Clinical trial NCT06525948: Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of TD-NSAA
Official title: Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of Transfusion-dependent Non-severe Aplastic Anaemia (TD-NSAA)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE4
Conditions: Aplastic Anemia
Transfusion-dependent Anemia
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2024-07-29
Enrollment: 54
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed 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

Eligibility criteria: Inclusion Criteria:

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

1. Meet the Camitta NSAA criteria;
2. 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.
3. 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.

Primary outcomes:
measure: ORR
timeFrame: 3 months
measure: CRR
timeFrame: 3 months
Secondary outcomes:
measure: ORR
timeFrame: 6 months
measure: CRR
timeFrame: 6 months
measure: Time for platelet count to recover to ≥20 x 10^9/L with a 1-fold increase in absolute value
timeFrame: through study completion, an average of 1 year
measure: Proportion of patients with adverse events
timeFrame: through study completion, an average of 1 year
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Peking Union Medical College
name: Bing Han, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.
citation: Dancan GD. Spectrophotometry of tissue glycogen. Clin Chem. 1984 Sep;30(9):1580-1. No abstract available.
citation: Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481. Epub 2017 Jan 17.
citation: Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.