Aplastic AnemiaPhase 4NCT06516484
Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
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
To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- 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
- 30 Jul 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years, male or female.
- Diagnosis consistent with refractory AA after radiotherapy. refractory is defined as patients who have failed to respond to at least an adequate amount of supportive therapy, cyclosporine or povidone TPO-RA for 3 months .
- At least one of the following conditions was met at enrolment: haemoglobin <90 g/L. Platelets <30 x 10^9/L, neutrophils <1.0 x 10^9/L.
- Baseline liver and renal function is less than two times the normal value.
- No active infection.
- Agreed to sign the consent form.
- 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%.
- Hematopoietic stem cell transplantation (HSCT) prior to enrolment.
- Prior treatment with ATG.
- Infection or bleeding uncontrolled by standard therapy.
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- 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.
- Previous 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
To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy.
- Conditions
- Aplastic Anemia
- Being tested
- Romiplostim
- Keywords
- After radiotherapy and chemotherapy, 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 were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim group | Experimental | Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.
Measured: 3 months, 6 months
CRR
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.
Measured: 3 months, 6months
Secondary outcomes (1)
safety events
Proportion and severity of adverse events in patients during the study period
Measured: 3 months，6months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2024
- Study start30 Jul 2024 (estimated)
- Primary completion31 Jul 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 (2)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
- Sponsor
- Investigators
- Bing Han, PhD, Principal investigator, Peking Union Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06516484
- Sponsor's protocol ID
- LRA-2024-001
- Status last verified
- Jul 2024
- First submitted
- 18 Jul 2024
- Last updated
- 24 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
PubMed 23821332Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.
PubMed 31474546
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
- ClinicalTrials.gov ID
- NCT06516484
- Acronym / sponsor ID
- LRA-2024-001
- Conditions
- Aplastic Anemia
- Keywords
- After radiotherapy and chemotherapy, Romiplostim
- 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
- 30 Jul 2024 (estimated)
- Primary completion
- 31 Jul 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 24 Jul 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.
To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy.
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: RomiplostimEnrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥18 years, male or female.
- Diagnosis consistent with refractory AA after radiotherapy. refractory is defined as patients who have failed to respond to at least an adequate amount of supportive therapy, cyclosporine or povidone TPO-RA for 3 months .
- At least one of the following conditions was met at enrolment: haemoglobin <90 g/L. Platelets <30 x 10^9/L, neutrophils <1.0 x 10^9/L.
- Baseline liver and renal function is less than two times the normal value.
- No active infection.
Key exclusion criteria (12 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%.
- Hematopoietic 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 were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months. | Drug | Romiplostim group |
Primary outcome
ORR
Time frame: 3 months, 6 months
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.
CRR
Time frame: 3 months, 6months
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.
1 secondary outcome
safety events
Time frame: 3 months，6months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2024.
- Background: PubMed 23821332Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
- Background: PubMed 31474546Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.
- Individual participant dataSharing planned: No
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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.
Bing Han, PhD principal investigator
Peking Union Medical College
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 7 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: status not confirmed, as of the sponsor's last update on 24 Jul 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?
Contact Bing Han, PhD (+8613601059938, Hanbing_li@sina.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR (time frame: 3 months, 6 months). The study lists 2 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 NCT06516484, which also lists 2 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 24 Jul 2024.