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Clinical Trials in China / NCT05664737
Active, not recruiting Phase 2

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

NCT05664737 · tracked via the Priya Life Science China tracker
Sponsor
Bristol-Myers Squibb
Phase
Phase 2
Started
2022-12-09
Last updated
2026-09-10

Condition(s) studied

Anemia

Investigational drug(s) / intervention(s)

LuspaterceptPlacebo

Luspatercept: Specified dose on specified days

Placebo: Specified dose on specified days

Study summary

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

Eligibility

Sex
ALL
Min age
12 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:. i) TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization. ii) NTD participant:\< 6 RBC units during the 24 weeks prior to randomization(transfusion due to conditions other than A-Thal will not be considered)and, RBC transfusion-free during at least 8 weeks prior to randomization(unless transfusion was required to treat an acute medical condition other than A-Thal) and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. * Adult participant has Eastern Cooperative Oncology Group (ECOG) 34 score of 0 or 1. * Adolescent participant 12 years to \< 18 years with documented diagnosis of A-Thal HbH disease with transfusion dependence defined as:. i) TD participant: ≥ 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for \> 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years. ii) NTD participant:\< 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded. iii) Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening. Exclusion Criteria: * Medical Conditions: Diagnosis of A-ThalTrait, Hb Bart hydrops, ATRx A-Thal, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Undergone episodes of hemolysis not related to A-Thal within the 8 weeks prior to randomization. Applicable for the EU only: Bleeding disorders manifested by frequent bleeding episodes (eg, menorrhagia, epistaxis, clotting disorders). * Participant has deep vein thrombosis (DVT), stroke or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24weeks prior to randomization. * Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment. * Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding. * Prior/Concomitant: Undergone HSCTs or gene therapy (candidates for HSCT or gene therapy with waiting period of ≥ 12 months are eligible). * Use of hydroxyurea treatment ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants. * Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to control the growth of EMH mass(es) during the screening period. * Any medical or psychiatric condition (including active infections, recent surgery, sequelae of diseases or interventions, clinically significant laboratory abnormalities or concurrent treatment) that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data. * Other protocol-defined inclusion/exclusion criteria apply.

Primary outcome measure(s)

Trial sites (31)

FacilityCityRegionStatus
Local Institution - 0029 Guangzhou GD
Local Institution - 0015 Guangzhou GD
Local Institution - 0013 Foshan Guangdong
Local Institution - 0017 Maoming Shi Guangdong
Local Institution - 0030 Shenzhen Shi Guangdong
Local Institution - 0023 Liuzhou Guangxi
Local Institution - 0014 Nanning GX
Local Institution - 0011 Haikou Hainan
Local Institution - 0012 Kunming Yunnan
Local Institution - 0002 Haikou China
Local Institution - 0016 Nanning China
Local Institution - 0018 Rio G
Local Institution - 0006 Athens Greece
Local Institution - 0009 Goudi Greece
Local Institution - 0007 Larissa Greece
Local Institution - 0025 Hong Kong HK
Local Institution - 0026 Genova GE
Local Institution - 0020 Orbassano TO
Local Institution - 0028 Naples Italy
Local Institution - 0019 Naples Italy
Local Institution - 0032 Kuala Lumpur WP
Local Institution - 0033 Johor Bahru Malaysia
Local Institution - 0034 Riyadh Saudi Arabia
Local Institution - 0036 Singapore Singapore
Local Institution - 0010 Kaohsiung City KHH
Local Institution - 0004 Nan Gang Qu TPE
Local Institution - 0003 Taichung TXG
Local Institution - 0001 Bangkok Noi Bangkok
Local Institution - 0031 Mueang Phitsanulok Thailand
Local Institution - 0027 Altındağ Turkey (Türkiye)
Local Institution - 0021 Topkapı Turkey (Türkiye)

More Bristol-Myers Squibb trials in China

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05664737 on ClinicalTrials.gov ↗ ← All trials in China