UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

NCT07001254 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 202308492
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage hematopoiesis in children and young adults.

Conditions

Aplastic Anemia

Keywords

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24

  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.

  During 24 weeks of therapy

Secondary Outcomes (2)

• Incidence of adverse events as assessed by CTCAE v5.0

  One year following completion of treatment

• Proportion of participants with a new cytogenetic abnormality

  One year following completion of treatment

Study Arms (2)

Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)

EXPERIMENTAL

Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).

Drug: Romiplostim; Drug: Immunosuppressive therapy (IST)

Relapsed or refractory SAA (Cohort B): Romiplostim

EXPERIMENTAL

Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.

Drug: Romiplostim

Interventions

Romiplostim DRUG

The investigational drug, Romiplostim, is a thrombopoietin receptor agonist (TPO-RA) that has been granted orphan drug designation by the FDA.

Relapsed or refractory SAA (Cohort B): Romiplostim; Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)

Immunosuppressive therapy (IST) DRUG

Standard of Care immunosuppressive therapy (IST) includes HORSE ANTI-THYMOCYTE GLOBULIN (H-ATG) and Cyclosporine (CSA)

Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)

Eligibility Criteria

• Age: 2 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• In order to be eligible to participate in this study, an individual must meet all of the following criteria:
• 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.
• Diagnosis of severe Aplastic anemia (newly diagnosed or refractory based on history of prior treatments) is established if bone marrow cellularity \<25% to 30% and at least two of the following criteria are met: (a) absolute neutrophil count \<0.5 × 10\^9
• /L, (b) platelet count \<20 × 10\^9/L, and (c) hemoglobin \<8 g/dL. In the event bone marrow cellularity is \>30% but patient presents with severe pancytopenia and its complications; the diagnosis of SAA will be considered at the discretion of PI. For relapsed or refractory AA, minor variations in hematological parameters will be acceptable, e.g., platelet count of \<50 x 10\^9/L or hemoglobin of ≤9 g/dL.
• OR Diagnosis of refractory aplastic anemia will include a confirmed diagnosis of SAA and the clinical assessment by the treating physician that the patient has not responded to the frontline IST by 6 months.
• OR Diagnosis of relapsed aplastic anemia will be determined by previous diagnosis of SAA and the prior history of successful hematologic response to IST with subsequent loss of response and/or requirement of supportive therapy\*.
• \*Adequate organ function within 7 days of enrollment defined as: Creatinine: ≤2.0 mg/dL Hepatic function: Elevation of liver enzymes is acceptable for patients with hepatitis-induced SAA if patient does not have history of chronic liver problem such as liver cirrhosis. If necessary, liver biopsy will be performed.
• 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).

You may not qualify if:

• An individual who meets any of the following criteria will be excluded from participation in this study:
• 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).

Sponsors & Collaborators

• Anjali Sharathkumar: lead
• Department of Health and Human Services: collaborator
• Food and Drug Administration (FDA): collaborator

Study Sites (1)

University of Iowa Health Care

Iowa City, Iowa, 52242, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

romiplostim; Immunosuppression Therapy

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques

Study Officials

• Anjali Sharathkumar, MD

  University of Iowa

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Anjali Sharathkumar, MD

CONTACT

319-384 5108; anjali-sharathkumar@uiowa.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: May 23, 2025
• First Posted: June 3, 2025
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): December 31, 2031
• Study Completion (Estimated): December 31, 2031
• Last Updated: December 19, 2025

Record last verified: 2025-12

Locations

US (1)