Safety and Effectiveness of Trappa Ethanolamine Tablets Combined With Ciclosporin in the Treatment of Primary Treatment of Non-severe Aplastic Anemia

A Multicenter, Randomized, Double-blind, and Open-label, Placebo-controlled Phase II Clinical Study of Trappa Ethanolamine Tablets Combined With Ciclosporin in Patients With Treatment of Non-severe Aplastic Anemia

This is a multicenter, randomized, double-blind, and open-label, placebo-controlled phase II clinical study. In order to evaluate the effectiveness and safety of trappa ethanolamine tablets combined with ciclosporin in the treatment of patients with non-severe aplastic anemia.

Study Overview

• Status: Recruiting
• Conditions: Non-severe Aplastic Anemia
• Intervention / Treatment: Drug: Trappa ethanolamine tablets ; ciclosporin; Drug: Placebo ; ciclosporin

• Study Type: Interventional
• Enrollment (Estimated): 78
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Wanyi Zhai; Phone Number: 0518-82342973; Email: wanyi.zhai.wz5@hengrui.com

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 30000
      • Recruiting
      • Hematology Hospital of Chinese Academy of Medical Sciences
      • Principal Investigator: fengkui zhang

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 14-75 years old (including boundary value, whichever is at the time of signing the informed consent form), Gender is not limited.
2. Diagnosed with non-heavy aplastic anemia within 6 months
3. Life Functional Status (ECOG) Score 0-1
4. Those who understand the research procedures and methods, voluntarily participate in this experiment, and sign the informed consent form in writing

Exclusion Criteria:

1. Total blood cell loss and myelohypoproliferative diseases caused by other reasons
2. Chromosome karyotype analysis during screening shows clonal cytogenetic abnormalities
3. Meet the severe aplastic anemia
4. Paroxysmal sleep hemoglobinuria (PNH) clone ≥50% or hemolytic PNH clone
5. Randomly receive ATG, ciclosporin, TPO-R agonists, androgens and other drugs to treat the relapse without completing the elution
6. Pre-randomized treatment with erythropoietin
7. Pre-randomized use of corticosteroids, G-CSF and GM-CSF treatment
8. People with a history of hematopoietic stem cell transplantation
9. subjects who had deep vein thrombosis, myocardial infarction, cerebral infarction or peripheral arterial embolism in the first 12 months
10. Previous history of liver cirrhosis or portal hypertension
11. When screening, alanine aminotransferase, barley transaminase, total bilirubin and blood creatinine are higher than the upper limit of normal value
12. HIV infection or carrier in the past or screening; hepatitis C antibody positive; hepatitis B surface antigen positive or hepatitis B core antibody positive, and HBV-DNA test indicates virus replication
13. Patients with randomized bleeding and/or infection that are still uncontrollable after standardized treatment
14. Uncontrolled hypertension at screening, severe arrhythmia, level III/IV (graded by the New York Heart Association) congestive heart failure
15. Those who are known or suspected to be contraindicated or highly sensitive to Trappa ethanolamine API or cyclosporine
16. The subjects had any malignant solid tumors of the organ system in the first 5 years of screening, regardless of whether they had been treated, metastasis or relapsed, except for local skin basal cell carcinoma; subjects with blood tumors found in the past or screening
17. Pregnant or lactating women
18. Male subjects of women of childbearing age or partners of women of childbearing age refuse to use acceptable contraceptive measures from the period of taking the drug to 28 days after the last time of taking the drug.
19. Randomly participated in other clinical trials and took research drugs in the first 3 months.
20. The researchers believe that there is any situation that may cause the subject to be unable to complete the study or pose an obvious risk to the subject, or other factors that reduce the possibility of joining the group.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Trappa ethanolamine tablets combined with ciclosporin	Drug: Trappa ethanolamine tablets ; ciclosporin; Trappa ethanolamine tablets combined with ciclosporin
Placebo Comparator: Placebo combined with ciclosporin	Drug: Placebo ; ciclosporin; Placebo combined with ciclosporin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Full hematological response rate	About 13 weeks from the first medication to the evaluation.

Secondary Outcome Measures

Outcome Measure	Time Frame
Partial hematological response rate and total hematological response rate	About 13 weeks from the first medication to the evaluation
Hematological response rate	About 26 weeks from the first medication to the evaluation
The time required for the first hematological response	About 26 weeks from the first medication to the evaluation
Changes in platelet count relative to the baseline of each visiting point of view	About 26 weeks from the first medication to the evaluation
Changes in hemoglobin relative to the baseline of each visiting point of view	About 26 weeks from the first medication to the evaluation
Changes in absolute neutrophil count relative to the baseline of each visiting point of view	About 26 weeks from the first medication to the evaluation
Changes in reticulocyte count relative to the baseline of each visiting point of view	About 26 weeks from the first medication to the evaluation
Cumulative proportion of patients who need blood transfusion	About 26 weeks from the first medication to the evaluation
Cumulative total amount of platelet transfusions	About 26 weeks from the first medication to the evaluation
Cumulative amount of red blood cell infusions	About 26 weeks from the first medication to the evaluation
Cumulative proportion of patients with disease progression	About 26 weeks from the first medication to the evaluation

Collaborators and Investigators

• Sponsor: Jiangsu HengRui Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): April 26, 2023
• Primary Completion (Estimated): May 30, 2024
• Study Completion (Estimated): May 30, 2025

Study Registration Dates

• First Submitted: March 22, 2023
• First Submitted That Met QC Criteria: March 22, 2023
• First Posted (Actual): April 4, 2023

Study Record Updates

• Last Update Posted (Actual): September 1, 2023
• Last Update Submitted That Met QC Criteria: August 31, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Antirheumatic Agents; Immunosuppressive Agents; Immunologic Factors; Dermatologic Agents; Antifungal Agents; Calcineurin Inhibitors; Cyclosporine; Cyclosporins
• Other Study ID Numbers: SHR8735-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No