Immune ThrombocytopeniaPhase 1/2NCT03460808
The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
A Phase 1/2 study testing atorvastatin, Acetylcysteine and Danazol in people with Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
Single-arm, open-lable, multicentre study to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroidresistant/relapsed ITP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- atorvastatin, Acetylcysteine and Danazol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteChina
- Study dates
- 10 Mar 2018to 1 Jan 2023
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ITP confirmed by excluding other supervened causes of thrombocytopenia;
- Platelet count of less than 30×10^9/L at 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;
- ECOG<2.
- EPCs in bone marrow less than 0.02%
Who cannot join
- Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
- congestive heart failure
- severe arrhythmia
- nursing or pregnant women
- aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
- creatinine or serum bilirubin levels each 1.5 times or more than the normal range
- active or previous malignancy
- Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Xiao-hui Zhang, Professor, zhangxh100@sina.com
About this study
Single-arm, open-lable, multicentre study to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroidresistant/relapsed ITP.
Read the detailed description
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. Atorvastatin was shown to enhance bone marrow endothelial cell function and N-acetylcysteine (NAC) was shown to inhibit PLT binding to endothelial cell.
A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to atorvastatin, acetylcysteine plus danazol with danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.
- Conditions
- Immune Thrombocytopenia
- Being tested
- atorvastatin, Acetylcysteine, Danazol
- Keywords
- steroid-resistant, refractory, Atorvastatin, Acetylcysteine, 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.
atorvastatinDrug
Atorvastatin was used in combination with acetylcysteine and danazol.
AcetylcysteineDrug
Acetylcysteine was used in combination with atorvastatin and danazol.
DanazolDrug
Also called Danocrine, Cleregil, Danol
Danazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy
| Study group | Role | What they receive |
|---|---|---|
| atorvastatin, acetylcysteine & danazol | Experimental | atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks Receives: Drug: atorvastatin; Drug: Acetylcysteine; Drug: Danazol |
What is being measured
The primary outcome is the main question the study is designed to answer.
the sustained platelet response at the 6-month follow-up
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count >=100x10^9/L (CR) and the absence of bleeding, without rescue medication at 6-month follow-up.
Measured: From the start of study treatment (Day 1) up to the end of Month 6
Secondary outcomes (4)
overall response
The number of participants with platelet count >=30×10^9/L at least once and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy.
Measured: From the start of study treatment (Day 1) up to the end of Month 6
time to response
Time to response was defined as the time from starting treatment to the time to achieve the response.
Measured: From the start of study treatment (Day 1) up to the end of Year 2
duration of response
Duration of response was measured from the achievement of response to the loss of response.
Measured: From the start of study treatment (Day 1) up to the end of Year 2
incidence of treatment-emergent adverse events
All patients were assessed for treatment-emergent adverse events every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Measured: From the start of study treatment (Day 1) up to the end of Year 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2018
- Study start10 Mar 2018 (estimated)
- Primary completion1 Jan 2020 (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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 200 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
- NCT03460808
- Sponsor's protocol ID
- Z171100001017084
- Status last verified
- Mar 2018
- First submitted
- 5 Mar 2018
- Last updated
- 9 Mar 2018
- 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 9 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT03460808
- Acronym / sponsor ID
- Z171100001017084
- Conditions
- Immune Thrombocytopenia
- Keywords
- steroid-resistant, refractory, Atorvastatin, Acetylcysteine, Danazol
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 10 Mar 2018 (estimated)
- Primary completion
- 1 Jan 2020 (estimated)
- Study completion
- 1 Jan 2023 (estimated)
- First posted
- 9 Mar 2018 (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-lable, multicentre study to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroidresistant/relapsed ITP.
Detailed description
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. Atorvastatin was shown to enhance bone marrow endothelial cell function and N-acetylcysteine (NAC) was shown to inhibit PLT binding to endothelial cell.
A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to atorvastatin, acetylcysteine plus danazol with danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| atorvastatin, acetylcysteine & danazol | Experimental | Drug: atorvastatin, Drug: Acetylcysteine, Drug: Danazolatorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- ITP confirmed by excluding other supervened causes of thrombocytopenia;
- Platelet count of less than 30×10^9/L at 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;
- ECOG<2.
- EPCs in bone marrow less than 0.02%
Key exclusion criteria (8 listed)
- Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
- congestive heart failure
- severe arrhythmia
- nursing or pregnant women
- aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
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 |
|---|---|---|
| atorvastatinAtorvastatin was used in combination with acetylcysteine and danazol. | Drug | atorvastatin, acetylcysteine & danazol |
| AcetylcysteineAcetylcysteine was used in combination with atorvastatin and danazol. | Drug | atorvastatin, acetylcysteine & danazol |
| DanazolAlso: Danocrine, Cleregil, DanolDanazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy | Drug | atorvastatin, acetylcysteine & danazol |
Primary outcome
the sustained platelet response at the 6-month follow-up
Time frame: From the start of study treatment (Day 1) up to the end of Month 6
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count >=100x10^9/L (CR) and the absence of bleeding, without rescue medication at 6-month follow-up.
4 secondary outcomes
overall response
Time frame: From the start of study treatment (Day 1) up to the end of Month 6
time to response
Time frame: From the start of study treatment (Day 1) up to the end of Year 2
duration of response
Time frame: From the start of study treatment (Day 1) up to the end of Year 2
incidence of treatment-emergent adverse events
Time frame: From the start of study treatment (Day 1) up to the end of Year 2
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2018.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing atorvastatin, Acetylcysteine 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 5 inclusion and 8 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 9 Mar 2018. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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, Professor (+86 010-88324516, zhangxh100@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the sustained platelet response at the 6-month follow-up (time frame: From the start of study treatment (Day 1) up to the end of Month 6). 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 9 Mar 2018.