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

NCT06525948 | Not Yet Recruiting Phase 4

• 1 other identifier: rTC-AA
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Aplastic Anemia; Transfusion-dependent Anemia

Outcome Measures

Primary Outcomes (2)

• ORR

  Overall Response Rate (ORR): meets the criteria for CR and PR.

  3 months

• CRR

  Complete Response Rate (CRR)：ANC\>1.5×10\^9/L，Hb\>100g/L，PLT\>100×10\^9/L；

  3 months

Secondary Outcomes (4)

• ORR

  6 months

• CRR

  6 months

• 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

• Proportion of patients with adverse events

  through study completion, an average of 1 year

Study Arms (2)

two-drug combination group

EXPERIMENTAL

rhTPO combined with cyclosporine

Drug: rhTPO; Drug: Cyclosporin A

drug-free group

ACTIVE COMPARATOR

cyclosporine alone

Drug: Cyclosporin A

Interventions

rhTPO DRUG

rhTPO

two-drug combination group

Cyclosporin A DRUG

cyclosporine A

drug-free group; two-drug combination group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who are dependent on transfusion therapy:
• Meet the Camitta NSAA criteria;
• 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.
• 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.

You may not qualify if:

• have used other clinical investigational drugs within 4 weeks;
• 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)
• history of cirrhosis or history of portal hypertension;
• 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;
• HIV infection;
• severe autoimmune disease or immunodeficiency disease;
• suffering from malignant tumour
• severe mental disorders;
• a known history of allergy to the drug components of this regimen;
• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (4)

• Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.

  PMID: 7616805 BACKGROUND

• Dancan GD. Spectrophotometry of tissue glycogen. Clin Chem. 1984 Sep;30(9):1580-1. No abstract available.

  PMID: 6467577 BACKGROUND

• 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.

  PMID: 28096088 BACKGROUND

• Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.

  PMID: 1813058 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Bing Han, PhD

  Peking Union Medical College

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bing Han, PhD

CONTACT

+8613601059938; Hanbing_li@sina.com.cn

Leyu Wang

CONTACT

18239490957; wangleyu_ys@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: chief physician

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: July 29, 2024
• Study Start: July 30, 2024
• Primary Completion: June 30, 2025
• Study Completion: December 30, 2025
• Last Updated: July 29, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)