Aplastic AnemiaPhase 2NCT06613880
Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
A Phase 2 study testing standard IST combined with Romiplostim N01 in people with Aplastic Anemia and TPO Receptor Agonists, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Aplastic Anemia, TPO Receptor Agonists
- What is being tested?
- standard IST combined with Romiplostim N01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 24 Oct 2024to 30 Sep 2027
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥ 12 years
- Diagnosis of severe or very severe aplastic anemia
- Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
- ECOG performance status ≤2
- Willing and able to comply with the requirements for this study and written informed consent.
Who cannot join
- Received > 4 weeks of TPO-RA drug before treatment
- Received > 4 weeks of immunosuppressive therapy before treatment
- History of thromboembolic disease
- Intolerance to Romiplostim N01 or cyclosporine
- Allergy to ALG
- Presence of uncontrolled active infection
Show all 12Show fewer
- Presence of uncontrolled hypertension (≥140/90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol/L or random blood glucose ≥ 11.1 mmol/L)
- Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) > 3 ULN, creatinine ≥ 2.5 ULN
- History of chemoradiotherapy for malignant solid tumors
- History of other systemic serious illnesses
- Females who are pregnant/lactating or need pregnancy
- Patients considered to be ineligible for the study by the investigator for reasons other than the above
Trial locations and contacts
1 site in 1 country.
China1 site
Regenerative Medicine Center and Red Blood Cell Disorders Center
Tianjin, Tianjin Municipality
About this study
Official title: A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.
- Conditions
- Aplastic Anemia, TPO Receptor Agonists
- Being tested
- standard IST combined with Romiplostim N01
- Keywords
- Severe aplastic anemia, Romiplostim N01
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
standard IST combined with Romiplostim N01Drug
Romiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation.
| Study group | Role | What they receive |
|---|---|---|
| p-ALG+Romiplostim N01+CsA | Experimental | The maximum screening period was 4 weeks, and after entering the treatment period, the patients were treated with p-ALG combined with cyclosporine and Romiplostim N01. Symptomatic leukocyte elevation, blood transfusion support, and anti-infective therapy are allowed during this period, according to the patient's blood routine. If a patient has a serious adverse event during treatment, it will be treated according to the criteria for suspension and recovery. Receives: Drug: standard IST combined with Romiplostim N01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Measured: Within 27 weeks post treatment
Secondary outcomes (3)
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Measured: Within 3 months post treatment
Time to hematological response
Duration time was calculated from enrollment to achieve hematological response.
Measured: Within 27 weeks post treatment
Incidence of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Measured: Within 27 weeks post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2024
- Study start24 Oct 2024
- Primary completion26 Sep 2025
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Qilu Pharmaceutical Co., Ltd.
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06613880
- Sponsor's protocol ID
- IIT2024070
- Status last verified
- Nov 2026
- First submitted
- 23 Sep 2024
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06613880
- Acronym / sponsor ID
- IIT2024070
- Conditions
- Aplastic Anemia, TPO Receptor Agonists
- Keywords
- Severe aplastic anemia, Romiplostim N01
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Qilu Pharmaceutical Co., Ltd.
- Study start
- 24 Oct 2024 (actual)
- Primary completion
- 26 Sep 2025 (actual)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 26 Sep 2024 (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 prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| p-ALG+Romiplostim N01+CsA | Experimental | Drug: standard IST combined with Romiplostim N01The maximum screening period was 4 weeks, and after entering the treatment period, the patients were treated with p-ALG combined with cyclosporine and Romiplostim N01. Symptomatic leukocyte elevation, blood transfusion support, and anti-infective therapy are allowed during this period, according to the patient's blood routine. If a patient has a serious adverse event during treatment, it will be treated according to the criteria for suspension and recovery. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 12 years
- Diagnosis of severe or very severe aplastic anemia
- Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
- ECOG performance status ≤2
- Willing and able to comply with the requirements for this study and written informed consent.
Key exclusion criteria (12 listed)
- Received > 4 weeks of TPO-RA drug before treatment
- Received > 4 weeks of immunosuppressive therapy before treatment
- History of thromboembolic disease
- Intolerance to Romiplostim N01 or cyclosporine
- Allergy to ALG
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 |
|---|---|---|
| standard IST combined with Romiplostim N01Romiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation. | Drug | p-ALG+Romiplostim N01+CsA |
Primary outcome
Overall response rate
Time frame: Within 27 weeks post treatment
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
3 secondary outcomes
Overall response rate
Time frame: Within 3 months post treatment
Time to hematological response
Time frame: Within 27 weeks post treatment
Incidence of adverse events
Time frame: Within 27 weeks post treatment
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 4 Nov 2026.
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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. This study is not recruiting at the moment.
Jun Shi, PhD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing standard IST combined with Romiplostim N01 in people with Aplastic Anemia and TPO Receptor Agonists, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 5 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: Within 27 weeks post treatment). 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 4 Nov 2026.