Primary Immune Thrombocytopenia (ITP)Phase 2NCT06992128
Romiplostim N01 Combined With Glucocorticoids as the First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Multicenter, Interventional Trial
A Phase 2 study testing Romiplostim N01 combined with dexamethasone and Dexamethasone monotherapy in people with Primary Immune Thrombocytopenia (ITP), run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This prospective, multicenter, randomized study aim to evaluate the efficacy and safety of romiplostim N01 combined with glucocorticoids as the first-line treatment for newly diagnosed adult primary immune thrombocytopenia (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?
- Romiplostim N01 combined with dexamethasone and Dexamethasone monotherapy
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 129 participants
- Where
- 1 siteChina
- Study dates
- 22 May 2025to 31 Aug 2028
- Phase
- Phase 2
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
- Sign the written informed consent form before enrollment;
- Age ranging from 18 to 75 years old;
- Be clinically diagnosed with primary immune thrombocytopenia for less than 3 months before randomization;
- have not received any prior treatments for ITP.
- Have not received romiplostim treatment;
- ECOG PS score: 0 - 2;
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- Platelet value < 30×10^9/L;
- The expected survival period at the screening is ≥ 12 weeks;
- For subjects of reproductive age, agree to take reliable contraceptive measures throughout the study period (including male or female condoms, contraceptive foams, contraceptive gels, contraceptive membranes, contraceptive ointments, contraceptive suppositories, abstinence, and intrauterine device placement, etc.); Female subjects who have undergone hysterectomy, bilateral salpingectomy, bilateral tubal ligation or menopause for more than 1 year, and male subjects who have undergone bilateral vasectomy or ligation are excluded;
- Voluntarily join this study, sign the informed consent form, and have good compliance.
Who cannot join
- Suffering from other secondary thrombocytopenia except ITP, including but not limited to leukemia, thrombocytopenia caused by tumor treatment, myeloproliferative diseases, multiple myeloma, myelodysplastic syndrome, common variable immunodeficiency, and hereditary thrombocytopenia, etc.;
- Having undergone splenectomy before the first administration;
- Having received ITP drug treatment (including emergency treatment) before the first administration;
- Having used drugs with c-Mpl (thrombopoietin receptor) stimulating effects within 4 weeks before the first administration;
- Having received hematopoietic growth factor preparations (such as granulocyte colony-stimulating factor, macrophage colony-stimulating factor, erythropoietin, interleukin-11, etc.) within 4 weeks before the first administration;
- Having received antibody drugs (such as rituximab, etc.) within 14 weeks before the first administration;
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- Having received any Chinese herbal medicine or nutritional supplement (except vitamin supplements and mineral supplements) for the purpose of increasing platelets within 1 week before the first administration;
- Having been diagnosed with arterial thrombosis (such as cerebral thrombosis, transient ischemic attack or myocardial infarction), or having a history or complication of venous thrombosis (such as deep vein thrombosis, pulmonary embolism), or using anticoagulants or antiplatelet drugs at the beginning of screening;
- Having a history of severe cardiovascular diseases (such as grade III/IV congestive heart failure, arrhythmia or angina pectoris that increases the risk of thromboembolic events, unstable angina pectoris, having undergone coronary artery stent implantation, angioplasty or coronary artery bypass grafting);
- Secondary thrombocytopenia caused by autoimmune diseases such as antiphospholipid antibody syndrome, systemic lupus erythematosus, Hashimoto's thyroiditis, Even's syndrome and Sjogren's syndrome;
- Positive results for either human immunodeficiency virus antibody or syphilis antibody screening; positive hepatitis C antibody and HCV-RNA exceeding the upper limit of the study center's laboratory test; positive hepatitis B surface antigen and HBV-DNA exceeding the upper limit of the study center's laboratory test;
- Having participated in other clinical studies within 3 months before the first administration;
- Being pregnant or lactating, or having a pregnancy plan;
- Having fertility and being judged by the researcher as not fully adopting contraceptive measures;
- Having a history of severe drug allergic reactions or being known to be allergic to glucocorticoids or Nplate® (romiplostim) or the components of QL0911;
- Unable to comply for mental reasons;
- Judged by the researcher as not suitable to participate in this trial;
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
Yunfei Chen, +8618502220788, chenyunfei@ihcams.ac.cn
About this study
Official title: Romiplostim N01 Combined With Glucocorticoids as the First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia (ITP): A Multicenter, Interventional Trial
This prospective, multicenter, randomized study aim to evaluate the efficacy and safety of romiplostim N01 combined with glucocorticoids as the first-line treatment for newly diagnosed adult primary immune thrombocytopenia (ITP).
