Idiopathic Pulmonary HemosiderosisPhase 1/2Lef for IPHNCT05937191
Leflunomide for Idiopathic Pulmonary Hemosiderosis in Children
A Phase 1/2 study testing Leflunomide and Steroid Drug in people with Idiopathic Pulmonary Hemosiderosis and Leflunomide, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University at 1 site in China.
Trial summary
The goal of this clinical trial is to compare Leflunomide in Idiopathic pulmonary hemosiderosis. The main questions it aims to answer are: The efficacy and safety of Leflunomide in Idiopathic pulmonary hemosiderosis The mechanism of leflunomide in treating Idiopathic pulmonary hemosiderosis Participants will be… Read more about this study
- Who is it for?
- Ages 5 months to 18 years
- Conditions the study is for
- Idiopathic Pulmonary Hemosiderosis, Leflunomide
- What is being tested?
- Leflunomide and Steroid Drug
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2023to 31 Dec 2026
- Phase
- Phase 1/2
- 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 5 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age range from 6 months to 18 years old;
- Diagnose as IPH;
- Parents or guardians agree to treatment and sign a written informed consent form.
Who cannot join
- Individuals who are allergic to any ingredients used in leflunomide tablets;
- Complicated with serious basic diseases (such as systemic malignant diseases, heart failure, liver and kidney failure, immune deficiency, serious infectious diseases, Lung transplantation or other patients with immediate surgical indications);
- Patients with other lung diseases;
- Have a history of abnormal coagulation or abnormal coagulation function in the past;
- Clinical trial participants who have previously participated in the treatment of flumiphene;
- Other situations where the researcher deems it inappropriate to participate in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Sun Yat-sen Memorial Hospital of Sun Yat-sen University
Guangzhou, Guangdong, 510120
Weiping Tan, PhD, +8613556196566, tanwp@mail.sysu.edu.cn
About this study
Official title: Leflunomide is Safe and Effective for the Induction and Maintenance of Idiopathic Pulmonary Hemosiderosis Remission
The goal of this clinical trial is to compare Leflunomide in Idiopathic pulmonary hemosiderosis. The main questions it aims to answer are:
- The efficacy and safety of Leflunomide in Idiopathic pulmonary hemosiderosis
- The mechanism of leflunomide in treating Idiopathic pulmonary hemosiderosis Participants will be treated with leflunomide plus stroid. A comparison group: Researchers will compare the control group treated with stroid to see if the efficacy of Leflunomide would be better than control group.
Read the detailed description
This study conducted a prospective, open, parallel controlled, single center clinical trial on the combination of Leflunomide and Stroid therapy for children with IPH, and conducted a one-year follow-up. The main indicators (pulmonary hemorrhage/hemoptysis frequency) and secondary indicators (serum iron metabolism level, T lymphocyte subpopulation, lung function, inflammatory cells, and inflammatory factors) were evaluated before and after treatment, Exploring the safety and effectiveness of leflunomide in the treatment of IPH, it is expected that the combination of leflunomide and conventional treatment can improve the remission rate of clinical symptoms in children with IPH, inhibit the progressive deterioration of lung function, and the research results are expected to bring new treatment methods and strategies for this group of patients.
- Conditions
- Idiopathic Pulmonary Hemosiderosis, Leflunomide
- Being tested
- Leflunomide, Steroid Drug
- Keywords
- Idiopathic pulmonary hemosiderosis, Leflunomide, glucocorticoid
- Medical subject headings
- Hemosiderosis, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeflunomideDrug
Leflunomide+Glucocorticoids treatment Group
Steroid DrugDrug
Steroid Treatment
| Study group | Role | What they receive |
|---|---|---|
| IPH Patients | Experimental | Leflunomide+Steroid treatment Receives: Drug: Leflunomide |
| Control Group | Active comparator | Steroid treatment Receives: Drug: Steroid Drug |
What is being measured
The primary outcome is the main question the study is designed to answer.
