Immune ThrombocytopeniaPhase 2NCT06190145
Teriflunomide for Steroid-resistant/Relapse Immune Thrombocytopenia
A Phase 2 study testing Teriflunomide in people with 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 teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Teriflunomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 5 Dec 2023to 15 Jun 2025
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with bleeding manifestations at the enrollment;
- 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;
- 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;
- Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease);
- Pregnancy or lactation;
- Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);
- Current or recent (< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- 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;
- Patients who are deemed unsuitable for the study by the investigator.
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
Xiao-Hui Zhang, MD, +8613522338836, zhangxh100@sina.com
Li-Ping Yang, MD, +8618519172033, lpyangvip@163.com
About this study
Official title: A Single-arm, Open-label Phase II Study to Determine the Safety and Efficacy of Teriflunomide in Patients With Steroid-resistant/Relapse Thrombocytopenia
Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Read the detailed description
This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Teriflunomide
- Keywords
- Teriflunomide
- 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.
TeriflunomideDrug
Also called AUBAGIO
Starting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts.
| Study group | Role | What they receive |
|---|---|---|
| Teriflunomide | Experimental | Oral Teriflunomide is given at a dose of 7 mg once daily with dose adjustments for 24 weeks. Treatment was discontinued if a dose-limiting toxic effect occurred, rescue medication was used, or concomitant medication for immune thrombocytopenia was changed beyond the 10% level as defined above. Receives: Drug: Teriflunomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained response
Platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least four of the six visits between weeks 19 and 24.
Measured: From the start of study treatment (Day 1) to the end of week 24
Secondary outcomes (6)
Overall response
Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding. Response (R) was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count and absence of bleeding.
Measured: From the start of study treatment (Day 1) to the end of week 24
Initial response
The number of participants with achievement of CR or R at 4 weeks.
Measured: From the start of study treatment (Day 1) up to week 4 of treatment
Time to response
The time from treatment initiation to achieve a CR or a R.
Measured: From the start of study treatment (Day 1) to the end of week 24
Bleeding events
Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale.
Measured: From the start of study treatment (Day 1) to the end of week 24
Adverse events
Adverse events (AEs) were reported and graded according to 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
Health-related quality of life (HRQoL)
ITP-PAQ was 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start5 Dec 2023
- Primary completion15 Oct 2024 (estimated)
- Study completion15 Jun 2025 (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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 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
- Xiao-Hui Zhang, Principal investigator, Peking University Institute of Hematology, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06190145
- Sponsor's protocol ID
- PKU-TFITP-02
- Status last verified
- Dec 2023
- First submitted
- 19 Dec 2023
- Last updated
- 5 Jan 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label Phase II Study to Determine the Safety and Efficacy of Teriflunomide in Patients With Steroid-resistant/Relapse Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06190145
- Acronym / sponsor ID
- PKU-TFITP-02
- Conditions
- Immune Thrombocytopenia
- Keywords
- Teriflunomide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 5 Dec 2023 (actual)
- Primary completion
- 15 Oct 2024 (estimated)
- Study completion
- 15 Jun 2025 (estimated)
- First posted
- 5 Jan 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.
Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Detailed description
This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Teriflunomide | Experimental | Drug: TeriflunomideOral Teriflunomide is given at a dose of 7 mg once daily with dose adjustments for 24 weeks. Treatment was discontinued if a dose-limiting toxic effect occurred, rescue medication was used, or concomitant medication for immune thrombocytopenia was changed beyond the 10% level as defined above. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with bleeding manifestations at the enrollment;
- 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;
- Willing and able to sign written informed consent.
Key exclusion criteria (7 listed)
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease);
- Pregnancy or lactation;
- Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);
- Current or recent (< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic 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 |
|---|---|---|
| TeriflunomideAlso: AUBAGIOStarting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts. | Drug | Teriflunomide |
Primary outcome
Sustained response
Time frame: From the start of study treatment (Day 1) to the end of week 24
Platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least four of the six visits between weeks 19 and 24.
6 secondary outcomes
Overall response
Time frame: From the start of study treatment (Day 1) to the end of week 24
Initial response
Time frame: From the start of study treatment (Day 1) up to week 4 of treatment
Time to response
Time frame: From the start of study treatment (Day 1) to the end of week 24
Bleeding events
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
Health-related quality of life (HRQoL)
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 5 Jan 2024.
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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.
Xiao-Hui Zhang principal investigator
Peking University Institute of Hematology, 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 Teriflunomide 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 7 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 5 Jan 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 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, 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?
Sustained response (time frame: From the start of study treatment (Day 1) to the end of week 24). 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 5 Jan 2024.