in Egyptian Patients with Aplastic Anaemia.

Aim of the work :

  1. To evaluate the efficacy, tolerability and toxicity of the combination of thrombopoietin mimetic and immunosuppressive therapy in Egyptian patients with AA.
  2. To study the influence of this combination on patients' quality of life.
  3. To access evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome , acute leukemia or development of fibrosis

Condition or disease Intervention/treatment Phase
Aplastic Anemia Drug: Combination of thrombopoietin mimetic and cyclosporin A Phase 4

Detailed Description:

Aplastic anemia (AA) is a bone marrow failure syndrome that, although benign in nature, it influences patients' quality of life and carries poor prognosis.The pathophysiological basis of development of acquired AA include immune-mediated attack, inherent hematopoietic stem cell insufficiency and telomere defects. Bone marrow transplantation (BMT) is the only curative treatment for AA. Unfortunately it is unavailable for many patients due to lack of matched donors furthermore others are ineligible for BMT due to old age or co-morbid conditions. Immunosuppressive therapy was the mainstay of treatment for AA for many years, however many patients developed resistance or refractoriness. Immunosuppressive therapy was in the form of antithymocyte globulin (ATG) which gave hematologic response in nearly 50% of patients , adding cyclosporin A increases this response to 70%. Why some patients became resistant to immunosuppressive therapy ? The answer is not known.

Thrombopoietin mimetic (Eltrombopag) was firstly FDA approved for treatment of immune thrombocytopenic purpura. Numerous clinical trials proved the efficacy of anthropometric mimetic in patients with refractory severe AA, leading to its FDA approval for this group of patients. Some researchers proven the efficacy of thrombopoietin mimetic in patients with moderate aplastic anemia.

This study aimed to asses the combination of thrombopoietin mimetic and immunosuppressive therapy in patients with AA.


Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Two groups of patients will be included one group will be treated with thrombopoietin mimetic and immunosuppressive therapy , another matched group treated with immunosuppressive therapy alone and enrolled retrospectively
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment With Thrombopoietin Mimetic Plus Immunosuppressive Therapy in Egyptian Patients With Aplastic Anaemia
Actual Study Start Date : May 15, 2019
Estimated Primary Completion Date : May 1, 2020
Estimated Study Completion Date : March 1, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Thrombopoietin mimetic plus immunosuppressive therapy
The intervention group will be given cyclosporin A plus thrombopoietin (TPO) mimetic starting with 50 mg orally daily that would be increased or decreased according to response, for 3-months. Patients will be kept on weekly follow up visits and assessed by peripheral hemogram .
Drug: Combination of thrombopoietin mimetic and cyclosporin A
This is a controlled interventional study that will be carried out in the Clinical Hematology Unit, at the Department of Internal Medicine , Assiut University Hospital. Patients will be recruited among those who attending the outpatient clinic or admitted in the unit.
Other Name: Combination of Eltrombopag plus cyclosporin A

No Intervention: Immunosuppressive therapy
A comparative group will be collected retrospectively and treated with cyclosporin A alone as standard.



Primary Outcome Measures :
  1. Number of patients with hematologjc response [ Time Frame: 2-3months ]
    1. Platelet number ( time frame(2-3 months) : Increase plt count 20, 000 or more from baseline or independence of platelet transfusion for 2 months
    2. Hemoglobin percent (time frame (2-3 months): increase Hb by 1.5 g/dl from baseline or reduction 4 units of packed red blood cell transfusions for 2- months C) Absolute neutrophilic count response (time frame (2-3 months): increase absolute neutrophil count by 500 per microliter from baseline.

    d) Bone marrow cellularity (time frame (2-3 months): increase bone marrow cellularity from baseline



Secondary Outcome Measures :
  1. Number of patients with one of the following [ Time Frame: 2-3months ]
    1. Toxicity: development of toxicity leading to cessation of treatment
    2. Evolution: evolution to paroxysmal nocturnal hemoglobinuria , myelodysplastic syndrome or acute leukemia
    3. Bleeding and cytopenias that met the criteria for severe aplastic anemia and require transfusions



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.


Layout table for eligibility information
Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Eligible for treatment with thrombopoietin mimetic.
  • Willing to participate in the study and signed informed consent.
  • Unavailability of BMT -

Exclusion Criteria:

  • Patient with inherited aplastic anemia
  • Underlying immune deficiency
  • Contra indication to cyclosporin A
  • Endstage hepatic or renal disease
  • Pregnancy and lactation

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): NCT03896971


Contacts
Layout table for location contacts
Contact: Safaa AA Khaled, Prof. 01064170058 ext 002 safaakhaled2003@gmail.com

Locations
Layout table for location information
Egypt
Assiut university hospital Internal Medicine Department Hematology and BMT Unit Recruiting
Assiut, Egypt, 71515
Contact: Ahmed MK El Menshawy, Professor    08822080150 ext 002    vp_grad@aun.edu.eg   
Sponsors and Collaborators
Safaa AA Khaled
Assiut University
Investigators
Layout table for investigator information
Study Director: Safinaz Hussien, MD Assiut University
Principal Investigator: Sawsan A Abdelaal, M.B.B.Ch Assiut University

Publications:

Layout table for additonal information
Responsible Party: Safaa AA Khaled, Associate professor, Assiut University
ClinicalTrials.gov Identifier: NCT03896971     History of Changes
Other Study ID Numbers: SKhaled19
First Posted: April 1, 2019    Key Record Dates
Last Update Posted: June 17, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Safaa AA Khaled, Assiut University:
thrombopoietin mimetic
Egyptian
Aplastic
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclosporine
Cyclosporins
Immunosuppressive Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors