Status:
NOT_YET_RECRUITING
Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study
Lead Sponsor:
The First Affiliated Hospital of Zhejiang Chinese Medical University
Conditions:
Transfusion-dependent Non-severe Aplastic Anemia
Eligibility:
All Genders
18-80 years
Phase:
NA
Brief Summary
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic ...
Eligibility Criteria
Inclusion
- Age \>= 18 years; 2.According to the "Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition)", the patient must be diagnosed with transfusion-dependent non-severe aplastic anemia (TD-NSAA) and meet the requirement of erythroid hyperplasia in bone marrow aspiration from the posterior iliac crest and/or sternum being more than 15%; 3.If not newly diagnosed with TD-NSAA, and there are combined primary disease maintenance medications, the following conditions must be met:
- The patient has not received and does not consider HSCT or ATG treatment for at least the next six months;
- If maintaining oral immunosuppressive therapy, the course must be at least 6 months and assessed as ineffective;
- If maintaining androgen therapy, the course must be at least 3 months and assessed as ineffective;
- If maintaining recombinant human erythropoietin therapy, the course must be at least 3 months and assessed as ineffective;
- If maintaining thrombopoietin receptor agonist (TPO-RA) therapy, the duration must be \>=6 months with confirmed inefficacy, and a washout period of \>=1 month is required before study enrollment;
- If the above maintenance medication durations are not met, a washout period of at least 1 month is required; 4.Serum ferritin level \>= 1000 ng/ml; 5.Complete whole exome sequencing and MDS/AA next-generation sequencing testing are required.
Exclusion
- 1\. Severe hepatic dysfunction (ALT or AST ≥ 3 × ULN); 2.Severe renal impairment (eGFR \< 30 ml/min/1.73m² or patients with end-stage renal disease); 3.Cardiac disease, including New York Heart Association (NYHA) Class 3 or higher heart failure, or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization; 4.Patients with uncontrolled hypertension, with controlled hypertension according to NCI CTCAE version 5.0 considered as ≤ Grade 1 for this protocol; 5.Patients with a PNH clone \> 1%; 6.Patients planning to become pregnant or who are pregnant; 7.Surgical or clinical conditions that may significantly alter drug absorption, distribution, metabolism, or excretion (e.g., gastritis, ulcers, history of gastrointestinal or rectal bleeding; history of major gastrointestinal surgery); 8.Patients carrying congenital bone marrow failure-related gene mutations (homozygous or heterozygous, regardless of whether they are pathogenic/benign/likely benign/ of uncertain significance).
Key Trial Info
Start Date :
May 20 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 30 2027
Estimated Enrollment :
90 Patients enrolled
Trial Details
Trial ID
NCT06964971
Start Date
May 20 2025
End Date
June 30 2027
Last Update
May 11 2025
Active Locations (1)
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1
The First Affiliated Hospital of Zhejiang Chinese Medical University
Hangzhou, Zhejiang, China, 310000