Leflunomide for Idiopathic Pulmonary Hemosiderosis in Children (Lef for IPH)

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.

Study Overview

• Status: Recruiting
• Conditions: Idiopathic Pulmonary Hemosiderosis; Leflunomide
• Intervention / Treatment: Drug: Leflunomide; Drug: Steroid Drug

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 Type: Interventional
• Enrollment (Estimated): 34
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Haiyan Wang, MD; Phone Number: 8613560489257; Email: wanghy78@mail.sysu.edu.cn
• Study Contact Backup: Name: Weiping Tan, PhD; Phone Number: 8613556196566; Email: tanweip@mail.sysu.edu.cn

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510120
      • Recruiting
      • Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
      • Contact: Weiping Tan, PhD; Phone Number: +8613556196566; Email: tanwp@mail.sysu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age range from 6 months to 18 years old;
2. Diagnose as IPH;
3. Parents or guardians agree to treatment and sign a written informed consent form.

Exclusion Criteria:

1. Individuals who are allergic to any ingredients used in leflunomide tablets;
2. 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);
3. Patients with other lung diseases;
4. Have a history of abnormal coagulation or abnormal coagulation function in the past;
5. Clinical trial participants who have previously participated in the treatment of flumiphene;
6. Other situations where the researcher deems it inappropriate to participate in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Times of pulmonary hemorrhage	Annual times of acute episodes of pulmonary hemorrhage	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of adverse reaction	The frequency of adverse reactions and their relationship with leflunomide	12 months

Collaborators and Investigators

• Sponsor: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2023
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: June 30, 2023
• First Submitted That Met QC Criteria: June 30, 2023
• First Posted (Actual): July 10, 2023

Study Record Updates

• Last Update Posted (Actual): July 10, 2023
• Last Update Submitted That Met QC Criteria: June 30, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: glucocorticoid; Leflunomide; Idiopathic pulmonary hemosiderosis
• Additional Relevant MeSH Terms: Metabolic Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Iron Metabolism Disorders; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Iron Overload; Lung Diseases; Hemochromatosis; Hemosiderosis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Immunosuppressive Agents; Immunologic Factors; Leflunomide
• Other Study ID Numbers: 2021-KY-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No