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New hope for tough anemia: drug boosts blood cells in failed cases

NCT ID NCT03988608

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called eltrombopag in 20 Chinese adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants had not responded to previous treatments. The drug was given daily for up to a year to see if it could improve blood cell counts and reduce the need for transfusions. The goal was to control the disease, not cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Dec 2019

Finished

May 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Chinese patients aged greater than or equal to 18 years old. * Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of \> 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab. * Platelet count ≤ 30 × 10\^9/L at screening. * Patient must not currently have the option of stem cell transplantation. * Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2. * Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or \<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site. Exclusion Criteria: * Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months. * Congenital aplastic anemia * AST or ALT ≥3 times the upper limit of normal. * Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome). * Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%. * Presence of chromosomal aberration (-7/7q- detected by fluorescence in situ hybridization (FISH), or other aberrations detected by G-band staining). * Evidence of a clonal hematologic bone marrow disorder on cytogenetics. * Past medical history of thromboembolism within 6 months or current use of anticoagulants. * Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years. * Patient with clinically significant (of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy) bacterial, fungal, mycobacterial, parasitic or viral infection (Patient with acute bacterial infections requiring antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed). * Patient with known hepatocellular disease * Presences of hepatitis B surface antigen (HBsAg), positive hepatitis C antibody test result at screening. * Cardiac disorder (NYHA) functional classification Grade II/III/IV * Past medical history of immediate or delayed hypersensitivity to compounds chemically similar to eltrombopag or their excipients. * Treatment with another investigational product within 30 days. * Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist. * Positive result for HIV (Human Immunodeficiency Virus) antibody test. * Pregnant or nursing (lactating) woman. * Woman of child-bearing potential.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Novartis Investigative Site

    Nanjing, Jiangsu, 210000, China

  • Novartis Investigative Site

    Nanchang, Jiangxi, 330006, China

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041, China

  • Novartis Investigative Site

    Tianjin, 300020, China

  • Novartis Investigative Site

    Tianjin, 300052, China

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