NCT04478227

TPO-Mimetic Use in Children for Hematopoietic Failure

Single Arm Prospective Open Label Pilot Study Evaluating Short-Term Safety and Efficacy of Romiplostim in Children With Inherited and Acquired Disorders of Hematopoietic Failure

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Anjali Sharathkumar · Academic / Other
• Sex: All
• Age: 0 Years – 21 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Bone Marrow Failure Disorders
• Aplastic Anemia
• Thrombocytopenia
• Refractory Cytopenia of Childhood
• Myelodysplastic Syndrome(MDS)

Interventions

Type	Name	Description
DRUG	Romiplostim	Nplate is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Timeline

• Start date: 2020-08-18
• Primary completion: 2024-01-18
• Completion: 2024-01-18
• First posted: 2020-07-20
• Last updated: 2024-07-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study