Primary Immune Thrombocytopenia (ITP)Phase 3NCT05029635
Phase III Study on HMPL-523 for Treatment of ITP
A Phase 3 study testing HMPL-523 in people with Primary Immune Thrombocytopenia (ITP), run by Hutchmed at 37 sites in China.
Trial summary
The purpose of this study is to determine whether HMPL-523 (sovleplenib) is safe and effective in the treatment of chronic Immune Thrombocytopenic Purpura (ITP). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- HMPL-523
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 272 participants
- Where
- 37 sitesChina
- Study dates
- 27 Oct 2021to 18 Sep 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntary signature of written informed consent form;
- Male or female aged 18~75 years;
- Performance Status score [Eastern Cooperative Oncology Group (ECOG) score] 0~1;
- Having been diagnosed as ITP prior to randomization, and duration of disease is more than 6 months;
- Intolerance or insufficient response, or recurrence after at least one anti-ITP standard drug therapy;
- Patients must have a history of response to previous ITP therapy;
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- One combined anti-ITP therapy is allowed in this study, however, the following criteria need to be met:
- The dose of glucocorticoid has been stable for 4 weeks prior to randomization (<20 mg Prednisone equivalent);
- The dose of Danazol has been stable for 3 months prior to randomization;
- The dose of immunosuppressant (only including Azathioprine, Ciclosporin A, Mycophenolate mofetil) has been stable for 3 months prior to randomization.
- The condition is relatively stable; WHO bleeding scale grade is 0-1; no emergency treatment is expected within 2 weeks as judged by investigators.
- The laboratory examinations need to meet the following conditions (no treatment for this abnormal variable is given within one week prior to blood collection):
- Average platelet count <30×10^9 /L (and none > 35×10^9 /L unless as a result of rescue therapy) from at least 3 qualifying counts;
- Hemoglobin ≥100 g/L, neutrophil count >1.5×10^9/L;
- Total bilirubin (TBIL), alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤1.5×upper limit of normal (ULN);
- Serum creatinine concentration ≤1.5×ULN and creatinine clearance ≥50 mL/min;
- Serum amylase and lipase ≤1.5×ULN;
- International normalized ratio (INR), activated partial thromboplastin time (APTT) not exceeding 20% of normal range.
- Male or female patients of childbearing potential must agree to use effective contraceptive methods during the study and within 90 days after last dose of study drug, e.g., double barrier contraceptive method, condom, oral or injectable contraceptives, intrauterine device, etc. Postmenopausal women (>50 years old and no menses for >1 year) and surgically sterilized women are not subject to this condition.
- Voluntary signing of the ICF for the sub-study;
- Performance status score (ECOG score) 0-1;
- Relatively stable disease, WHO bleeding grade 0-1;
- Female patients of childbearing potential must agree to use highly effective treatment of contraception from screening until 30 days after discontinuation of study treatment of;
- Male patients with fertile female partners must consent to use barrier contraception during the study period and for 30 days after the termination of study treatment.
Who cannot join
- Evidence on the presence of secondary causes of immune thrombocytopenia;
- Clinically serious hemorrhage requiring immediate adjustment of platelet (e.g., hypermenorrhea with significantly decreased hemoglobin);
- Clinically symptomatic gastrointestinal hemorrhage within 6 months prior to screening visit (e.g., haematemesis, tarry stool, however, the positive occult blood test without any sign or symptom of gastrointestinal hemorrhage will not be considered as "clinically symptomatic", or hemorrhoids hemorrhage is one exception);
- known history of vital organ transplantation or hematopoietic stem cell / bone marrow transplantation;
- Has received live vaccine within 8 weeks prior to Day 1 (baseline visit); or plan for immunization with live vaccine during the study;
- Splenectomy within 12 weeks prior to randomization;
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- Major surgery within 4 weeks prior to the randomization, or plan for major elective surgery during the study;
- Previous history of malignant tumors (except for the basal cell carcinoma of skin or cervical carcinoma in situ that have been cured);
- History of important arterial / venous embolic disease;
- Intracranial hemorrhage within 6 months before screening visit;
- History of serious cardiovascular disease (e.g., grade III/IV congestive heart failure, arrhythmia or angina pectoris requiring drug therapy, unstable angina pectoris, intracoronary stent implantation, angioplasty or coronary artery bypass grafting, or QTc ≥450 ms);
- Hypertension that can not be controlled with drugs (systolic blood pressure ≥140 mmHg or diastolic blood pressure ≥90 mmHg);
- Previous history of serious gastrointestinal disease, such as dysphagia, active gastric ulcer, inability to take drugs orally or absorption disorder for oral drugs;
