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

NCT06964971 | Not Yet Recruiting

• 1 other identifier: 2025-KLS-046-02
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

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.

Conditions

Transfusion-dependent Non-severe Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Hemoglobin

  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.

  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).

Secondary Outcomes (4)

• 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).

• Ferritin

  Baseline (30 days before treatment), treatment phase: 12 weeks ( 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

Study Arms (2)

Treatment Group1

EXPERIMENTAL

basic treatment regimen + Luspatercept alone

Drug: Luspatercept

Treatment Group2

EXPERIMENTAL

basic treatment regimen + Luspatercept combine with Deferasirox

Drug: Luspatercept combine with Deferasirox

Interventions

Luspatercept DRUG

The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks).

Treatment Group1

Luspatercept combine with Deferasirox DRUG

The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks). For patient with platelet counts \< 50×10⁹/L: Deferasirox dosage: 8-10 mg/kg/day, orally. For platelet counts ≥ 50×10⁹/L: Deferasirox dosage: 20 mg/kg/day, orally. Discontinue deferasirox if serum ferritin drops below 500 μg/L.

Treatment Group2

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• \. 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).

Sponsors & Collaborators

• The First Affiliated Hospital of Zhejiang Chinese Medical University: lead

Study Sites (1)

The First Affiliated Hospital of Zhejiang Chinese Medical University

Hangzhou, Zhejiang, 310000, China

Location

MeSH Terms

Interventions

luspatercept; Deferasirox

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Central Study Contacts

Dijiong Wu, Ph.D.

CONTACT

+86 13989463963; wudijiong@zcmu.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Control Group: Patients will continue with their original basic treatment regimen for the disease (immunosuppressants, androgens（either alone or in combination）) under observation. Treatment Group: In addition to the original basic treatment for the disease, patients will be further divided based on whether iron chelation therapy is used, into two subgroups: Luspatercept monotherapy group and Luspatercept combined with deferasirox group.

Study Record Dates

• First Submitted: May 7, 2025
• First Posted: May 11, 2025
• Study Start: May 20, 2025
• Primary Completion (Estimated): May 20, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: May 11, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)