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

Phase 4

Not yet recruiting

• Conditions: Aplastic Anemia; Transfusion-dependent Anemia
• Interventions: Drug: rhTPO; Drug: Cyclosporin A

• Registration Number: NCT06525948
• Lead Sponsor: Peking Union Medical College Hospital

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

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Status Verified: 2024-07
• Study First Submitted: 2024-07-18
• Study First Submitted QC: 2024-07-26
• Last Update Submitted: 2024-07-26
• Study First Posted: 2024-07-29
• Last Update Posted: 2024-07-29
• Study Start: 2024-07-30
• Primary Completion: 2025-06-30
• Study Completion: 2025-12-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 54

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
two-drug combination group	rhTPO	rhTPO combined with cyclosporine
two-drug combination group	Cyclosporin A	rhTPO combined with cyclosporine
drug-free group	Cyclosporin A	cyclosporine alone

Primary Outcome Measures

Name	Time	Method
ORR	3 months	Overall Response Rate (ORR): meets the criteria for CR and PR.
CRR	3 months	Complete Response Rate (CRR)：ANC\>1.5×10\^9/L，Hb\>100g/L，PLT\>100×10\^9/L；

Secondary Outcome Measures

Name	Time	Method
ORR	6 months	Overall Response Rate (ORR): meets the criteria for CR and PR.
CRR	6 months	Complete Response Rate (CRR)：ANC\>1.5×10\^9/L，Hb\>100g/L，PLT\>100×10\^9/L；
Time for platelet count to recover to ≥20 x 10^9/L with a 1-fold increase in absolute value	through study completion, an average of 1 year	Time for platelet count to recover to ≥20 x 10\^9/L with a 1-fold increase in absolute
Proportion of patients with adverse events	through study completion, an average of 1 year	safety event

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China