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

NCT ID: NCT06964971

Last Updated: 2025-05-11

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-20
• Study Completion Date: 2027-06-30

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Group1

basic treatment regimen + Luspatercept alone

Group Type: EXPERIMENTAL

Luspatercept

Intervention Type: 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 Group2

basic treatment regimen + Luspatercept combine with Deferasirox

Group Type: EXPERIMENTAL

Luspatercept combine with Deferasirox

Intervention Type: 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.

Interventions

Luspatercept

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).

Intervention Type: DRUG

Luspatercept combine with Deferasirox

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.

Intervention Type: DRUG

Eligibility Criteria

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).

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The First Affiliated Hospital of Zhejiang Chinese Medical University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The First Affiliated Hospital of Zhejiang Chinese Medical University

Hangzhou, Zhejiang, China

Countries

China

Central Contacts

Dijiong Wu, Ph.D.

Role: CONTACT

wudijiong@zcmu.edu.cn

+86 13989463963

Facility Contacts

Hangping Ge

Role: primary

canthy1216@126.com

+86 13738092542

Other Identifiers

2025-KLS-046-02

Identifier Type: -

Identifier Source: org_study_id