Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA
A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia
1 other identifier
interventional
100
1 country
1
Brief Summary
The analysis of our own clinical data suggests that majority of the hematologic responses observed after the course of h-ATG/CsA is partial, and about 10% tend to have cyclosporine dependence. The aim of the current study is to improve the rate and the quality of hematologic response as well as to prevent delayed complications such as relapse and clonal progression by means of adding eltrombopag to standard immunosuppressive therapy
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2016
Longer than P75 for phase_3
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 10, 2016
CompletedFirst Submitted
Initial submission to the registry
December 22, 2017
CompletedFirst Posted
Study publicly available on registry
January 29, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 20, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
October 20, 2022
CompletedMarch 15, 2021
March 1, 2021
5.9 years
December 22, 2017
March 12, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
ORR (CR + PR)
The primary objective of this trial is to investigate whether Eltrombopag added to standard immunosupressive treatment hATG and CsA increases the overal (partial + complete) response rate at four months in untreated children with severe aquired aplastic anemia.
4 months
Secondary Outcomes (13)
Platelet count
4 and 6 months
Hemoglobin
4 and 6 months
Neutrophil count
4 and 6 months
Cumulative incidence of response
4 and 6 months
Duration of hematologic response
2 years
- +8 more secondary outcomes
Study Arms (2)
Eltrombopag + IST (ATG + CsA)
EXPERIMENTALIST (ATG + CsA)
ACTIVE COMPARATORInterventions
Eltrombopag is an oral mimetic thrombopoietin selectively binding transmembrane and juxtamembrane domains of the thrombopoietin receptor different from the binding site of thrombopoietin. Therefore it does not compete for binding with the native molecule. It is promoting thrombopoiesis and release platelets from mature megakaryocytes. Also it promotes more primitive multilineage progenitors and hematopoietic stem cells to proliferate and differentiate into thrombocytes, erythrocytes and leukocytes.
Eligibility Criteria
You may qualify if:
- Clinical diagnosis of severe and very severe Aplastic anemia
- years old
- Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.
- Absence of HLA-identical family member
You may not qualify if:
- \. myelodysplastic syndrome 4. Prior immunosuppressive therapy 5. Patients with hepatic, renal or cardiac failure, or any other life- threatening concurrent disease 6. hypersensitivity to any of the component medications 7. Creatinine \>2.5 mg/dL× the upper limit of normal, 8. Total bilirubin \>1.5 × the upper limit of normal mg/dL , 9. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3-5 × the upper limit of normal
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117198, Russia
Related Publications (1)
Goronkova O, Novichkova G, Salimova T, Kalinina I, Baidildina D, Petrova U, Antonova K, Sadovskaya M, Suntsova E, Evseev D, Matveev V, Venyov D, Khachatryan L, Litvinov D, Pshonkin A, Ovsyannikova G, Kotskaya N, Gobadze D, Olshanskaya Y, Popov A, Raykina E, Mironenko O, Voronin K, Purbueva B, Boichenko E, Dinikina Y, Guseynova E, Sherstnev D, Kalinina E, Mezentsev S, Streneva O, Yudina N, Plaksina O, Erega E, Maschan M, Maschan A. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood Adv. 2023 Mar 28;7(6):953-962. doi: 10.1182/bloodadvances.2021006716.
PMID: 35446936DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Galina Novichkova
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
- PRINCIPAL INVESTIGATOR
Alexei Maschan
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 22, 2017
First Posted
January 29, 2018
Study Start
December 10, 2016
Primary Completion
October 20, 2022
Study Completion
October 20, 2022
Last Updated
March 15, 2021
Record last verified: 2021-03