Lef for IPH: Leflunomide for Idiopathic Pulmonary Hemosiderosis in Children

Sponsor

Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University (Other)

Overall Status

Recruiting

CT.gov ID

NCT05937191

Collaborator

(none)

Enrollment

Location

Arms

Anticipated Duration (Months)

0.8

Patients Per Site Per Month

Study Details

Study Description

Brief 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 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.

Condition or Disease	Intervention/Treatment	Phase
Idiopathic Pulmonary Hemosiderosis Leflunomide	Drug: Leflunomide Drug: Steroid Drug	Phase 1/Phase 2

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

Study Type:

Interventional

Anticipated Enrollment :

34 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Leflunomide+Stroid treatment GroupLeflunomide+Stroid treatment Group

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Leflunomide is Safe and Effective for the Induction and Maintenance of Idiopathic Pulmonary Hemosiderosis Remission

Actual Study Start Date :

Jun 1, 2023

Anticipated Primary Completion Date :

Dec 31, 2026

Anticipated Study Completion Date :

Dec 31, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: IPH Patients Leflunomide+Steroid treatment	Drug: Leflunomide Leflunomide+Glucocorticoids treatment Group
Active Comparator: Control Group Steroid treatment	Drug: Steroid Drug Steroid Treatment

Arm

Intervention/Treatment

Experimental: IPH Patients

Leflunomide+Steroid treatment

Drug: Leflunomide

Leflunomide+Glucocorticoids treatment Group

Active Comparator: Control Group

Steroid treatment

Drug: Steroid Drug

Steroid Treatment

Outcome Measures

Primary Outcome Measures

Times of pulmonary hemorrhage [12 months]
Annual times of acute episodes of pulmonary hemorrhage

Secondary Outcome Measures

Rate of adverse reaction [12 months]
The frequency of adverse reactions and their relationship with leflunomide

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Months to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

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.