Aplastic AnemiaPhase 4NCT06535685
A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA
A Phase 4 study testing Romiplostim in people with Aplastic Anemia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Romiplostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 2 Aug 2024to 31 Dec 2025
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age ≥18 years, male or female.
- Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
- Satisfy at least one of the following conditions at the time of enrolment: haemoglobin <90 g/L, platelets <30×10^9/L, neutrophils <1.0×10^9/L.
- Agree to sign the consent form.
- An Eastern Cooperative Oncology Group (ECOG) score of 0-2.
Who cannot join
- Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
- Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
- PNH clones ≥50%.
- Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
- Prior treatment with ATG.
- Infection or haemorrhage uncontrolled by standard therapy.
Show all 14Show fewer
- Allergy to roprostin.
- Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
- Liver and renal function at baseline that is more than two times normal.
- Active infection.
- Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
- Pregnant or lactating (breastfeeding) women.
- Participation in another clinical trial within 3 months.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
About this study
Official title: A Single-centre, Prospective, Open-label, Single-arm Study of Romiplostim for the Treatment of Refractory Transfusion-dependent Non-severe Aplastic Anaemia (NSAA)
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
- Conditions
- Aplastic Anemia
- Being tested
- Romiplostim
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Enrolled patients will be given Romiplostim (20 µg/kg subcutaneously once a week) for a minimum of 3 months.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim group | Experimental | Romiplostim group Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR)
Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC <0.5 × 10^9/L increased by at least 0.5 × 10^9/L after treatment, or initial PLT <20 × 10^9/L increased by at least 20 × 10^9/L after treatment.
Measured: 3 months
Overall response rate (ORR)
Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC <0.5 × 10^9/L increased by at least 0.5 × 10^9/L after treatment, or initial PLT <20 × 10^9/L increased by at least 20 × 10^9/L after treatment.
Measured: 6 months
CRR
CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.
Measured: 3 months
CRR
CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.
Measured: 6 months
Secondary outcomes (2)
safety events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: 3 months
safety events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: 6months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2024
- Study start2 Aug 2024 (estimated)
- Primary completion30 Mar 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06535685
- Sponsor's protocol ID
- LRA-2024-002
- Status last verified
- Jul 2024
- First submitted
- 24 Jul 2024
- Last updated
- 2 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.
PubMed 7616805Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.
PubMed 30354958Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481. Epub 2017 Jan 17.
PubMed 28096088Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.
PubMed 1813058
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-centre, Prospective, Open-label, Single-arm Study of Romiplostim for the Treatment of Refractory Transfusion-dependent Non-severe Aplastic Anaemia (NSAA)
- ClinicalTrials.gov ID
- NCT06535685
- Acronym / sponsor ID
- LRA-2024-002
- Conditions
- Aplastic Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 2 Aug 2024 (estimated)
- Primary completion
- 30 Mar 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 2 Aug 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.
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim group | Experimental | Drug: RomiplostimRomiplostim group |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥18 years, male or female.
- Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
- Satisfy at least one of the following conditions at the time of enrolment: haemoglobin <90 g/L, platelets <30×10^9/L, neutrophils <1.0×10^9/L.
- Agree to sign the consent form.
- An Eastern Cooperative Oncology Group (ECOG) score of 0-2.
Key exclusion criteria (14 listed)
- Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
- Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
- PNH clones ≥50%.
- Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
- Prior treatment with ATG.
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 |
|---|---|---|
| RomiplostimEnrolled patients will be given Romiplostim (20 µg/kg subcutaneously once a week) for a minimum of 3 months. | Drug | Romiplostim group |
Primary outcome
Overall response rate (ORR)
Time frame: 3 months
Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC <0.5 × 10^9/L increased by at least 0.5 × 10^9/L after treatment, or initial PLT <20 × 10^9/L increased by at least 20 × 10^9/L after treatment.
Overall response rate (ORR)
Time frame: 6 months
Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC <0.5 × 10^9/L increased by at least 0.5 × 10^9/L after treatment, or initial PLT <20 × 10^9/L increased by at least 20 × 10^9/L after treatment.
CRR
Time frame: 3 months
CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.
CRR
Time frame: 6 months
CR was defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients who did not receive a transfusion.
2 secondary outcomes
safety events
Time frame: 3 months
safety events
Time frame: 6months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2024.
- Background: PubMed 7616805Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.
- Background: PubMed 30354958Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.
- Background: PubMed 28096088Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481. Epub 2017 Jan 17.
- Background: PubMed 1813058Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.
- Individual participant dataSharing planned: No
Related trials for Aplastic Anemia
RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Romiplostim in people with Aplastic Anemia, run by Peking Union Medical College Hospital 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 14 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: status not confirmed, as of the sponsor's last update on 2 Aug 2024. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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 (ORR) (time frame: 3 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06535685, which also lists 4 linked publications.
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 2 Aug 2024.