Treatment-naïve Severe Aplastic AnemiaPhase 3NCT07345000
A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia
A Phase 3 study testing Romiplostim N01, Cyclosporine A (CsA) and pALG/ rATG in people with Treatment-naïve Severe Aplastic Anemia, run by Qilu Pharmaceutical Co., Ltd..
Trial summary
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Treatment-naïve Severe Aplastic Anemia
- What is being tested?
- Romiplostim N01, Cyclosporine A (CsA) and pALG/ rATG
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 210 participants
- Where
- No sites listed yet
- Study dates
- Jan 2026to Aug 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥15 years, regardless of sex (subjects ≥18 years old will be enrolled first; enrollment of subjects aged 15-18 years will commence after sufficient PK/PD data are obtained).
- Diagnosis of SAA or VSAA according to the British Journal of Haematology (BJH) guidelines. The diagnostic criteria for SAA are as follows:
- ①Bone marrow cellularity <25% of normal; or between 25% and <50%, with residual hematopoietic cells comprising <30%.
- ②Peripheral blood counts must meet at least two of the following three criteria (based on the lowest values from tests within 28 days prior to the first dose):
- Absolute neutrophil count (ANC) <0.5×10⁹/L
- Platelet count (PLT) <20×10⁹/L
- Absolute reticulocyte count (RET) <60×10⁹/L The diagnostic criterion for VSAA is: meeting the SAA criteria + ANC <0.2×10⁹/L.
- Written informed consent
Who cannot join
- History and/or concomitant presence of other primary or secondary bone marrow failure (BMF) syndromes, such as:
- ①Primary: Fanconi anemia, dyskeratosis congenita, congenital amegakaryocytic thrombocytopenia or Shwachman-Diamond syndrome, symptomatic paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), clonal cytopenia of undetermined significance (CCUS), antibody-mediated BMF, idiopathic cytopenia of undetermined significance (ICUS), etc.
- Secondary: large granular lymphocyte (LGL) leukemia, infiltration of the bone marrow by other systemic malignancies, myelofibrosis, and acute hematopoietic arrest, etc.
- Note: Asymptomatic PNH and hepatitis-associated SAA may be included if they meet all other inclusion criteria.
- Evidence of clonal cytogenetic abnormalities at screening.
- Participation in another clinical trial with investigational drugs or medical devices within 30 days prior to the first dose or within 5 half-lives of the investigational product (whichever is longer).
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- Previous use of any of the following agents prior to the first dose:
- ATG/ALG
- Alemtuzumab
- Mycophenolate mofetil ④Sirolimus
- Tacrolimus ⑥High-dose cyclophosphamide (≥45 mg/kg/day)
- Cumulative cyclosporine A (CsA) therapy exceeding 4 weeks prior to the first dose. If cumulative use is ≤4 weeks, a washout period of >14 days prior to the first dose is required.
- Cumulative use of thrombopoietin receptor agonists (TPO-RAs) for >14 days prior to the first dose, or cumulative use ≤14 days with a washout period of <14 days, including:
- Romiplostim / Nplate® (romiplostim)
- Eltrombopag ③Hetrombopag ④Recombinant human thrombopoietin, etc.
- Previous history of hematopoietic stem cell transplantation.
- Uncontrolled bleeding and/or infection after standard treatment prior to the first dose [defined as persistent signs/symptoms related to infection without improvement despite appropriate antibiotic and/or other therapy], or requiring intravenous (IV) antibiotic administration.
- Concomitant active CMV and EBV infection (positive test).
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Randomized, Controlled, Multicenter, Double-blind Phase III Study Evaluating the Efficacy and Safety of Romiplostim N01 Combined With Standard Immunosuppressive Therapy (IST) Versus Placebo Combined With IST in Treatment-naïve Subjects With Severe Aplastic Anemia
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
- Conditions
- Treatment-naïve Severe Aplastic Anemia
- Being tested
- Romiplostim N01, Cyclosporine A (CsA), pALG/ rATG, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Romiplostim N01Drug
Starting from Day 1 of Week 1, administer the initial dose of romiplostim N01: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.
Cyclosporine A (CsA)Drug
Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.
pALG/ rATGDrug
25 mg/kg/day on Days 1 through 5 of Week 1.
PlaceboDrug
Starting from Day 1 of Week 1, administer the initial dose of placebo: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.
