Immune ThrombocytopeniaPhase 2NCT05634824
IVIg Plus Low Dose rhTPO for ITP in Pregnancy
A Phase 2 study testing intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO) and intravenous immunoglobulin in people with Immune Thrombocytopenia and Pregnancy Related, run by Peking University People's Hospital.
Trial summary
Randomized, open-label, multicenter study to investigate the efficacy and safety of IVIg plus low-dose recombinant thrombopoietin in pregnant patients with corticosteroid or IVIg monotherapy-resistant ITP. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- Immune Thrombocytopenia, Pregnancy Related
- What is being tested?
- intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO) and intravenous immunoglobulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2023to 1 Jan 2023
- Phase
- Phase 2
- 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 to 50 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Patients have a diagnosis of primary ITP before or first onset during pregnancy; Patients ≥ 18 years; patients complicate with bleeding manifestations and/or have a platelet count < 30 × 10^9/L and failed to respond to initial treatment of corticosteroids or intravenous immunoglobulin (IVIg) monotherapy or relapsed during the tapering or discontinuation of corticosteroids.
Who cannot join
- Patients whose thrombocytopenia was secondary to cancer (solid tumor or leukemia), infections, preeclampsia, and HELLP syndrome (hemolysis, elevated liver enzymes, low platelets), and those who had primary immune deficiency, and other hematology or connective tissue diseases during follow-up; patients have heart, kidney, liver, or lung dysfunction; patients received chemotherapy or anticoagulants within 3 months before screening or other second-line ITP-specific treatments within 3 months before screening.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: The Combination of IVIg and Low Does Recombinant Human Thrombopoietin for the Management of Immune Thrombocytopenia in Pregnancy
Randomized, open-label, multicenter study to investigate the efficacy and safety of IVIg plus low-dose recombinant thrombopoietin in pregnant patients with corticosteroid or IVIg monotherapy-resistant ITP.
Read the detailed description
Primary immune thrombocytopenia (ITP) is a common autoimmune disease characterized by a low platelet count resulting from antibody-mediated platelet destruction and insufficient platelet production. ITP, with a prevalence of 1 in 1000-10 000 pregnancies, accounts for about 3% of all causes of thrombocytopenia in pregnancy and is reported to be the most common cause of thrombocytopenia in the first and early second trimesters. It is revealed that severe ITP is associated with devastating outcomes (e.g., postpartum hemorrhage, placental abruption, significant bleeding complication in both women patients and neonates, and even fetal death). It remains a challenge to manage pregnant patients with ITP. First-line treatment options in pregnant ITP patients include corticosteroids and intravenous immunoglobulin (IVIg) currently. However, previous studies reported that the response rate of pregnant ITP patients to corticosteroids was less than 40% and that to IVIg was 38%-56%, which was lower than that of nonpregnant ITP patients. The second-line treatment options are limited for pregnant ITP patients who have no response to the first-line treatments and no agreement has been reached about the optimum second-line treatment for pregnant ITP patients. In addition, single-agent therapy requires an increased dose of the agent and a long-term exposure, which is associated with an increased risk of treatment-related adverse effects. Therefore, the combination of agents sharing disparate mechanisms in the treatment of ITP might be a promising option to maximize efficacy while minimizing side effects.
Thrombopoietin (TPO) mimetics, including eltrombopag and romiplostim, are recommended to be used in the management of nonpregnant corticosteroid-resistant or relapsed ITP patients. However, they may pass through the placenta to have an influence on the fetus, which hinder their use in the management of pregnant ITP patients. Recombinant human thrombopoietin (rhTPO), a full-length and glycosylated TPO with a molecular weight of 90 000 Daltons developed by 3SBIO (Shenyang, China), has also been approved by the China State Food and Drug Administration as a second-line option for nonpregnant ITP patients. It could not cross the placenta theoretically with such a large molecular weight, and thus it has an apparent theoretical advantage over the two TPO mimetics since it would not impact the fetus. Previous data of a multicenter randomized phase 3 clinical trial on nonpregnant ITP patients resistant to corticosteroids showed that rhTPO increased platelet counts rapidly. A study investigating the application of rhTPO in pregnancy suggested rhTPO was a potentially effective and well-tolerated treatment option for ITP patients during pregnancy. A previous case report described a patient with refractory ITP who failed to response to monotherapy of IVIg and TPO mimetics, but responded rapidly with the combination of IVIg and TPO mimetics. It might be explained that the development of ITP was associated with increased platelet destruction and impaired platelet production, and the combination of IVIg and TPO mimetics directed to these two targets since IVIg could reduce platelet destruction whereas TPO mimetics could increase platelet production. Therefore, we conducted a multicenter, single-arm, open-label study to investigate the efficacy and safety of low dose rhTPO plus IVIg in pregnant corticosteroids/IVIg monotherapy-resistant ITP patients.
