Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study

Not Applicable

Not yet recruiting

• Conditions: Transfusion-dependent Non-severe Aplastic Anemia
• Interventions: Drug: Luspatercept; Drug: Luspatercept combine with Deferasirox

• Registration Number: NCT06964971
• Lead Sponsor: The First Affiliated Hospital of Zhejiang Chinese Medical University

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 anemia, as well as to assess the safety and efficacy of this treatment approach.

The main questions it aims to answer is:

whether the combination therapy of Luspatercept and Deferasirox can improve hemoglobin levels in these patients.

Participants will receive Luspatercept every 3 to 5 weeks based on hemoglobin response, undergo complete blood counts every 1 to 3 weeks, and receive other necessary evaluations as required.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-04
• Study First Submitted: 2025-05-07
• Study First Submitted QC: 2025-05-07
• Last Update Submitted: 2025-05-07
• Study First Posted: 2025-05-11
• Last Update Posted: 2025-05-11
• Study Start: 2025-05-20
• Primary Completion: 2027-05-20
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

• 1.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:

  1. The patient has not received and does not consider HSCT or ATG treatment for at least the next six months;
  2. If maintaining oral immunosuppressive therapy, the course must be at least 6 months and assessed as ineffective;
  3. If maintaining androgen therapy, the course must be at least 3 months and assessed as ineffective;
  4. If maintaining recombinant human erythropoietin therapy, the course must be at least 3 months and assessed as ineffective;
  5. 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;
  6. 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 Criteria

• 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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment Group1	Luspatercept	basic treatment regimen + Luspatercept alone
Treatment Group2	Luspatercept combine with Deferasirox	basic treatment regimen + Luspatercept combine with Deferasirox

Primary Outcome Measures

Name	Time	Method
Hemoglobin	Baseline (1 month before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks), follow-up period: 48 weeks (once every 12 weeks).	Mild anemia: Defined as a hemoglobin level \<120 g/L in adult males or \<110 g/L in adult females, but \>90 g/L.; Moderate anemia: Hemoglobin level between 60-90 g/L. Severe anemia: Hemoglobin level between 30-60 g/L. Extremely severe anemia: Hemoglobin level \<30 g/L.

Secondary Outcome Measures

Name	Time	Method
Ferritin	Baseline (30 days before treatment), treatment phase: 12 weeks ( once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
platelets	Baseline (30 days before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks).
Proportion of patients transfusion-free for ≥8 weeks and ≥12 weeks	treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
Overall response rate (ORR; CR + PR) at Week 12 and Week 24 of treatment	Week 12 and Week 24	ORR: Overall Response Rate (defined as the proportion of patients achieving complete response \[CR\] or partial response \[PR\]).

Trial Locations

Locations (1)

The First Affiliated Hospital of Zhejiang Chinese Medical University; 🇨🇳 Hangzhou, Zhejiang, China