Cyclosporine A (CsA)Drug
Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.
pALG/ rATGDrug
25 mg/kg/day on Days 1 through 5 of Week 1.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim N01+ IST | Experimental | Not described Receives: Drug: Romiplostim N01; Drug: Cyclosporine A (CsA); Drug: pALG/ rATG |
| Placebo+ IST | Active comparator | Not described Receives: Drug: Placebo; Drug: Cyclosporine A (CsA); Drug: pALG/ rATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response (CR) rate at the 6-month time point of treatment.
Proportion of subjects achieving hematopoietic complete response at the 6-month.
Hematopoietic complete response is defined as: hemoglobin ≥10 g/dL, absolute neutrophil count ≥1×10⁹/L, and platelet count ≥100×10⁹/L.
Measured: During 6 months of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2026
- Study startJan 2026 (estimated)
- Primary completionOct 2029 (estimated)
- Study completionAug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Treatment-naïve Severe Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Qilu Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 210 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Qilu Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07345000
- Sponsor's protocol ID
- QL0911-304
- Status last verified
- Jan 2026
- First submitted
- 23 Dec 2025
- Last updated
- 15 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Controlled, Multicenter, Double-blind Phase III Study Evaluating the Efficacy and Safety of Romiplostim N01 Combined With Standard Immunosuppressive Therapy (IST) Versus Placebo Combined With IST in Treatment-naïve Subjects With Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07345000
- Acronym / sponsor ID
- QL0911-304
- Conditions
- Treatment-naïve Severe Aplastic Anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- Qilu Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jan 2026 (estimated)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 15 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Romiplostim N01+ IST | Experimental | Drug: Romiplostim N01, Drug: Cyclosporine A (CsA), Drug: pALG/ rATG |
| Placebo+ IST | Active comparator | Drug: Placebo, Drug: Cyclosporine A (CsA), Drug: pALG/ rATG |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Age ≥15 years, regardless of sex (subjects ≥18 years old will be enrolled first; enrollment of subjects aged 15-18 years will commence after sufficient PK/PD data are obtained).
- Diagnosis of SAA or VSAA according to the British Journal of Haematology (BJH) guidelines. The diagnostic criteria for SAA are as follows:
- ①Bone marrow cellularity <25% of normal; or between 25% and <50%, with residual hematopoietic cells comprising <30%.
- ②Peripheral blood counts must meet at least two of the following three criteria (based on the lowest values from tests within 28 days prior to the first dose):
- Written informed consent
Key exclusion criteria (18 listed)
- History and/or concomitant presence of other primary or secondary bone marrow failure (BMF) syndromes, such as:
- ①Primary: Fanconi anemia, dyskeratosis congenita, congenital amegakaryocytic thrombocytopenia or Shwachman-Diamond syndrome, symptomatic paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), clonal cytopenia of undetermined significance (CCUS), antibody-mediated BMF, idiopathic cytopenia of undetermined significance (ICUS), etc.
- Note: Asymptomatic PNH and hepatitis-associated SAA may be included if they meet all other inclusion criteria.
- Evidence of clonal cytogenetic abnormalities at screening.
- Participation in another clinical trial with investigational drugs or medical devices within 30 days prior to the first dose or within 5 half-lives of the investigational product (whichever is longer).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Romiplostim N01Starting from Day 1 of Week 1, administer the initial dose of romiplostim N01: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg. | Drug | Romiplostim N01+ IST |
| Cyclosporine A (CsA)Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance. | Drug | Romiplostim N01+ IST |
| pALG/ rATG25 mg/kg/day on Days 1 through 5 of Week 1. | Drug | Romiplostim N01+ IST |
| PlaceboStarting from Day 1 of Week 1, administer the initial dose of placebo: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg. | Drug | Placebo+ IST |
| Cyclosporine A (CsA)Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance. | Drug | Placebo+ IST |
| pALG/ rATG25 mg/kg/day on Days 1 through 5 of Week 1. | Drug | Placebo+ IST |
Primary outcome
Complete Response (CR) rate at the 6-month time point of treatment.
Time frame: During 6 months of therapy
Proportion of subjects achieving hematopoietic complete response at the 6-month. Hematopoietic complete response is defined as: hemoglobin ≥10 g/dL, absolute neutrophil count ≥1×10⁹/L, and platelet count ≥100×10⁹/L.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Romiplostim N01, Cyclosporine A (CsA) and pALG/ rATG in people with Treatment-naïve Severe Aplastic Anemia, run by Qilu Pharmaceutical Co., Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 15 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact jun shi, Professor (022-23608326, shijun@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Response (CR) rate at the 6-month time point of treatment. (time frame: During 6 months of therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2026; AzTrials last refreshed its copy on 26 Oct 2026.