Aplastic AnemiaPhase 4NCT06525948
Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of TD-NSAA
A Phase 4 study testing rhTPO and Cyclosporin A in people with Aplastic Anemia and Transfusion-dependent Anemia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia, Transfusion-dependent Anemia
- What is being tested?
- rhTPO and Cyclosporin A
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 1 siteChina
- Study dates
- 30 Jul 2024to 30 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
- 1. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who are dependent on transfusion therapy:
- Meet the Camitta NSAA criteria;
- accompanied by at least one of the following abnormalities: (1) dependence on component blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, the indication of component blood transfusion: HGB ≤ 60g / L; (2) PLT ≤ 10 × 10 ^ 9 / L, or PLT ≤ 30 × 10 ^ 9 / L with a significant tendency to bleed; (3) neutrophils ≤ 0.5 × 10 ^ 9 / L.
- Excluding other haematological and non-haematological diseases that cause pancytopenia; 3. ECOG PS score 0-2, expected survival ≥ 3 months with follow-up; 4. functional levels of major organs must meet the following requirements: 1) Total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) blood creatinine (Cr) ≤ 1.5 x ULN; 5. has not been treated with platelet receptor agonist (TPO-RA) analogues and other immunosuppressant analogues; 6. the subject is not suitable or willing to receive haematopoietic stem cell transplantation therapy; 7. no history of serious heart, lung, liver, kidney and other important organs and endocrine system diseases; 8. Voluntarily enroll in the study, sign the informed consent, have good compliance and willing to cooperate with the follow-up.
Who cannot join
- have used other clinical investigational drugs within 4 weeks;
- a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleep haemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failure syndromes (IBMFS), such as Fanconi's anaemia (FA) and congenital dyskeratosis (DC)
- history of cirrhosis or history of portal hypertension;
- congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication within 1 year prior to enrolment, or myocardial infarction or cerebral infarction within 3 months prior to enrolment;
- HIV infection;
- severe autoimmune disease or immunodeficiency disease;
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- suffering from malignant tumour
- severe mental disorders;
- a known history of allergy to the drug components of this regimen;
- in the opinion of the investigator, it is not appropriate to participate in this trial, e.g., any other medical, social or psychological factors that may affect safety or compliance with the study procedures.
- Compliance with study procedures.
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: Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of Transfusion-dependent Non-severe Aplastic Anaemia (TD-NSAA)
Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA
Read the detailed description
Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of transfusion-dependent aplastic anaemia of the non-severe type
- Conditions
- Aplastic Anemia, Transfusion-dependent Anemia
- Being tested
- rhTPO, Cyclosporin A
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhTPODrug
rhTPO
Cyclosporin ADrug
cyclosporine A
| Study group | Role | What they receive |
|---|---|---|
| two-drug combination group | Experimental | rhTPO combined with cyclosporine Receives: Drug: rhTPO; Drug: Cyclosporin A |
| drug-free group | Active comparator | cyclosporine alone Receives: Drug: Cyclosporin A |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
Overall Response Rate (ORR): meets the criteria for CR and PR.
Measured: 3 months
CRR
Complete Response Rate (CRR)：ANC>1.5×10^9/L，Hb>100g/L，PLT>100×10^9/L；
Measured: 3 months
Secondary outcomes (4)
ORR
Overall Response Rate (ORR): meets the criteria for CR and PR.
Measured: 6 months
CRR
Complete Response Rate (CRR)：ANC>1.5×10^9/L，Hb>100g/L，PLT>100×10^9/L；
Measured: 6 months
Time for platelet count to recover to ≥20 x 10^9/L with a 1-fold increase in absolute value
Time for platelet count to recover to ≥20 x 10^9/L with a 1-fold increase in absolute
Measured: through study completion, an average of 1 year
Proportion of patients with adverse events
safety event
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2024
- Study start30 Jul 2024 (estimated)
- Primary completion30 Jun 2025 (estimated)
- Study completion30 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 54 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
- Investigators
- Bing Han, PhD, Principal investigator, Peking Union Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06525948
- Sponsor's protocol ID
- rTC-AA
- Status last verified
- Jul 2024
- First submitted
- 18 Jul 2024
- Last updated
- 29 Jul 2024
- Results posted
- No
- Data monitoring committee
- 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 7616805Dancan GD. Spectrophotometry of tissue glycogen. Clin Chem. 1984 Sep;30(9):1580-1. No abstract available.