Read the detailed description
The primary endpoint of this study is to assess the efficacy of romiplostim N01 combined with glucocorticoids in untreated newly diagnosed adult ITP patients after 6 months of administration. 129 eligible subjects were randomized to either romiplostim N01 combining glucocorticoids or glucocorticoids treatment in 2:1 ratio. Administration protocol: Experimental group: Dexamethasone (HD-DXM) 40mg/d × 4d, one cycle. If there is no response on the 10th day, repeat once, administered orally or intravenously. Simultaneously, romiplostim N01 is administered, with an initial dose of 3µg/kg, by subcutaneous injection once a week, for up to 6 months. Control group: Dexamethasone (HD-DXM) 40mg/d × 4d, one cycle. If there is no response on the 10th day, repeat once, administered orally or intravenously. The initial dose of romiplostim N01 administration was 3µg/kg and can be initiated within 4 days of dexamethasone treatment. The dose of romiplostim N01 was adjusted according to the subject platelet count during the treatment period. When the platelet count is < 50 × 10^9/L, the patient will receive an increment in the dose of romiplostim N01 by 2µg/kg weekly, with a maximum dose of 10µg/kg. When 200 × 10^9/L > platelet count ≥ 50 × 10^9/L, the administration dosage remains unchanged. When 400 × 10^9/L > platelet count ≥ 200 × 10^9/L for two consecutive weeks, the dose is reduced by 1µg/kg. When the platelet count is ≥ 400 × 10^9/L, discontinue the drug. When the platelet count < 200 × 10^9/L, resume administration, and the administration dose is 1µg/kg less than before drug cessation. All subjects were followed up until the 24th week after the end of treatment through clinical follow-up or telephone follow-up. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events, concomitant medications and concomitant treatments are also recorded throughout the study. The researcher can increase the number of visits as necessary for AE follow-up to monitor the alleviation of AEs.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Romiplostim N01 combined with dexamethasone, Dexamethasone monotherapy
- Keywords
- ITP, Romiplostim, Continuous Remission, First-line Treatment
- 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.
Romiplostim N01 combined with dexamethasoneDrug
Dexamethasone (HD-DXM) at a dose of 40mg/d for 4 days constitutes one cycle. If there is no response on the 10th day, repeat it once. The administration can be either oral or intravenous. Meanwhile, romiplostim N01 is administered with an initial dose of 3µg/kg by subcutaneous injection within 4 days of dexamethasone treatment once a week for up to 6 months.
Dexamethasone monotherapyDrug
Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously.
| Study group | Role | What they receive |
|---|---|---|
| romiplostim N01 combined with glucocorticoids | Experimental | Not described Receives: Drug: Romiplostim N01 combined with dexamethasone |
| glucocorticoids | Active comparator | Not described Receives: Drug: Dexamethasone monotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of patients with continuous remission
Continuous remission is defined as the maintenance of the therapeutic effect of patients for at least 6 months since achieving remission, without the need for additional ITP-specific treatment.
Measured: 6 months
Secondary outcomes (9)
The total effective rate OR
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10^9/L and 100×10^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms).
Measured: 6 months
The proportion of patients with the initial response (reaching the effective standard within one month of the start of treatment)
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10^9/L and 100×10^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within one month).
Measured: one month of the start of treatment
The proportion of patients reaching the effective standard 3 months after the start of treatment
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10^9/L and 100×10^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 3 month).
Measured: 3 months after the start of treatment]
The proportion of patients reaching the effective standard 6 months after the start
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10^9/L and 100×10^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 6 month).
Measured: 6 months after the start of treatment
The proportion of patients reaching the effective standard 9 and 12 months after the start of treatment
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10^9/L and 100×10^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 9 and 12 month).
Measured: 9 and 12 months after the start of treatment
The maximum consecutive weeks of platelet response
The consecutive weeks with platelet count > 30×10^9/L in the absence of any rescue treatment.
Measured: 6 months
The proportion of subjects receiving rescue treatment.
The proportion of subjects receiving rescue treatment.
Measured: 6 months
According to the WHO bleeding score standard, the incidence and severity of bleeding symptoms.
According to the WHO bleeding score standard, the incidence and severity of bleeding symptoms
Measured: 6 months
The incidence of adverse events
Evaluated using Version 5.0 of the "Common Terminology Criteria for Adverse Events (NCI CTC AE)".
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2025
- Study start22 May 2025 (estimated)
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 129 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Collaborators
- Weifang People's Hospital, Shenzhen Second People's Hospital, The Affiliated Hospital of Qingdao University, Affiliated Hospital of Nantong University, The First Affiliated Hospital of Bengbu Medical University, The Second Affiliated Hospital of Dalian Medical University, Affiliated Hospital of North Sichuan Medical College, Changzhi Medical College, North China University of Science and Technology, Tianjin Hospital of ITCWM-Nankai Hospital, Baoding First Central Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06992128
- Sponsor's protocol ID
- QLMA-ITP-IIT-001
- Status last verified
- Apr 2025
- First submitted
- 19 May 2025
- Last updated
- 28 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No