Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

NCT04193592 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: 14172/2019
• Study Type: interventional
• Target: 50
• Locations: 2 countries
• Sites: 2

Brief 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.

Conditions

Hermansky Pudlak Syndrome; Interstitial Lung Disease

Keywords

safety; efficacy; patient reported outcomes

Outcome Measures

Primary Outcomes (1)

• Change in Forced Vital Capacity (FVC)

  The incidence of decline in percent predicted FVC of 10 % or greater from baseline measured at 6 and 12 months

  baseline, 6 months, 12 months

Secondary Outcomes (4)

• Change in Forced Vital Capacity (FVC)

  week 52

• Change in Diffusion Capacity (DLCO)

  week 52

• Incidence of Treatment Emergent Adverse Events

  week 52

• Incidence of Treatment Emergent Serious Adverse Events

  week 52

Study Arms (1)

Oral Pirfenidone 2403 mg per day

EXPERIMENTAL

Enrolled subjects will receive oral pirfenidone 801 mg taken three times a day. Pirfenidone will be supplied in 267 mg capsules.

Drug: Pirfenidone

Interventions

Pirfenidone DRUG

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.

Also known as: Esbriet

Oral Pirfenidone 2403 mg per day

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Probable or definite diagnosis of HPS based on confirmed genetic mutation or clinical picture characterized by oculo-cutaneous albinism, bleeding disorder, and possible colitis and ILD.
• Diagnosis of ILD supported by clinically indicated HRCT prior to Screening, and presence of fibrotic abnormality affecting more than 5% of the lung parenchyma, with or without traction bronchiectasis or honeycombing, on Screening
• No features supporting an alternative diagnosis (e.g., infection)
• Change in pre-bronchodilator FVC (measured in liters) between Screening (Visit 1) and
• Baseline (Visit 2) must be a \< 10% relative difference, calculated as:
• %\*\[absolute value (Screening FVC - Baseline FVC)/Screening FVC
• Stable dose (at least three months at the time of Screening) of corticosteroids.
• No cytotoxic, immunosuppresive agents, cytokine-modulating, or receptor antagonists agents are allowed (including but not limited to azathioprine, cyclophosphamide, cyclosporine, etanercept, iloprost, infliximab, methotrexate, mycophenolate mofetil, nintedanib, tacrolimus, tetrathiomolybdate, TNF-α inhibitors, rituximab, abatacept, tofacitintib, tociluzimab).
• Able to understand and sign a written informed consent form

You may not qualify if:

• Not a suitable candidate for enrollment or unlikely to comply with the requirements of this study, in the opinion of the investigator
• Cigarette smoking within 3 months of Screening or unwilling to avoid tobacco products throughout the study
• History of clinically significant environmental exposure known to cause pulmonary fibrosis (PF), including but not limited to drugs (such as amiodarone), asbestos, beryllium, radiation, and domestic birds
• Concurrent presence of other interstitial lung disease, including but not limited to radiation, drug toxicity, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans organizing pneumonia, human immunodeficiency virus (HIV), viral hepatitis, and cancer
• Concurrent presence of other pleuropulmonary manifestations inconsistent with HPS- ILD
• Presence of pleural effusion occupying more than 10% of the hemithorax on Screening HRCT
• Clinical diagnosis of a connective tissue disease or overlap syndrome (including but not limited to rheumatoid arthritis, scleroderma, polymyositis/dermatomyositis, systemic lupus erythematosus)
• Coexistent clinically significant COPD/emphysema or asthma in the opinion of the site principle investigator
• Clinical evidence of active infection, including but not limited to bronchitis, pneumonia, sinusitis, urinary tract infection, or cellulitis
• Any history of malignancy diagnosed within 5 years of screening, other than basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or low grade cervical carcinoma in situ.
• History of severe hepatic impairment or end-stage liver disease
• History of end-stage renal disease requiring dialysis
• History of unstable or deteriorating cardiac or disease, myocardial infarction within the previous year, heart failure within the last 3 years, or cardiac arrhythmia requiring drug therapy
• Any condition that, in the opinion of the investigator, might be significantly exacerbated by the known side effects associated with the administration of pirfenidone
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use two adequate methods of contraception, including at least one method with a failure rate of \<1% per year, during the 52 weeks of treatment.

Sponsors & Collaborators

• Jesse Roman: lead
• Genentech, Inc.: collaborator

Study Sites (2)

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Mayaguez Medical Center

Mayagüez, 00680, Puerto Rico

Location

MeSH Terms

Conditions

Hermanski-Pudlak Syndrome; Lung Diseases, Interstitial

Interventions

pirfenidone

Condition Hierarchy (Ancestors)

Albinism, Oculocutaneous; Albinism; Eye Diseases, Hereditary; Eye Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Platelet Storage Pool Deficiency; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Skin Diseases, Genetic; Hypopigmentation; Pigmentation Disorders; Skin Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Jesse Roman, MD

  Thomas Jefferson University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tamra Perez, BSN

CONTACT

1-215-955-9181; tamra.perez@jefferson.edu

Melissa McCarey

CONTACT

267 503-7417; Melissa.McCarey@jefferson.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine

Model Details: open label drug

Study Record Dates

• First Submitted: November 6, 2019
• First Posted: December 10, 2019
• Study Start: December 1, 2019
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: December 10, 2019

Record last verified: 2019-12

Data Sharing

• IPD Sharing: Will not share

Locations

PR (1); US (1)