PubMed 6467577Bacigalupo 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 29 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of Transfusion-dependent Non-severe Aplastic Anaemia (TD-NSAA)
- ClinicalTrials.gov ID
- NCT06525948
- Acronym / sponsor ID
- rTC-AA
- Conditions
- Aplastic Anemia, Transfusion-dependent Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 30 Jul 2024 (estimated)
- Primary completion
- 30 Jun 2025 (estimated)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 29 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.
Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA
Detailed description
Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of transfusion-dependent aplastic anaemia of the non-severe type
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| two-drug combination group | Experimental | Drug: rhTPO, Drug: Cyclosporin ArhTPO combined with cyclosporine |
| drug-free group | Active comparator | Drug: Cyclosporin Acyclosporine alone |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 1. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who are dependent on transfusion therapy:
- Meet the Camitta NSAA criteria;
- accompanied by at least one of the following abnormalities: (1) dependence on component blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, the indication of component blood transfusion: HGB ≤ 60g / L; (2) PLT ≤ 10 × 10 ^ 9 / L, or PLT ≤ 30 × 10 ^ 9 / L with a significant tendency to bleed; (3) neutrophils ≤ 0.5 × 10 ^ 9 / L.
- Excluding other haematological and non-haematological diseases that cause pancytopenia; 3. ECOG PS score 0-2, expected survival ≥ 3 months with follow-up; 4. functional levels of major organs must meet the following requirements: 1) Total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) blood creatinine (Cr) ≤ 1.5 x ULN; 5. has not been treated with platelet receptor agonist (TPO-RA) analogues and other immunosuppressant analogues; 6. the subject is not suitable or willing to receive haematopoietic stem cell transplantation therapy; 7. no history of serious heart, lung, liver, kidney and other important organs and endocrine system diseases; 8. Voluntarily enroll in the study, sign the informed consent, have good compliance and willing to cooperate with the follow-up.
Key exclusion criteria (11 listed)
- have used other clinical investigational drugs within 4 weeks;
- a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleep haemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failure syndromes (IBMFS), such as Fanconi's anaemia (FA) and congenital dyskeratosis (DC)
- history of cirrhosis or history of portal hypertension;
- congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication within 1 year prior to enrolment, or myocardial infarction or cerebral infarction within 3 months prior to enrolment;
- HIV infection;
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 |
|---|---|---|
| rhTPOrhTPO | Drug | two-drug combination group |
| Cyclosporin Acyclosporine A | Drug | drug-free group, two-drug combination group |
Primary outcome
ORR
Time frame: 3 months
Overall Response Rate (ORR): meets the criteria for CR and PR.
CRR
Time frame: 3 months
Complete Response Rate (CRR)：ANC>1.5×10^9/L，Hb>100g/L，PLT>100×10^9/L；
4 secondary outcomes
ORR
Time frame: 6 months
CRR
Time frame: 6 months
Time for platelet count to recover to ≥20 x 10^9/L with a 1-fold increase in absolute value
Time frame: through study completion, an average of 1 year
Proportion of patients with adverse events
Time frame: through study completion, an average of 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 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 6467577Dancan GD. Spectrophotometry of tissue glycogen. Clin Chem. 1984 Sep;30(9):1580-1. 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
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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 rhTPO and Cyclosporin A in people with Aplastic Anemia and Transfusion-dependent 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 4 inclusion and 11 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 29 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). 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 NCT06525948, 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 29 Jul 2024.