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

NCT05323617 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: 20210112
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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+)

Conditions

Severe Aplastic Anemia (SAA)

Keywords

Severe Aplastic Anemia; SAA; Very Severe Aplastic Anemia; vSAA; Romiplostim

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (12)

• Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14

  Week 14

• 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

• Arms 1 and 2: number of participants with serious adverse events

  24 Weeks

• Arms 1 and 2: number of participants with clinically significant changes in laboratory values

  24 Weeks

• Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14

  Baseline and Week 14

Study Arms (2)

Arm 1: Previously Untreated IST

EXPERIMENTAL

Participants with SAA/vSAA that are previously untreated with IST.

Drug: Romiplostim; Drug: Antithymocyte Globulin; Drug: Cyclosporine A

Arm 2: Refractory IST

EXPERIMENTAL

Participants with SAA/vSAA that are refractory to IST.

Drug: Romiplostim

Interventions

Romiplostim DRUG

Administered as a subcutaneous injection.

Also known as: Nplate®

Arm 1: Previously Untreated IST; Arm 2: Refractory IST

Antithymocyte Globulin DRUG

Horse or rabbit antithymocyte globulin administered as an intravenous infusion.

Arm 1: Previously Untreated IST

Cyclosporine A DRUG

Administered orally.

Arm 1: Previously Untreated IST

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amgen: lead

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

romiplostim; Antilymphocyte Serum; Cyclosporine

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2022
• First Posted: April 12, 2022
• Study Start: August 31, 2023
• Primary Completion: February 25, 2025
• Study Completion: February 25, 2025
• Last Updated: September 29, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.