Skip to main content
OpenTrials
Not Yet Recruiting

NCT Number: NCT07548671

Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA

This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Transfusion-dependent Non-severe Aplastic Anemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 years old;
  • Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD NSAA) according to the Camitta criteria; Hemoglobin<90g/L;
  • Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
  • With baseline liver and kidney functions <2 ULN;
  • ECOG score ≤ 2;
  • Signed the informed consent;

Exclusion criteria

  • Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
  • With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
  • PNH clone ≥ 50%;
  • Received HSCT before enrollment;
  • Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs), roxadustat;
  • Allergic or intolerant to enarodustat or CsA;
  • Pregnant or lactating patients;
  • Severe bleeding or infection that cannot be controlled by standard treatment;
  • Complicated with malignant tumors;
  • Participated in other clinical trials within 3 months;
  • Patients considered not suitable to participate in this study by the investigator.

Treatment and study plan

Enarodustat

Drug

Enarodustat 8mg qd

Cyclosporine (CsA)

Drug

CsA 3-5mg/kg/d, trough concentration 100-200ng/ml

Primary outcomes

  1. ORR

    Time frame: 6-month

    ORR=CRR+PRR

Secondary outcomes

  1. ORR

    Time frame: 3-month, 12-month

    ORR=CRR+PRR

  2. RBC-TI rate

    Time frame: 3-month, 6-month, 12-month

    Proportion of patients who achieve red blood cell transfusion independence for 8 weeks or longer

  3. hemoglobin response rate

    Time frame: 3-month, 6-month, 12-month

    Proportion of patients with hemoglobin response

  4. AE rate

    Time frame: through study completion, an average of 1 year

    According to CTCAE, the proportion of patients with adverse events (AEs)

Study contacts

Contact information is provided by the study sponsor or research team.

Bing Han

CONTACT

[email protected]

+86 13601059938

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Registry information

Official study title

Efficacy and Safety of Enarodustat Combined With Cyclosporine Versus Cyclosporine Monotherapy in the Treatment of Newly Diagnosed TD-NSAA

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Apr 23, 2026
Registry last updated
Apr 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.