Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of TD-NSAA

Last updated: July 26, 2024
Sponsor: Peking Union Medical College Hospital
Overall Status: Active - Not Recruiting

Phase

4

Condition

Red Blood Cell Disorders

Aplastic Anemia

Anemia

Treatment

Cyclosporin A

rhTPO

Clinical Study ID

NCT06525948
rTC-AA
  • Ages > 18
  • All Genders

Study Summary

Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who aredependent on transfusion therapy:

  2. Meet the Camitta NSAA criteria;

  3. accompanied by at least one of the following abnormalities: (1) dependence oncomponent blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, theindication of component blood transfusion: HGB ≤ 60g / L; (2) PLT ≤ 10 × 10 ^ 9 / L,or PLT ≤ 30 × 10 ^ 9 / L with a significant tendency to bleed; (3) neutrophils ≤ 0.5 × 10 ^ 9 / L.

  4. Excluding other haematological and non-haematological diseases that causepancytopenia; 3. ECOG PS score 0-2, expected survival ≥ 3 months with follow-up; 4.functional levels of major organs must meet the following requirements: 1) Totalbilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanineaminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) bloodcreatinine (Cr) ≤ 1.5 x ULN; 5. has not been treated with platelet receptor agonist (TPO-RA) analogues and other immunosuppressant analogues; 6. the subject is notsuitable or willing to receive haematopoietic stem cell transplantation therapy; 7.no history of serious heart, lung, liver, kidney and other important organs andendocrine system diseases; 8. Voluntarily enroll in the study, sign the informedconsent, have good compliance and willing to cooperate with the follow-up.

Exclusion

Exclusion Criteria:

  1. have used other clinical investigational drugs within 4 weeks;

  2. a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleephaemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failuresyndromes (IBMFS), such as Fanconi's anaemia (FA) and congenital dyskeratosis (DC)

  3. history of cirrhosis or history of portal hypertension;

  4. congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiringmedication within 1 year prior to enrolment, or myocardial infarction or cerebralinfarction within 3 months prior to enrolment;

  5. HIV infection;

  6. severe autoimmune disease or immunodeficiency disease;

  7. suffering from malignant tumour

  8. severe mental disorders;

  9. a known history of allergy to the drug components of this regimen;

  10. in the opinion of the investigator, it is not appropriate to participate in thistrial, e.g., any other medical, social or psychological factors that may affectsafety or compliance with the study procedures.

Compliance with study procedures.

Study Design

Total Participants: 54
Treatment Group(s): 2
Primary Treatment: Cyclosporin A
Phase: 4
Study Start date:
July 30, 2024
Estimated Completion Date:
December 30, 2025

Study Description

Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of transfusion-dependent aplastic anaemia of the non-severe type

Connect with a study center

  • Peking Union Medical College Hospital

    Beijing, Beijing 100730
    China

    Site Not Available

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