TPO-Mimetic Use in Children for Hematopoietic Failure (NCT04478227) | Clinical Trial Compass
CompletedEarly Phase 1
TPO-Mimetic Use in Children for Hematopoietic Failure
United States15 participantsStarted 2020-08-18
Plain-language 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.
Who can participate
Age range0 Years β 21 Years
SexALL
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Inclusion criteria
β. Age β₯ 0 to β€ 21 years
β. Child should be receiving ongoing care with pediatric hematology/oncology providers
β. Those enrolled in Arm A of the study should have a confirmed diagnosis of any of the following
β. Those enrolled in Arm B of the study should have a confirmed diagnosis of any of the following:
β. myelo-suppression specifically thrombocytopenia as defined by primary oncologist in children with solid tumors secondary to chemotherapy or radiation therapy contributing to delay in chemotherapy
β. patient undergoing stem cell transplantation and experiencing persistent thrombocytopenia. This will include children not requiring platelet transfusions with a platelet count of \<10 x 109/L, as well as those requiring platelet transfusions (transfusion dependent) for prevention of bleeding diathesis regardless of their platelet count at the time of recruitment (note: due to delayed engraftment these patients may have a higher platelet count because of platelet transfusion needs at the time of recruitment).
β. Adequate organ function within 7 days of enrollment defined as:
β. Creatinine: β€ 2.0 mg/dL
Exclusion criteria
β. Gestational age \< 32 weeks or Age \> 21 years at the time of study enrolment
β. Preexisting condition with predisposition for thrombosis
β
What they're measuring
1
Occurrence of treatment-related adverse events (AEs) according to NCI CTCAE v5.0
Timeframe: During treatment and through 90 days following discontinuation of treatment
2
To estimate preliminary efficacy of Romiplostim as measured by improvement in hematopoiesis
. Diagnosis of bone marrow failure syndrome with cancer predisposition including chromosomal fragility disorders (Fanconi anemia, Bloom syndrome, Ataxia Telangiectasia) and other conditions with known association towards cancer predisposition
β. Presence of complex karyotype or monosomy 7 or 5q- or other cytogenetic abnormality with known predisposition to cancer.
β. Diagnosis of MDS with excess blasts in transformation
β. Female subjects who are nursing or pregnant (positive serum or urine Ξ²-human chorionic gonadotropin \[Ξ²-hCG\] pregnancy test) at screening or pre-dose on Day 1.
β. Current alcohol or drug abuse.
β. Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.