Immune ThrombocytopeniaPhase 2NCT07064889
The Combination of Upadacitinib and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
A Phase 2 study testing Dexamethasone and Upadacitinib in people with Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
Randomized, open-label, multicenter study to compare the efficacy and safety of Upadacitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Dexamethasone and Upadacitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 132 participants
- Where
- 1 siteChina
- Study dates
- 30 Jul 2025to 30 Dec 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed newly-diagnosed, treatment-naive ITP;
- Platelet counts <30×109/L ;
- Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- Willing and able to sign written informed consent.
Who cannot join
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
- Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- Active infection;
- Maligancy;
- Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
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- Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
- Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
- Patients who are deemed unsuitable for the study by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: The Combination of Oral Upadacitinib and High-dose Dexamethasone vs High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Trial
Randomized, open-label, multicenter study to compare the efficacy and safety of Upadacitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Read the detailed description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of adults ITP patients in China. Patients were randomized to Upadacitinib + high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Dexamethasone, Upadacitinib
- 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.
DexamethasoneDrug
Dexamethasone, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
UpadacitinibDrug
Upadacitinib 15mg qd for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Upadacitinib and HD-DXM | Experimental | Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and Upadacitinib 15mg qd po, 12 consecutive weeks Receives: Drug: Dexamethasone; Drug: Upadacitinib |
| HD-DXM | Active comparator | Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) Receives: Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained response
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Measured: 6 month
Secondary outcomes (8)
complete response (CR)
complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding
Measured: day 14
Response (R)
Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Measured: day 14
Number of patients with bleeding
Number of patients with bleeding complication ( WHO bleeding score).
Measured: 6 month
Number of patients with adverse events
Number of patients with adverse events
Measured: 6 month
Time to response
The time from starting treatment to time of achievement of CR or R
Measured: 6 month
Duration of response (DOR)
Duration of response at 6-month follow up
Measured: 6 month
Loss of response
Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)
Measured: 6 month
Health related quality of life
Health-related quality of life using the Immune Thrombocytopenic Purpura-Patient Assessment Questionnaire (ITP-PAQ)
Measured: 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2025
- Study start30 Jul 2025 (estimated)
- Primary completion30 Jul 2027 (estimated)
- Study completion30 Dec 2027 (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
- 132 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Beijing Hospital, Navy General Hospital, Beijing, Beijing Aerospace General Hospital, Qilu Hospital of Shandong University, Beijing Tongren Hospital
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07064889
- Sponsor's protocol ID
- ITP-UPA-001
- Status last verified
- Jul 2025
- First submitted
- 7 Jul 2025
- Last updated
- 15 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to data, including deidentified individual participant data, and the study protocol from this clinical trial. These data will be available beginning 3 months and ending 36 months following publication. All requests must include a description of the research proposal and be sent to the corresponding author (zhangxh@bjmu.edu.cn). Data requestors will need to sign a data access agreement to obtain access.
Study at a glance
From the registry record, last updated by the sponsor on 15 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Combination of Oral Upadacitinib and High-dose Dexamethasone vs High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Trial
- ClinicalTrials.gov ID
- NCT07064889
- Acronym / sponsor ID
- ITP-UPA-001
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 132 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Beijing Hospital, Navy General Hospital, Beijing, Beijing Aerospace General Hospital, Qilu Hospital of Shandong University, Beijing Tongren Hospital
- Study start
- 30 Jul 2025 (estimated)
- Primary completion
- 30 Jul 2027 (estimated)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 15 Jul 2025 (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 compare the efficacy and safety of Upadacitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of adults ITP patients in China. Patients were randomized to Upadacitinib + high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Upadacitinib and HD-DXM | Experimental | Drug: Dexamethasone, Drug: UpadacitinibDexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and Upadacitinib 15mg qd po, 12 consecutive weeks |
| HD-DXM | Active comparator | Drug: DexamethasoneDexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed newly-diagnosed, treatment-naive ITP;
- Platelet counts <30×109/L ;
- Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- Willing and able to sign written informed consent.
Key exclusion criteria (9 listed)
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
- Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- Active infection;
- Maligancy;
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 |
|---|---|---|
| DexamethasoneDexamethasone, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10) | Drug | HD-DXM, Upadacitinib and HD-DXM |
| UpadacitinibUpadacitinib 15mg qd for 12 weeks | Drug | Upadacitinib and HD-DXM |
Primary outcome
Sustained response
Time frame: 6 month
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
8 secondary outcomes
complete response (CR)
Time frame: day 14
Response (R)
Time frame: day 14
Number of patients with bleeding
Time frame: 6 month
Number of patients with adverse events
Time frame: 6 month
Time to response
Time frame: 6 month
Duration of response (DOR)
Time frame: 6 month
Loss of response
Time frame: 6 month
Health related quality of life
Time frame: 6 month
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 15 Jul 2025.
- Individual participant dataSharing planned: Yes. Qualified researchers can request access to data, including deidentified individual participant data, and the study protocol from this clinical trial. These data will be available beginning 3 months and ending 36 months following publication. All requests must include a description of the research proposal and be sent to the corresponding author (zhangxh@bjmu.edu.cn). Data requestors will need to sign a data access agreement to obtain access.
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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.
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 Dexamethasone and Upadacitinib in people with Immune Thrombocytopenia, run by Peking University People's 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 9 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: opening soon, as of the sponsor's last update on 15 Jul 2025. 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?
Contact Xiaohui Zhang (+8613522338836, zhangxh100@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sustained response (time frame: 6 month). 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 15 Jul 2025.