- Human immunodeficiency virus (HIV) infection, or hepatitis B (in case of positive HBsAg or HBcAb, positive HBV DNA needs to be determined), or hepatitis C (positive HCV RNA), or liver cirrhosis;
- Significant active infection that is not controlled clinically (e.g., sepsis, pneumonia or abscess), or serious infection within 6 weeks prior to randomization (leading to hospitalization or requiring treatment with antibiotic injections);
- Has received rescue therapy for ITP within 2 weeks prior to randomization; Has received the treatment for the objective of increasing platelet within 4 weeks prior to randomization (including but not limited to glucocorticoid, thrombopoietin, thrombopoietin receptor agonist, Cyclosporine A, Mycophenolate mofetil, etc.), except those meeting the inclusion criterion 7;
- Having received Rituximab within 14 weeks prior to randomization;
- Having received traditional Chinese medicine within 1 week prior to randomization;
- Requiring long-term/continuous use of the drugs that may affect platelet function [including but not limited to aspirin, Clopidogrel, ticagrelor, NSAIDs, etc.], or anticoagulants;
- Intake of potent CYP3A inhibitor or inducer, as well as sensitive or narrow therapeutic window substrates of CYP3A, CYP1A2 or CYP2B6 two weeks (three weeks for Hypericum perforatum) or 5 half-lives prior to randomization (whichever is longer);
- Having participated in the clinical study for drugs or invasive medical device 4 weeks prior to randomization (or within 5 half-lives of the study drug prior to randomization, whichever is longer);
- Having received spleen tyrosine kinase Syk inhibitor (e.g., Fostamatinib) previously;
- Known allergy to the active ingredient or excipient of study drug;
- Presence of serious psychological or mental disorder;
- Alcoholic or drug abuser;
- Female patients in pregnancy or breast feeding;
- Being unsuitable to participate in this study, as considered by investigators.
- History of significant arterial/venous embolic disease;
- History of serious cardiovascular disease；
- Hypertension uncontrolled by medications；
- Known hypersensitivity to the active ingredient or excipients of the study drug;
- Patients with severe psychological or mental disorders;
- Alcoholics or drug abusers;
- Female patients who are pregnant and lactating;
- Patients who, in the opinion of the investigator, are not suitable for this study.
Trial locations and contacts
37 sites in 1 country.
37 sites in 1 country
China37 sites
Beijing Chaoyang Hospital of Capital Medical University
Beijing, Beijing Municipality
Peking Union Medical College Hospital
Beijing, Beijing Municipality
People's Hospital of Peking University
Beijing, Beijing Municipality
The Third Xiangya Hospital of Central South University
Changsha, Hunan
Xiangya Hospital Central South University
Changsha, Hunan
Heping Hospital Affiliated to Changzhi Medical College
Changzhi, Shanxi
West China Hospital，Sichuan University
Chengdu, Sichuan
Fujian Medical University Union Hospital
Fuzhou, Fujian
Guangdong General Hospital
Guangzhou, Guangdong
Southern Hospital of Southern Medical University
Guangzhou, Guangdong
Zhejiang Provincial Hospital of Chinese Medicine
Hangzhou, Zejiang Province
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui
The First Affiliated hospital of USTC
Hefei, Anhui
The Affiliated Huai'an No.1 People's Hospital of Nanjing Medical University
Huai'an, Nanjing Province
Jinan Central Hospital Affilated to Sandong University
Jinan, Shandong
The second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan
Lanzhou University Second Hospital
Lanzhou, Gansu
LiaoCheng People's Hospital
Liaocheng, Shandong
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi
The First Affiliated Hospital Of GuangXi Medical University
Nanning, Guangxi
The Affiliated Hospital of Qingdao University
Qingdao, Shandong
Jinshan Hospital Affiliated To Fudan University
Shanghai, Shanghai Municipality
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality
Shengjing Hospital of China Medical University
Shenyang, Liaoning
The Second People's Hospital Of Shenzhen
Shenzhen, Guangdong
The First Hospital of Hebei Medical University
Shijiazhuang, Hebei
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu
The second hospital of Shanxi Medical University
Taiyuan, Shanxi
Affiliated Hospital of North China University of Technology
Tangshan, Hebei
Blood Institute of the Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality
The First Affilicated Hospital of Xinjiang Medical University
Ürümqi, The Xinjiang Uygur Autonomous Region
Union Hospital affiliated to Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei
Shanxi Provincial People's Hospital
Xi’an, Shanxi
Xiangyang Central Hospital
Xiangyang, Hubei
Qinghai province people's hospital
Xining, Qinghai
First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan
Henan Cancer Hospital
Zhengzhou, Henan
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of HMPL-523 in Treatment of Primary Immune Thrombocytopenia (ITP) in Adults (ESLIM-01 Study)
The purpose of this study is to determine whether HMPL-523 (sovleplenib) is safe and effective in the treatment of chronic Immune Thrombocytopenic Purpura (ITP).
