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

Part of paid clinical trials in Iowa City, Iowa.

Sponsor
Anjali Sharathkumar
Study ID
NCT07001254
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Romiplostim — DRUG
    The investigational drug, Romiplostim, is a thrombopoietin receptor agonist (TPO-RA) that has been granted orphan drug designation by the FDA.
  • Immunosuppressive therapy (IST) — DRUG
    Standard of Care immunosuppressive therapy (IST) includes HORSE ANTI-THYMOCYTE GLOBULIN (H-ATG) and Cyclosporine (CSA)

Study Details

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.

Key Dates

Start date
Aug 1, 2026
Status verified
Dec 2025
Primary completion
Dec 31, 2031
Completion
Dec 31, 2031

Study Design

Enrollment
15 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)
    Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).
  • Experimental: Relapsed or refractory SAA (Cohort B): Romiplostim
    Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.

Primary Outcome Measure

To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24 [ Time Frame: During 24 weeks of therapy ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Iowa Health CareIowa CityIowa52242
Anjali Sharathkumar, MD
[email protected]
319-384-5108
Anjali Sharathkumar, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Iowa City, IA

By research site
University of Iowa Health Care· Iowa City, IA

Related Studies