NCT04193592

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

Pirfenidone in the Treatment of Hermansky Pudlak Syndrome (HPS) - Related Interstitial Lung Disease (ILD)

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Jesse Roman · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This research study will explore the safety and efficacy of the drug, pirfenidone, in patients with a diagnosis of Hermansky-Pudlak Syndrome (HPS) who have an associated interstitial lung disease (ILD) over a planned period of 56 weeks.

Detailed description

An open-label clinical study designed to evaluate the efficacy and safety of administering pirfeniodne for 52 weeks to subjects with HPS-ILD. Patients meeting the eligibility criteria without contraindications for the study will be provided pirfenidone 2403 mg/day. Efficacy will be evaluated through interval testing of pulmonary function tests, patient reported outcomes, adverse events and survival. Safety will be assessed by determining adverse events, hospitalizations, and all-cause mortality.

Conditions

• Hermansky Pudlak Syndrome
• Interstitial Lung Disease

Interventions

Type	Name	Description
DRUG	Pirfenidone	Pirfenidone will be titrated over 14 days, as tolerated, to the full dose of 2403 mg per day, as follows: Days 1 - 7: one capsule TID; Days 8 - 14: two capsules TID; Days 15 to week 52: three capsules TID. Dose may be reduced to manage an adverse event.

Timeline

• Start date: 2019-12-01
• Primary completion: 2022-12-31
• Completion: 2022-12-31
• First posted: 2019-12-10
• Last updated: 2019-12-10

Locations

2 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study