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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-31

Study Completion Date

2025-02-25

Brief Summary

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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+)

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Detailed Description

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Conditions

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Severe Aplastic Anemia (SAA)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm 1: Previously Untreated IST

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

Group Type EXPERIMENTAL

Romiplostim

Intervention Type DRUG

Administered as a subcutaneous injection.

Antithymocyte Globulin

Intervention Type DRUG

Horse or rabbit antithymocyte globulin administered as an intravenous infusion.

Cyclosporine A

Intervention Type DRUG

Administered orally.

Arm 2: Refractory IST

Participants with SAA/vSAA that are refractory to IST.

Group Type EXPERIMENTAL

Romiplostim

Intervention Type DRUG

Administered as a subcutaneous injection.

Interventions

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Romiplostim

Administered as a subcutaneous injection.

Intervention Type DRUG

Antithymocyte Globulin

Horse or rabbit antithymocyte globulin administered as an intravenous infusion.

Intervention Type DRUG

Cyclosporine A

Administered orally.

Intervention Type DRUG

Other Intervention Names

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Nplate®

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No