- Conditions
- Immune Thrombocytopenia, Pregnancy Related
- Being tested
- intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO), intravenous immunoglobulin
- Keywords
- immune thrombocytopenia, pregnancy related
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO)Drug
rhTPO at an initial does of 150U/kg qd subcutaneously for 28 days and IVIg 400mg/kg qd for 5 days. The maintenance treatment consisted rhTPO 150U/kg qd.
intravenous immunoglobulinDrug
intravenous immunoglobulin
| Study group | Role | What they receive |
|---|---|---|
| IVIg plus low dose rhTPO | Experimental | Patients were randomized into experimental and control groups. For the experimental patients, the initial combination therapy consisted rhTPO at an initial does of 150U/kg once daily subcutaneously for 28 days and IVIg 400mg/kg per day for 5 days. If the platelet count was in the range of 30 to 50 × 10^9/L, the combination therapy was repeated. To reduce the risk for thrombocytosis, patients received maintenance therapy consisting rhTPO at a dose of 150U/kg per day if the platelet count rose above 50 × 10^9/L, and treatment discontinued when platelet counts exceeded 100 × 10^9/L. After delivery, the treatment consisted rhTPO 150U/kg per day, and maintained the dose if the platelet count exceeded 30 × 10^9/L. In addition, IVIg 400mg/kg per day for 5 days was added if the platelet count could not maintain above 30 × 10^9/L. If patients did not achieve 30 × 10^9/L within 4 weeks treatment was also discontinued. Receives: Drug: intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO) |
| IvIg treatment | Active comparator | Patients would be treated with IVIg 400mg/kg per day for 5 days monthly. And treatment discontinued when platelet counts exceeded 100 × 10^9/L. Receives: Drug: intravenous immunoglobulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
4-week response rate
Measured: 4 weeks
Secondary outcomes (5)
Time to relapse
the interval from response to platelet count of less than 30 × 10^9/L
Measured: 13 months
Bleeding manifestations
presence of bleeding
Measured: 13 months
Additional ITP therapy
Starting of additional ITP therapy
Measured: 13 months
Maternal and neonatal adverse events
According to the Common Terminology Criteria for Adverse Events, Version 6.0
Measured: 13 months
Neonatal platelet count
at birth, day 7 and day 42
Measured: up to 34 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2022
- Study start1 Jan 2023 (estimated)
- Primary completion1 Jan 2023 (estimated)
- Study completion1 Jan 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Peking University People's 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05634824
- Sponsor's protocol ID
- PKU-ITP022
- Status last verified
- Nov 2022
- First submitted
- 23 Nov 2022
- Last updated
- 2 Dec 2022
- 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 2 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Combination of IVIg and Low Does Recombinant Human Thrombopoietin for the Management of Immune Thrombocytopenia in Pregnancy
- ClinicalTrials.gov ID
- NCT05634824
- Acronym / sponsor ID
- PKU-ITP022
- Conditions
- Immune Thrombocytopenia, Pregnancy Related
- Keywords
- immune thrombocytopenia, pregnancy related
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2023 (estimated)
- Primary completion
- 1 Jan 2023 (estimated)
- Study completion
- 1 Jan 2023 (estimated)
- First posted
- 2 Dec 2022 (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.
Randomized, open-label, multicenter study to investigate the efficacy and safety of IVIg plus low-dose recombinant thrombopoietin in pregnant patients with corticosteroid or IVIg monotherapy-resistant ITP.
Detailed description
Primary immune thrombocytopenia (ITP) is a common autoimmune disease characterized by a low platelet count resulting from antibody-mediated platelet destruction and insufficient platelet production. ITP, with a prevalence of 1 in 1000-10 000 pregnancies, accounts for about 3% of all causes of thrombocytopenia in pregnancy and is reported to be the most common cause of thrombocytopenia in the first and early second trimesters. It is revealed that severe ITP is associated with devastating outcomes (e.g., postpartum hemorrhage, placental abruption, significant bleeding complication in both women patients and neonates, and even fetal death). It remains a challenge to manage pregnant patients with ITP. First-line treatment options in pregnant ITP patients include corticosteroids and intravenous immunoglobulin (IVIg) currently. However, previous studies reported that the response rate of pregnant ITP patients to corticosteroids was less than 40% and that to IVIg was 38%-56%, which was lower than that of nonpregnant ITP patients. The second-line treatment options are limited for pregnant ITP patients who have no response to the first-line treatments and no agreement has been reached about the optimum second-line treatment for pregnant ITP patients. In addition, single-agent therapy requires an increased dose of the agent and a long-term exposure, which is associated with an increased risk of treatment-related adverse effects. Therefore, the combination of agents sharing disparate mechanisms in the treatment of ITP might be a promising option to maximize efficacy while minimizing side effects.
