Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS

NCT06006949 | Unknown Phase 4

• 1 other identifier: Lus-2
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 1

Brief Summary

In a randomized controlled phase II/III clinical trial, 58% of patients with lower-risk MDS had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks after roxadustat treatment. In a randomized controlled phase III clinical trial, luspatercept significantly improved transfusion dependence in erythropoietin-stimulating agents (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life, compared to placebo. This study aimed to evaluate the efficacy and safety of roxadustat combined with luspatercept versus luspatercept monotherapy in the treatment of refractory MDS-RS.

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• overall response rate (ORR)

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

  6 month

Secondary Outcomes (2)

• transfusion indenpendence (TI)

  6 month

• adverse event rate

  6 month

Study Arms (2)

roxadustat and luspatercept

EXPERIMENTAL

Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg. Roxadustat (150mgqod) was administered for at least 6 months to evaluate efficacy. Hemoglobin ≥120g/L can be discontinued, and hemoglobin \<120g/L can continue to use. Those who are effective will continue to be given the combination therapy until ineffective or intolerant.

Drug: Roxadustat; Drug: Luspatercept

luspatercept

EXPERIMENTAL

Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg. Luspatercept was given for at least 6 months to evaluate the efficacy. Hemoglobin ≥120g/L can be discontinued, and hemoglobin \<120g/L can continue to use. Those who are effective will continue to be given the therapy until it is ineffective or intolerant

Drug: Luspatercept

Interventions

Roxadustat DRUG

Roxadustat (150 mgqod)

roxadustat and luspatercept

Luspatercept DRUG

Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg)

luspatercept; roxadustat and luspatercept

Eligibility Criteria

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

You may qualify if:

• Age \>18 years old.
• Patients with a definite diagnosis of MDS-RS and stratified as lower-risk according to IPSS-R.
• After at least 6 weeks of rhEPO treatment, with hemoglobin\<100g/L
• Adequate hepatic functions with alanine transaminase (ALT)/aspartate. transaminase (AST) levels within 2 times of the normal upper limit and total bilirubin levels within 2 times of the normal upper limit.
• ECOG≤2 with an expected life span of more than 6 months
• Documented patient consent.

You may not qualify if:

• Age \<18 years old.
• Complicated with active or uncontrolled infections.
• Complicated with other malignancies.
• Creatinine/transaminase ≥ 2 normal upper limit.
• Complicated with myelofibrosis.
• Pregnant or lactating women, or men with recent fertility needs
• Allergic to luspatercept or excipients
• Patients with history of polysorbate 80 allergy

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking union medical college hospital

Beijing, China

Location

Related Publications (13)

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  PMID: 31340116 BACKGROUND

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  PMID: 34724251 BACKGROUND

• Kubasch AS, Fenaux P, Platzbecker U. Development of luspatercept to treat ineffective erythropoiesis. Blood Adv. 2021 Mar 9;5(5):1565-1575. doi: 10.1182/bloodadvances.2020002177.

  PMID: 33687432 BACKGROUND

• Attie KM, Allison MJ, McClure T, Boyd IE, Wilson DM, Pearsall AE, Sherman ML. A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers. Am J Hematol. 2014 Jul;89(7):766-70. doi: 10.1002/ajh.23732. Epub 2014 Apr 26.

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• Suragani RN, Cadena SM, Cawley SM, Sako D, Mitchell D, Li R, Davies MV, Alexander MJ, Devine M, Loveday KS, Underwood KW, Grinberg AV, Quisel JD, Chopra R, Pearsall RS, Seehra J, Kumar R. Transforming growth factor-beta superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis. Nat Med. 2014 Apr;20(4):408-14. doi: 10.1038/nm.3512. Epub 2014 Mar 23.

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  PMID: 35220402 BACKGROUND

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

roxadustat; luspatercept

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Bing Han

CONTACT

13601059938; hanbing_li@sina.com.cn

Study Design

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

Study Record Dates

• First Submitted: August 17, 2023
• First Posted: August 23, 2023
• Study Start: August 1, 2023
• Primary Completion: August 1, 2024
• Study Completion: August 1, 2025
• Last Updated: August 23, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)