Immune ThrombocytopeniaPhase 2NCT06176911
The Combination of Teriflunomide and Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia
A Phase 2 study testing Teriflunomide and Danazol in people with Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse 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 and Danazol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 124 participants
- Where
- 1 siteChina
- Study dates
- 5 Dec 2023to 15 Jul 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
- Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
- 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 bleeding manifestations at the enrollment;
- Willing and able to sign written informed consent.
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;
- Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
- Pregnancy or lactation;
- Current or recent (< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
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- 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;
- 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, Beijing Municipality, 100010
Xiao-hui Zhang, MD, +8613522338836, zhangxh@bjmu.edu.cn
Li-Ping Yang, MD, +8618519172033, lpyangvip@163.com
About this study
Official title: A Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial Comparing the Efficacy and Safety of Teriflunomide Plus Danazol in Patients With Steroid-resistant/Relapse ITP
To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP
Read the detailed description
This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Teriflunomide, Danazol
- Keywords
- Teriflunomide, Danazol
- 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 are made throughout the study based on individual platelet counts.
DanazolDrug
200 mg twice daily.
| Study group | Role | What they receive |
|---|---|---|
| Teriflunomide plus danazol | Experimental | Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request. Receives: Drug: Teriflunomide; Drug: Danazol |
| Danazol | Active comparator | Danazol was given at a dose of 200 mg twice a day for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request. Receives: Drug: Danazol |
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 (7)
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
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
Duration of response
The time from the achievement of a complete response or a partial response to the loss of response.
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
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 posted20 Dec 2023
- Study start5 Dec 2023
- Primary completion30 Dec 2024 (estimated)
- Study completion15 Jul 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients were randomly assigned at a 1:1 ratio to receive teriflunomide plus danazol or danazol. Each group requires 62 patients (considering a 10% drop-off).
- Masking
- None (open label)
- Enrolment
- 124 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Beijing Luhe Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Beijing Hospital, Beijing Friendship Hospital, Peking University First Hospital, Peking University Third Hospital, China-Japan Friendship Hospital, Beijing Tsinghua Changgeng Hospital
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiao-Hui Zhang, MD, Principal investigator, Peking University Institute of Hematology, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06176911
- Sponsor's protocol ID
- PKU-TFITP-01
- Status last verified
- Dec 2023
- First submitted
- 6 Dec 2023
- Last updated
- 20 Dec 2023
- 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 20 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial Comparing the Efficacy and Safety of Teriflunomide Plus Danazol in Patients With Steroid-resistant/Relapse ITP
- ClinicalTrials.gov ID
- NCT06176911
- Acronym / sponsor ID
- PKU-TFITP-01
- Conditions
- Immune Thrombocytopenia
- Keywords
- Teriflunomide, Danazol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 124 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Beijing Luhe Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Beijing Hospital, Beijing Friendship Hospital, Peking University First Hospital, Peking University Third Hospital, China-Japan Friendship Hospital, Beijing Tsinghua Changgeng Hospital
- Study start
- 5 Dec 2023 (actual)
- Primary completion
- 30 Dec 2024 (estimated)
- Study completion
- 15 Jul 2025 (estimated)
- First posted
- 20 Dec 2023 (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.
To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP
Detailed description
This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients were randomly assigned at a 1:1 ratio to receive teriflunomide plus danazol or danazol. Each group requires 62 patients (considering a 10% drop-off).
| Arm | Type | Receives |
|---|---|---|
| Teriflunomide plus danazol | Experimental | Drug: Teriflunomide, Drug: DanazolOral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request. |
| Danazol | Active comparator | Drug: DanazolDanazol was given at a dose of 200 mg twice a day for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
- 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 bleeding manifestations at the enrollment;
- Willing and able to sign written informed consent.
Key exclusion criteria (9 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;
- Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
- Pregnancy or lactation;
- 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 are made throughout the study based on individual platelet counts. | Drug | Teriflunomide plus danazol |
| Danazol200 mg twice daily. | Drug | Danazol, Teriflunomide plus danazol |
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.
7 secondary outcomes
Overall response
Time frame: From the start of study treatment (Day 1) to the end of week 24
Time to response
Time frame: From the start of study treatment (Day 1) to the end of week 24
Duration of 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
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 20 Dec 2023.
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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, MD 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 and Danazol 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: status not confirmed, as of the sponsor's last update on 20 Dec 2023. 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 20 Dec 2023.