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

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Not yet enrolling; Phase 4

Conditions: Transfusion-dependent Anemia; Aplastic Anemia

Treatments: Drug: Cyclosporin A; Drug: rhTPO

Study type: Interventional

Funder types: Other

Identifiers

NCT06525948

rTC-AA

Details and patient eligibility

About

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

Full 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

Enrollment: 54 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

2. Meet the Camitta NSAA criteria;

3. accompanied by at least one of the following abnormalities: (1) dependence on component blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, the indication 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 cause pancytopenia; 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) Total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) blood creatinine (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 not suitable or willing to receive haematopoietic stem cell transplantation therapy; 7. no history of serious heart, lung, liver, kidney and other important organs and endocrine system diseases; 8. Voluntarily enroll in the study, sign the informed consent, have good compliance and willing to cooperate with the follow-up.

Exclusion criteria

1. have used other clinical investigational drugs within 4 weeks;
2. a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleep haemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failure syndromes (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 requiring medication within 1 year prior to enrolment, or myocardial infarction or cerebral infarction 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 this trial, e.g., any other medical, social or psychological factors that may affect safety or compliance with the study procedures.

Compliance with study procedures.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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54 participants in 2 patient groups

two-drug combination group

Experimental group

Description:

rhTPO combined with cyclosporine

Treatment:

Drug: rhTPO

Drug: Cyclosporin A

drug-free group

Active Comparator group

Description:

cyclosporine alone

Treatment:

Drug: Cyclosporin A