Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

NCT05399732 | Completed Phase 2 FDA Drug

• 1 other identifier: HanB-NSAA-lus
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 1

Brief Summary

Aplastic anemia (AA) is a rare bone marrow failure disease characterized by bone marrow hypocellularity and peripheral blood pancytopenia. AA is divided into severe AA (SAA) and non-severe AA (NSAA) based on the degree of cytopenia. The first line therapy for SAA or transfusion dependent NSAA is either immunosuppression therapy (IST) or hematopoietic stem cell transplantation (HSCT). Little attention has been paid to patients with anemia but not transfusion dependent, whose quality of life is significantly impaired due to the anemia and other complications.

Conditions

Aplastic Anemia

Keywords

NSAA; luspatercept; ORR; non-transfusion dependent

Outcome Measures

Primary Outcomes (1)

• overall response rate (ORR)

  Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR); HR defined as a hemoglobin increase from baseline of ≥1.5 g/dL for ≥2 weeks (in the absence of RBC transfusions)

  6 month

Secondary Outcomes (3)

• Hematologic response-erythroid（HR-E)

  6 month

• side effects

  1 year

• predictive factors

  6 month

Study Arms (2)

efficiency and safety in luspatercept plus cyclosporine

EXPERIMENTAL

luspatercept is at a dose of 1.0 mg per kilogram of body weight, administered subcutaneously every 3 weeks，and cyclosporine is at a dose of 3\~5mg/kg /day for at least 6 months.

Drug: Luspatercept; Drug: Cyclosporine

controll group in cyclosporine alone

ACTIVE COMPARATOR

cyclosporine is at a dose of 3\~5mg/kg /day for at least 6 months.

Drug: Cyclosporine

Interventions

Luspatercept DRUG

Patients in each group will be treated for at least 6 months and continue the treatment for an additional 6 months unless disease progress or have intolerable side effects.

efficiency and safety in luspatercept plus cyclosporine

Cyclosporine DRUG

Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.

Also known as: Ciclosporin, Cyclosporin

controll group in cyclosporine alone; efficiency and safety in luspatercept plus cyclosporine

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 year-old;
• hemoglobin level between 60g/L\~10 g/dL;
• newly diagnosed patients have at least one of the followings: #absolute neutrophil count \<1.5×109/L, #platelet count \< 30×109/L, # hemoglobin level \< 100g/L;
• with normal baseline liver and kidney function;
• with no active infection; are not pregnant or nursing;
• agree to sign consent forms;
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2

You may not qualify if:

• Congenital aplastic anemia;
• Presence of chromosomal aberration;
• Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
• Presence with PNH clone ≥50%;
• Patients received HSCT before;
• Uncontrolled infection or bleeding with standard treatment;
• Allergic to luspatercept CsA or accessories;
• HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
• Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or\<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(\>180/100mmHg)#pulmonary artery hypertension;
• Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
• Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
• Pregnant or nursing (lactating) woman;
• Have attended other clinical trials within 3 months

Sponsors & Collaborators

• Bing Han: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Peking union medical college hospital

Beijing, China

Location

Related Publications (7)

• Howard SC, Naidu PE, Hu XJ, Jeng MR, Rodriguez-Galindo C, Rieman MD, Wang WC. Natural history of moderate aplastic anemia in children. Pediatr Blood Cancer. 2004 Oct;43(5):545-51. doi: 10.1002/pbc.20131.

  PMID: 15382271 BACKGROUND

• Zhang ML, Chen WS, Han B. [Evaluation of the efficacy of cyclosporin A combined with recombined human thrombopoietin for treating patients with non-severe aplastic anemia]. Zhonghua Xue Ye Xue Za Zhi. 2020 Aug 14;41(8):637-642. doi: 10.3760/cma.j.issn.0253-2727.2020.08.004. Chinese.

  PMID: 32942816 BACKGROUND

• Chen WS, Zhang ML, Han B. [Evaluation of the Efficacy of Cyclosporin A Combined with Recombined Human Erythropoietin in the Treatment of Patients with Chronic Aplastic Anemia]. Zhongguo Yi Xue Ke Xue Yuan Xue Bao. 2021 Oct;43(5):736-742. doi: 10.3881/j.issn.1000-503X.13201. Chinese.

  PMID: 34728034 BACKGROUND

• Furlong E, Carter T. Aplastic anaemia: Current concepts in diagnosis and management. J Paediatr Child Health. 2020 Jul;56(7):1023-1028. doi: 10.1111/jpc.14996. Epub 2020 Jul 3.

  PMID: 32619069 BACKGROUND

• Desmond R, Townsley DM, Dunbar C, Young NS. Eltrombopag in aplastic anemia. Semin Hematol. 2015 Jan;52(1):31-7. doi: 10.1053/j.seminhematol.2014.10.002. Epub 2014 Oct 31.

  PMID: 25578417 BACKGROUND

• Matsuda K, Koya J, Arai S, Nakazaki K, Nakamura F, Kurokawa M. Cyclosporine Therapy in Patients with Transfusion-independent Non-severe Aplastic Anemia: A Retrospective Analysis. Intern Med. 2019 Feb 1;58(3):355-360. doi: 10.2169/internalmedicine.1372-18. Epub 2018 Aug 24.

  PMID: 30146592 BACKGROUND

• Drexler B, Passweg J. Current evidence and the emerging role of eltrombopag in severe aplastic anemia. Ther Adv Hematol. 2021 Mar 3;12:2040620721998126. doi: 10.1177/2040620721998126. eCollection 2021.

  PMID: 33747425 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

luspatercept; Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: professor

Model Details: Cyclosporine soft capsules（25mg） are produced by North China pharmaceutical Group Corporation

Study Record Dates

• First Submitted: May 24, 2022
• First Posted: June 1, 2022
• Study Start: December 19, 2022
• Primary Completion: February 1, 2025
• Study Completion: April 15, 2025
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 10years
• Access Criteria: email request

Locations

CN (1)