Read the detailed description
This study consists of a double-blind phase (primary study) followed by an open-label phase (sub-study). The double-blind phase(n=188) is a randomized, double-blind, placebo-controlled phase III clinical trial in adult patients with primary ITP to determine whether HMPL-523 (sovleplenib) is safe and effective in the treatment of chronic ITP. The sub-study provides open-label HMPL-523 treatment to assess long-term safety and efficacy. The open-label phase have Cohort1 and Cohort 2. Cohort 1 enrolled patients from primary study who have received double-blind study treatment for 12 weeks with sustained platelet count <50×10⁹/L or have received double-blind study treatment for 24 weeks. Cohort 2 enrolled primary ITP patients(n=84) with disease duration >3 months.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- HMPL-523, Placebo
- Keywords
- HMPL-523, sovleplenib, ITP, ESLIM-01
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HMPL-523Drug
Also called Sovleplenib
HMPL-523(300mg PO QD)
PlaceboDrug
Placebo(300mg PO QD)
| Study group | Role | What they receive |
|---|---|---|
| Drug: HMPL-523 | Active comparator | Primacy study (Randomized, Double-Blind Phase): Eligible subjects receive 300 mg HMPL-523 treatment once daily for 24 weeks.
Sub study (Open-label Phase): Eligible subjects receive 300 mg HMPL-523 treatment once daily for 76 weeks after the enrollment of the last patient enrolled in open-label phase. Receives: Drug: HMPL-523 |
| Drug: placebo | Placebo comparator | Primacy study (Randomized, Double-Blind Phase): Eligible subjects will receive 300 mg HMPL-523 matched placebo treatment once daily for 24 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
the durable response rate in the primary study
Platelet count ≥50×10^9 /L on at least 4 of 6 scheduled visits of Week14-Week24 in the primary study
Measured: treatment period Week14-Week24
Secondary outcomes (5)
the overall response rate in the primary study
At least one platelet count ≥50×10^9 /L (except that induced by the rescue therapy) in the 24-week double-blind treatment period
Measured: treatment period Week1-Week24 in the primary study
Incidence of treatment emergent adverse events
Adverse events classified according to NCI CTCAE version 5.0
Measured: treatment period Week1-Week24 in the primary study
Plasma concentration at steady state 2 hours post dose (C2h,ss)
Plasma concentration of HMPL-523 and its main metabolites at steady state 2 hours post dose (C2h,ss) will be determined.
Measured: treatment period Week1-Week24 in the primary study
Plasma concentration at steady state 2 hours post dose (C4h,ss)
Plasma concentration of HMPL-523 and its main metabolites at steady state 4 hours post dose (C4h,ss) will be determined.
Measured: treatment period Week1-Week24 in the primary study
Plasma concentration at steady-state trough concentration (Cmin,ss)
Plasma concentration of HMPL-523 and its main metabolites at steady-state trough concentration (Cmin,ss) will be determined.
Measured: treatment period Week1-Week24 in the primary study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2021
- Study start27 Oct 2021
- Primary completion16 May 2025
- Study completion18 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 272 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hutchmed
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Renchi Yang, professor, Principal investigator, offices director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05029635
- Sponsor's protocol ID
- 2020-523-00CH1
- Status last verified
- Apr 2026
- First submitted
- 8 Aug 2021
- Last updated
- 4 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hu Y, Liu X, Zhou H, Wang S, Huang R, Wang Y, Du X, Sun J, Zhou Z, Yan Z, Chen W, Wang W, Liu Q, Zeng Q, Gong Y, Yin J, Shen X, Ye B, Chen Y, Xu Y, Sun H, Cheng Y, Liu Z, Wang C, Yuan G, Zhang X, Li X, Cheng P, Guo X, Jiang Z, Yang F, Yang L, Luo C, Xiao T, Fu S, Yin H, Guo X, Xu Q, Fan S, Shi MM, Su W, Mei H, Yang R. Efficacy and safety of sovleplenib (HMPL-523) in adult patients with chronic primary immune thrombocytopenia in China (ESLIM-01): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Haematol. 2024 Aug;11(8):e567-e579. doi: 10.1016/S2352-3026(24)00139-X. Epub 2024 Jun 14.
PubMed 38885672