Thrombopoietin (TPO) mimetics, including eltrombopag and romiplostim, are recommended to be used in the management of nonpregnant corticosteroid-resistant or relapsed ITP patients. However, they may pass through the placenta to have an influence on the fetus, which hinder their use in the management of pregnant ITP patients. Recombinant human thrombopoietin (rhTPO), a full-length and glycosylated TPO with a molecular weight of 90 000 Daltons developed by 3SBIO (Shenyang, China), has also been approved by the China State Food and Drug Administration as a second-line option for nonpregnant ITP patients. It could not cross the placenta theoretically with such a large molecular weight, and thus it has an apparent theoretical advantage over the two TPO mimetics since it would not impact the fetus. Previous data of a multicenter randomized phase 3 clinical trial on nonpregnant ITP patients resistant to corticosteroids showed that rhTPO increased platelet counts rapidly. A study investigating the application of rhTPO in pregnancy suggested rhTPO was a potentially effective and well-tolerated treatment option for ITP patients during pregnancy. A previous case report described a patient with refractory ITP who failed to response to monotherapy of IVIg and TPO mimetics, but responded rapidly with the combination of IVIg and TPO mimetics. It might be explained that the development of ITP was associated with increased platelet destruction and impaired platelet production, and the combination of IVIg and TPO mimetics directed to these two targets since IVIg could reduce platelet destruction whereas TPO mimetics could increase platelet production. Therefore, we conducted a multicenter, single-arm, open-label study to investigate the efficacy and safety of low dose rhTPO plus IVIg in pregnant corticosteroids/IVIg monotherapy-resistant ITP patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IVIg plus low dose rhTPO | Experimental | Drug: intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO)Patients were randomized into experimental and control groups. For the experimental patients, the initial combination therapy consisted rhTPO at an initial does of 150U/kg once daily subcutaneously for 28 days and IVIg 400mg/kg per day for 5 days. If the platelet count was in the range of 30 to 50 × 10^9/L, the combination therapy was repeated. To reduce the risk for thrombocytosis, patients received maintenance therapy consisting rhTPO at a dose of 150U/kg per day if the platelet count rose above 50 × 10^9/L, and treatment discontinued when platelet counts exceeded 100 × 10^9/L. After delivery, the treatment consisted rhTPO 150U/kg per day, and maintained the dose if the platelet count exceeded 30 × 10^9/L. In addition, IVIg 400mg/kg per day for 5 days was added if the platelet count could not maintain above 30 × 10^9/L. If patients did not achieve 30 × 10^9/L within 4 weeks treatment was also discontinued. |
| IvIg treatment | Active comparator | Drug: intravenous immunoglobulinPatients would be treated with IVIg 400mg/kg per day for 5 days monthly. And treatment discontinued when platelet counts exceeded 100 × 10^9/L. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- Patients have a diagnosis of primary ITP before or first onset during pregnancy; Patients ≥ 18 years; patients complicate with bleeding manifestations and/or have a platelet count < 30 × 10^9/L and failed to respond to initial treatment of corticosteroids or intravenous immunoglobulin (IVIg) monotherapy or relapsed during the tapering or discontinuation of corticosteroids.
Key exclusion criteria (1 listed)
- Patients whose thrombocytopenia was secondary to cancer (solid tumor or leukemia), infections, preeclampsia, and HELLP syndrome (hemolysis, elevated liver enzymes, low platelets), and those who had primary immune deficiency, and other hematology or connective tissue diseases during follow-up; patients have heart, kidney, liver, or lung dysfunction; patients received chemotherapy or anticoagulants within 3 months before screening or other second-line ITP-specific treatments within 3 months before screening.
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 |
|---|---|---|
| intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO)rhTPO at an initial does of 150U/kg qd subcutaneously for 28 days and IVIg 400mg/kg qd for 5 days. The maintenance treatment consisted rhTPO 150U/kg qd. | Drug | IVIg plus low dose rhTPO |
| intravenous immunoglobulinintravenous immunoglobulin | Drug | IvIg treatment |
Primary outcome
4-week response rate
Time frame: 4 weeks
5 secondary outcomes
Time to relapse
Time frame: 13 months
Bleeding manifestations
Time frame: 13 months
Additional ITP therapy
Time frame: 13 months
Maternal and neonatal adverse events
Time frame: 13 months
Neonatal platelet count
Time frame: up to 34 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2022.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing intravenous immunoglobulin plus low dose recombinant human thrombopoietin (rhTPO) and intravenous immunoglobulin in people with Immune Thrombocytopenia and Pregnancy Related, run by Peking University People's Hospital.
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 1 inclusion and 1 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 Dec 2022. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiao-Hui Zhang, Prof. (+8613522338836, zhangxh100@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
4-week response rate (time frame: 4 weeks). 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 2 Dec 2022.