A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag

NCT06426043 | Not Yet Recruiting Phase 4

• 1 other identifier: LNA-2024
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.

Conditions

Aplastic Anemia

Keywords

Non-severe aplastic anemia; Lusutrombopag; Refractory; Recurrent

Outcome Measures

Primary Outcomes (2)

• Overall response rate at 3 months

  Proportion of patients who achieved complete response, partial response and hematological response

  3 month

• Overall response rate at 6 months

  Proportion of patients who achieved complete response, partial response and hematological response

  6 month

Secondary Outcomes (2)

• adverse event rate at 3 months

  3 month

• adverse event rate at 6 months

  6 month

Study Arms (1)

Lusutrombopag

EXPERIMENTAL

Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is \<20×10\^9/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10\^9/L and ≤200×10\^9/L, the dose can be maintained; when the platelet count is ≥200×10\^9/L and ≤400×10\^9/L, the daily dose can be reduced by 3mg; when the platelet count is \>400×10\^9/L, the drug can be suspended and resumed when the platelet count decreases to \<200×10\^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.

Drug: Lusutrombopag

Interventions

Lusutrombopag DRUG

Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is \<20×109/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10\^9/L and ≤200×10\^9/L, the dose can be maintained; when the platelet count is ≥200×10\^9/L and ≤400×10\^9/L, the daily dose can be reduced by 3mg; when the platelet count is \>400×10\^9/L, the drug can be suspended and resumed when the platelet count decreases to \<200×10\^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.

Lusutrombopag

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must be at least 18 years old, male or female.
• Participants must be diagnosed with NSAA and have a refractory/relapsed/intolerable response to standard-dose cyclosporine (CsA). The definition of refractory/relapsed is patients who have been treated with sufficient doses of cyclosporine (3-5mg/kg) for at least 6 months without response or relapse. The definition of intolerable is patients who cannot tolerate CsA and have stopped treatment due to significant side effects.
• Participants must meet the following criteria at enrollment: platelets \<30×109/L.
• Baseline liver and kidney function must be within 2 times of normal range.
• No active infection; no pregnancy or breastfeeding.
• Participants must agree to sign the informed consent form.
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.

You may not qualify if:

• Other causes of pancytopenia, such as myelodysplastic syndrome (MDS).
• Evidence of clonal hematopoietic system bone marrow disease (MDS, AML) with cytogenetics.
• PNH clone ≥50%.
• Received hematopoietic stem cell transplant (HSCT) prior to enrollment.
• Received ATG treatment within 6 months prior to enrollment.
• Infection or bleeding that cannot be controlled with standard therapy.
• Allergic to ruxolitinib.
• Active HIV, HCV, or HBV infection, cirrhosis, or portal hypertension.
• Any malignant tumor within 5 years, or local basal cell carcinoma of the skin.
• History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid syndrome) and current use of anticoagulants.
• Pregnant or breastfeeding (lactating) women.
• Participated in another clinical trial within 3 months.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (5)

• Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.

  PMID: 30354958 BACKGROUND

• Ruan J, Zuo W, Chen M, Yang C, Han B. Eltrombopag is effective in patients with relapse/refractory aplastic anemia-report from a single center in China. Ann Hematol. 2020 Dec;99(12):2755-2761. doi: 10.1007/s00277-020-04266-1. Epub 2020 Sep 17.

  PMID: 32944791 BACKGROUND

• Katsube T, Wajima T, Fukuhara T, Kano T. Effects of Food and Calcium Carbonate on the Pharmacokinetics of Lusutrombopag, a Novel Thrombopoietin Receptor Agonist. Clin Ther. 2019 Sep;41(9):1747-1754.e2. doi: 10.1016/j.clinthera.2019.06.004. Epub 2019 Jul 11.

  PMID: 31303281 BACKGROUND

• Hidaka H, Kurosaki M, Tanaka H, Kudo M, Abiru S, Igura T, Ishikawa T, Seike M, Katsube T, Ochiai T, Kimura K, Fukuhara T, Kano T, Nagata T, Tanaka K, Kurokawa M, Yamamoto K, Osaki Y, Izumi N, Imawari M. Lusutrombopag Reduces Need for Platelet Transfusion in Patients With Thrombocytopenia Undergoing Invasive Procedures. Clin Gastroenterol Hepatol. 2019 May;17(6):1192-1200. doi: 10.1016/j.cgh.2018.11.047. Epub 2018 Nov 28.

  PMID: 30502505 BACKGROUND

• Wan Z, Chen M, Han B. Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial. Ann Med. 2023 Dec;55(1):2224044. doi: 10.1080/07853890.2023.2224044.

  PMID: 37318085 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic; Recurrence

Interventions

lusutrombopag

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Bing Bing, PhD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bing Bing, PhD

CONTACT

13601059938; Hanbing_li@sina.com.cn

QLin Hu, PhD

CONTACT

15810785167; HQLin2012@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 17, 2024
• First Posted: May 23, 2024
• Study Start: June 1, 2024
• Primary Completion: June 1, 2025
• Study Completion: August 1, 2025
• Last Updated: May 23, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)