Romiplostim for Severe Aplastic Anemia in Children and Young Adults
This study is looking at how well Romiplostim works when added to standard treatment for severe aplastic anemia (SAA) in children and young adults. Romiplostim is a drug that helps your body make more blood cells. The standard treatment includes two drugs: Horse Anti-thymocyte Globulin (H-ATG) and Cyclosporine (CSA). This study includes patients who are newly diagnosed with SAA, as well as those whose SAA has come back or didn't respond to previous treatments. To join, you must be between 2 and 21 years old and have a confirmed diagnosis of SAA. The main goal is to see how many participants have a complete response in their blood counts after 24 weeks of treatment. The study is currently unclear about its recruitment status.
- Study design
- This is an open-label, interventional study, meaning both you and your doctors will know which treatments you are receiving. It plans to include 15 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The main treatment response will be measured during 24 weeks of therapy.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT07001254
UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
Not Yet Recruiting
PHASE2Ages 2–21InterventionalTreatmentAnjali SharathkumarInvestigator-initiated
~15 participants
Updated 2025-12-19 on ClinicalTrials.gov
What's tested:RomiplostimImmunosuppressive therapy (IST)
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24
Measured over During 24 weeks of therapy
Conditions
Where it's being run
1 sites across 1 statesIowa1
Study leadership
- Anjali Sharathkumar, MD · PRINCIPAL_INVESTIGATOR · University of Iowa
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age ≥2 years to ≤21 years
Child should be receiving ongoing care with pediatric hematology/oncology provider.
Confirmed Diagnosis of SAA and other related conditions based on following criteria.
Females of childbearing potential agree to use effective contraception during the study period and for 4 months after completion of therapy.
Patient with available allogenic stem cell donor chooses to defer HSCT option at least for 12-weeks.
Parent, guardian, or patient (if \>18 years of old) must be able to provide written and voluntary informed consent and assent (if \>12 years of age).
Prior to participation in the study, patients will be offered available therapeutic options and concurrent competing studies evaluating alternative therapies for SAA. Matched sibling donor transplant is the preferred curative option and will be offered to all patients. If patient has a HLA-matched unrelated donor (9/10 or 10/10), and if there is an option to participate in a research study evaluating a cure with matched-unrelated donor transplant, the patient will offered those options prior to recruiting in the study. In US, TransIT (NCT05600426): "A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia" is currently recruiting newly diagnosed children with SAA for matched unrelated donor transplant who are not eligible for matched sibling donor transplant. However, the study requires availability of two matched unrelated donors (9/10 or 10/10). If the patient chooses to undergo matched-unrelated donor transplantation either with participation with the research study or outside the research study, the patient will not be recruited in the study.
Prior history of use of another TPO-mimetic (eltrombopag, avatrombopag and lusutrombopag) and androgen for more than 30 days ago will not be a contraindication. Dysplastic changes with diagnosis of SAA are acceptable as long as hematopathologist is not concerned about alternative diagnosis of MDS or refractory cytopenia of childhood. Patients who have discontinued androgen therapy for more than 2- weeks will be considered for study participation.
Exclusion
Age \< 2 years or \>21 years \*Availability of suitable HLA-matched related or HLA-matched unrelated (9/10 or 10/10) allogenic stem cell donor and the participant fulfills the requirement for HSCT and opts to undergo allogenic HSCT.\*\*
Patients with Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones \>50% of granulocytes or RBC at time of enrollment.
Preexisting condition with predisposition for thrombosis such as protein C, S, antithrombin deficiency, homozygous factor V Leiden or prothrombin 20210 polymorphism, history of idiopathic thromboembolism with patient or first degree relative.
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.
Patients taking concurrent therapy with eltrombopag, avatrombopag, and lusutrombopag\*\*\*.
Patients taking concurrent therapy with androgens\*\*\*.
Females 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.
Active and uncontrolled infections (e.g., sepsis, hepatitis B, hepatitis C).
Chronic liver disease, i.e., fibrosis or cirrhosis.
Patients infected with Human Immunodeficiency Virus (HIV).
Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to romiplostim that contraindicates the patient's participation.
Known history of sensitivity or allergy to the active substance, to any of the excipients, or to any E. coli-derived product.
Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely.
Current pregnancy or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during this study.
Inability to understand investigational nature of the study or give informed consent.
What this trial measures
- To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24During 24 weeks of therapy
Proportion of participants with hematopoietic complete response at 24 weeks defined as hemoglobin ≥10 g/dL, ANC ≥1 x 10\^9/L, and platelet count ≥100 x 10\^9/L without having received transfusion of packed red blood cells within the last 6 weeks or platelets or G-CSF/GM-CSF within the last 2 weeks.