TPO-Mimetic Use in Children for Hemotopoietic Failure

  • STATUS
    Recruiting
  • End date
    Dec 3, 2022
  • participants needed
    25
  • sponsor
    Anjali Sharathkumar
Updated on 18 June 2021
platelet count
stem cell transplantation
anemia
cell transplantation
leukemia
cytopenia
thrombocytopenia
neutrophil count
blood transfusion
platelet transfusion
aplastic anemia
severe aplastic anemia
pancytopenia
reticulocyte count
thrombopoietin receptor agonist
romiplostim
splenectomy
thrombocytopenic purpura
monosomy 7

Summary

This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure disorders including acquired and inherited conditions as a first line of therapy along with standard of care.

Description

This investigator-initiated study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure disorders (BMF) including acquired and inherited conditions as a first line of therapy along with standard of care.

Objectives: Primary objectives are to evaluate safety and preliminary efficacy of Romiplostim in children with BMF. Methods: This open label, prospective Pilot interventional study has two arms.

Arm A will include acquired bone marrow failure (BMF) disorders including aplastic anemia, refractory cytopenia of childhood without monosomy 7 and 5q deletion abnormalities, toxin induced myelosuppression due to infection and inherited cytopenia with or without involvement of other cell lines who are transfusion dependent and or showing progression to bone marrow failure. Arm B will include children with chemo and or radiotherapy induced thrombocytopenia/cytopenia and children undergoing stem cell transplantation (SCT). Children with cancer predisposition and other morbidities which are considered significant by the investigator will be excluded from the study.

Details
Condition Refractory Cytopenia of Childhood, Anemia; Non-Hodgkin’s Lymphoma, Thrombocytopenia, myelodysplastic syndromes, Bone Marrow Failure Disorders, Thrombocytopenia and Thrombocytopenia Prevention, Aplastic Anemia, myelodysplastic syndrome (mds), Anemia; Non-Small-Cell Lung Cancer, Bone marrow disorder, Red Blood Cell Disorders, Myelodysplastic Syndromes (MDS), Anemia, MYELODYSPLASTIC SYNDROME, Bone Marrow Failure Syndromes, Preleukemia, Bone Marrow Disorder
Treatment romiplostim
Clinical Study IdentifierNCT04478227
SponsorAnjali Sharathkumar
Last Modified on18 June 2021

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