ITPPhase 2BAITPNCT05446831
Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia
A Phase 2 study testing Baricitinib in people with ITP and Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
Single-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- ITP, Immune Thrombocytopenia
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteChina
- Study dates
- 13 Jul 2022to 1 Dec 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia
- Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count
- Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment
- Over 18 years old
- Willing and able to provide written informed consent, and agreeable to the schedule of assessment
Who cannot join
- Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease)
- Active or a history of malignancy
- Pregnancy or lactation
- Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
- A history of symptomatic herpes zoster infection within 12 weeks prior to screening
- Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
Show all 12Show fewer
- Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB
- Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled
- Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure
- A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data
- Any of the following specific abnormalities on screening laboratory tests:
- 1) ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN 2) hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL) 3) eGFR <50 mL/min/1.73 m^2
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, 100010
Xiaohui Zhang, MD, +8613522338836, zhangxh100@sina.com
Peng Zhao, MD, +8618810323668, zpeng702@163.com
About this study
Official title: Efficacy and Safety of Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia: A Single-arm, Open-label Phase II Study
Single-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Read the detailed description
The investigators are undertaking a prospective trial of 20 adults with ITP in China. Baricitinib is administered as 4 mg po. daily. Safety outcomes and efficacy outcomes are assessed on scheduled study visits (primary endpoint defined as durable response at 6-month follow-up).
- Conditions
- ITP, Immune Thrombocytopenia
- Being tested
- Baricitinib
- 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.
BaricitinibDrug
Oral baricitinib was given at a dose of 4 mg daily. The decision to initiate rescue therapy was made after assessment of the extent of bleeding, patient preferences, lifestyle and activity, the complications of specific therapies, comorbidities that predisposed patients to bleeding and the tolerance of side effects. If a platelet count over 300,000/μL was observed for two consecutive tests at least 2 weeks apart, baricitinib treatment was interrupted.
| Study group | Role | What they receive |
|---|---|---|
| Baricitinib | Experimental | Oral baricitinib was given at a dose of 4 mg daily for 6 months. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patients' request. Receives: Drug: Baricitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Durable response
The maintenance of a platelet count ≥30,000/μL, 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 months
Secondary outcomes (9)
Complete response (CR)
Complete response (CR) was defined as a platelet count over 100,000/μL and absence of bleeding.
Measured: 1 month
Response (R)
Response (R) as a platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Measured: 1 month
Time to response
The time from starting treatment to time of achievement of CR or R.
Measured: 6 months
Duration of response
Duration of response at 6-month follow up.
Measured: 6 months
Early response
Achievement of CR or R at day 7
Measured: 7 days
Initial response
Achievement of CR or R at day 28
Measured: 28 days
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Measured: From the start of study treatment (Day 1) to the end of week 24
Health-related quality of life (HRQoL)
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Measured: From the start of study treatment (Day 1) to the end of week 24
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Measured: From the start of study treatment (Day 1) to the end of week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2022
- Study start13 Jul 2022
- Primary completion1 Jun 2023 (estimated)
- Study completion1 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ITPStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 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
- Investigators
- Xiaohui Zhang, MD, Principal investigator, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05446831
- Sponsor's protocol ID
- PKU-ITP2207
- Acronym
- BAITP
- Status last verified
- Sep 2022
- First submitted
- 1 Jul 2022
- Last updated
- 28 Sep 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Zhao P, An ZY, Fu HX, Liu HX, Feng CJ, Huang QS, Wu J, Wu YJ, Yang LP, Qu QY, Chen YX, Li ML, Wang CC, Chen Q, Zhu XL, He Y, Zhang YY, Jiang Q, Jiang H, Lu J, Chang YJ, Zhao XS, Zhao XY, Huang XJ, Zhang XH. Safety and efficacy of baricitinib in steroid-resistant or relapsed immune thrombocytopenia: An open-label pilot study. Am J Hematol. 2024 Oct;99(10):1951-1958. doi: 10.1002/ajh.27433. Epub 2024 Jul 9.
PubMed 38980207
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia: A Single-arm, Open-label Phase II Study
- ClinicalTrials.gov ID
- NCT05446831
- Acronym / sponsor ID
- BAITP / PKU-ITP2207
- Conditions
- ITP, Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 13 Jul 2022 (actual)
- Primary completion
- 1 Jun 2023 (estimated)
- Study completion
- 1 Dec 2023 (estimated)
- First posted
- 7 Jul 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.
Single-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a prospective trial of 20 adults with ITP in China. Baricitinib is administered as 4 mg po. daily. Safety outcomes and efficacy outcomes are assessed on scheduled study visits (primary endpoint defined as durable response at 6-month follow-up).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Baricitinib | Experimental | Drug: BaricitinibOral baricitinib was given at a dose of 4 mg daily for 6 months. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patients' request. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia
- Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count
- Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment
- Over 18 years old
Key exclusion criteria (12 listed)
- Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease)
- Active or a history of malignancy
- Pregnancy or lactation
- Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
- A history of symptomatic herpes zoster infection within 12 weeks prior to 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 |
|---|---|---|
| BaricitinibOral baricitinib was given at a dose of 4 mg daily. The decision to initiate rescue therapy was made after assessment of the extent of bleeding, patient preferences, lifestyle and activity, the complications of specific therapies, comorbidities that predisposed patients to bleeding and the tolerance of side effects. If a platelet count over 300,000/μL was observed for two consecutive tests at least 2 weeks apart, baricitinib treatment was interrupted. | Drug | Baricitinib |
Primary outcome
Durable response
Time frame: 6 months
The maintenance of a platelet count ≥30,000/μL, 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.
9 secondary outcomes
Complete response (CR)
Time frame: 1 month
Response (R)
Time frame: 1 month
Time to response
Time frame: 6 months
Duration of response
Time frame: 6 months
Early response
Time frame: 7 days
Initial response
Time frame: 28 days
Bleeding events
Time frame: From the start of study treatment (Day 1) to the end of week 24
Health-related quality of life (HRQoL)
Time frame: From the start of study treatment (Day 1) to the end of week 24
Adverse events
Time frame: From the start of study treatment (Day 1) to the end of week 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2022.
- Derived from this study: PubMed 38980207Zhao P, An ZY, Fu HX, Liu HX, Feng CJ, Huang QS, Wu J, Wu YJ, Yang LP, Qu QY, Chen YX, Li ML, Wang CC, Chen Q, Zhu XL, He Y, Zhang YY, Jiang Q, Jiang H, Lu J, Chang YJ, Zhao XS, Zhao XY, Huang XJ, Zhang XH. Safety and efficacy of baricitinib in steroid-resistant or relapsed immune thrombocytopenia: An open-label pilot study. Am J Hematol. 2024 Oct;99(10):1951-1958. doi: 10.1002/ajh.27433. Epub 2024 Jul 9.
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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.
Xiaohui Zhang, MD principal investigator
Peking University People's Hospital
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 Baricitinib in people with ITP and 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 6 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 28 Sep 2022. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaohui Zhang, MD (+8613522338836, zhangxh100@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Durable response (time frame: 6 months). 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 NCT05446831, which also lists 1 linked publication.
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 28 Sep 2022.