Times of pulmonary hemorrhage
Annual times of acute episodes of pulmonary hemorrhage
Measured: 12 months
Secondary outcomes (1)
Rate of adverse reaction
The frequency of adverse reactions and their relationship with leflunomide
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2023
- Study start1 Jun 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Pulmonary HemosiderosisStudies of the same condition, open and under way.
- More trials run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityThe 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Leflunomide+Stroid treatment Group
- Masking
- None (open label)
- Enrolment
- 34 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Weiping Tan, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05937191
- Sponsor's protocol ID
- 2021-KY-001
- Acronym
- Lef for IPH
- Status last verified
- Jun 2023
- First submitted
- 30 Jun 2023
- Last updated
- 10 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Leflunomide is Safe and Effective for the Induction and Maintenance of Idiopathic Pulmonary Hemosiderosis Remission
- ClinicalTrials.gov ID
- NCT05937191
- Acronym / sponsor ID
- Lef for IPH / 2021-KY-001
- Conditions
- Idiopathic Pulmonary Hemosiderosis, Leflunomide
- Keywords
- Idiopathic pulmonary hemosiderosis, Leflunomide, glucocorticoid
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 34 participants (estimated)
- Lead sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- 1 Jun 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 10 Jul 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.
The goal of this clinical trial is to compare Leflunomide in Idiopathic pulmonary hemosiderosis. The main questions it aims to answer are:
- The efficacy and safety of Leflunomide in Idiopathic pulmonary hemosiderosis
- The mechanism of leflunomide in treating Idiopathic pulmonary hemosiderosis Participants will be treated with leflunomide plus stroid. A comparison group: Researchers will compare the control group treated with stroid to see if the efficacy of Leflunomide would be better than control group.
Detailed description
This study conducted a prospective, open, parallel controlled, single center clinical trial on the combination of Leflunomide and Stroid therapy for children with IPH, and conducted a one-year follow-up. The main indicators (pulmonary hemorrhage/hemoptysis frequency) and secondary indicators (serum iron metabolism level, T lymphocyte subpopulation, lung function, inflammatory cells, and inflammatory factors) were evaluated before and after treatment, Exploring the safety and effectiveness of leflunomide in the treatment of IPH, it is expected that the combination of leflunomide and conventional treatment can improve the remission rate of clinical symptoms in children with IPH, inhibit the progressive deterioration of lung function, and the research results are expected to bring new treatment methods and strategies for this group of patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Leflunomide+Stroid treatment Group
| Arm | Type | Receives |
|---|---|---|
| IPH Patients | Experimental | Drug: LeflunomideLeflunomide+Steroid treatment |
| Control Group | Active comparator | Drug: Steroid DrugSteroid treatment |
Who may qualify
- Ages
- 5 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age range from 6 months to 18 years old;
- Diagnose as IPH;
- Parents or guardians agree to treatment and sign a written informed consent form.
Key exclusion criteria (6 listed)
- Individuals who are allergic to any ingredients used in leflunomide tablets;
- Complicated with serious basic diseases (such as systemic malignant diseases, heart failure, liver and kidney failure, immune deficiency, serious infectious diseases, Lung transplantation or other patients with immediate surgical indications);
- Patients with other lung diseases;
- Have a history of abnormal coagulation or abnormal coagulation function in the past;
- Clinical trial participants who have previously participated in the treatment of flumiphene;
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 |
|---|---|---|
| LeflunomideLeflunomide+Glucocorticoids treatment Group | Drug | IPH Patients |
| Steroid DrugSteroid Treatment | Drug | Control Group |
Primary outcome
Times of pulmonary hemorrhage
Time frame: 12 months
Annual times of acute episodes of pulmonary hemorrhage
1 secondary outcome
Rate of adverse reaction
Time frame: 12 months
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 10 Jul 2023.
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 1/2 study testing Leflunomide and Steroid Drug in people with Idiopathic Pulmonary Hemosiderosis and Leflunomide, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University 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 3 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 10 Jul 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 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?
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 Haiyan Wang, MD (8613560489257, wanghy78@mail.sysu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Times of pulmonary hemorrhage (time frame: 12 months). 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 10 Jul 2023.