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 range
0 Years – 21 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥ 0 to ≤ 21 years
. Child should be receiving ongoing care with pediatric hematology/oncology providers
. Those enrolled in Arm A of the study should have a confirmed diagnosis of any of the following
. Those enrolled in Arm B of the study should have a confirmed diagnosis of any of the following:
. 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
. 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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. Adequate organ function within 7 days of enrollment defined as:
. Creatinine: ≤ 2.0 mg/dL
Exclusion criteria
. Gestational age \< 32 weeks or Age \> 21 years at the time of study enrolment
. Preexisting condition with predisposition for thrombosis
. 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
. Presence of complex karyotype or monosomy 7 or 5q- or other cytogenetic abnormality with known predisposition to cancer.
. Diagnosis of MDS with excess blasts in transformation
. Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotropin \[β-hCG\] pregnancy test) at screening or pre-dose on Day 1.
. Current alcohol or drug abuse.
. Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.