Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy

Severe Aplastic Anemia (SAA) Clinical Trial

Official title:

Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Adult Severe Aplastic Anemia Participants Who Are Either Previously Untreated With IST or Refractory to IST

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT05323617
• Other study ID #: 20210112
• Status: Withdrawn
• Phase: Phase 2
• Start date: August 31, 2023
• Est. completion date: February 25, 2025

Study information

• Verified date: September 2023
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of participants with SAA/vSAA. The primary objectives of this study are to: Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L) Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: February 25, 2025
• Est. primary completion date: February 25, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Age = 18 years at time of enrollment
• Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies
• An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening
• Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA
• Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA

Exclusion Criteria:

• Diagnosed as having congenital aplastic anemia (AA) (Fanconi anemia, congenital dyskeratosis, etc)
• History of other malignancy within the past 5 years, with exceptions.
• Aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (PNH) (hemolytic predominant is defined as lactate dehydrogenase (LDH) > 1.5 x the upper limit of site normal
• Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab
• Previously treated with PEGylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin protein (TPO), romiplostim and other TPO-receptor agonist (eltrombopag, etc)
• Patients who are eligible for allogenic HCT and have an available matched related donor

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia (SAA)

Intervention

Drug:
• Romiplostim
Administered as a subcutaneous injection.
• Antithymocyte Globulin
Horse or rabbit antithymocyte globulin administered as an intravenous infusion.
• Cyclosporine A
Administered orally.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Arms 1 and 2: proportion of participants achieving any hematologic response at week 14	Proportion of participants achieving any hematologic response at week 14 based on response criteria: Platelet response; Erythroid response; Red blood cell count; Hemoglobin concentration; Neutrophil response	Week 14
Secondary	Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14		Week 14
Secondary	Arms 1 and 2: number of participants who have a decrease in frequency of platelet and/or red blood cell (RBC) transfusions, or become platelet and/or RBC transfusion independent at week 14		Week 14
Secondary	Arms 1 and 2: number of participants with serious adverse events		24 Weeks
Secondary	Arms 1 and 2: number of participants with clinically significant changes in laboratory values		24 Weeks
Secondary	Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14	The Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto is as follows: 0: No bleeding; Petecjoae or mucosal or retinal bleeding that did not require red-cell transfusion; Melena, hematemesis, hematuria, or hemoptysis; Any bleeding that required red-cell transfusion; Retinal bleeding accompanied by visual impairment; Nonfatal cerebral bleeding; Fatal cerebral bleeding; Fatal noncerebral bleeding	Baseline and Week 14
Secondary	Arms 1 and 2: serum romiplostim trough concentrations		Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24
Secondary	Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim		Weeks 1, 2, 4, 5, 9, 13, and 24
Secondary	Arms 1 and 2: area under the curve (AUC) of romiplostim		Weeks 1, 2, 4, 5, 9, 13, and 24
Secondary	Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim		Weeks 1, 2, 4, 5, 9, 13, and 24
Secondary	Arms 1 and 2: half-life (t1/2) of romiplostim		Weeks 1, 2, 4, 5, 9, 13, and 24
Secondary	Arms 1 and 2: number of participant with anti-romiplostim antibodies		Prior to romiplostim administration on Weeks 1 and 13
Secondary	Arms 1 and 2: number of participants with antibodies to thrombopoietin		Prior to romiplostim administration on Weeks 1 and 13

External Links

•   AmgenTrials clinical trials website