Study to Evaluate the Efficacy, Safety, and Tolerability of PIPE 791 in Subjects With Idiopathic Pulmonary Fibrosis

Official title

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of Oral PIPE 791 in Subjects With Idiopathic Pulmonary Fibrosis

Overview

This is a Ph 2, randomized, double-blind, placebo-controlled global multicenter study to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of PIPE-791 in participants with a diagnosis of Idiopathic Pulmonary Fibrosis (IPF) with or without background treatment.

Detailed description

This is a Ph 2, randomized, double-blind, placebo-controlled global multicenter study to evaluate the efficacy, safety, tolerability, and PK of PIPE-791 in participants with a diagnosis of Idiopathic Pulmonary Fibrosis with or without background treatment. The treatment period is 26 weeks and full study duration is up to 36 weeks including Screening and Follow-Up. Approximately 324 participants will be enrolled into one of three treatment arms, PIPE-791 Dose A, PIPE-791 Dose B, or placebo.

Interventions

• Drug PIPE-791 Dose A
  Participants will receive a daily oral dose of PIPE-791 in tablet form
• Drug PIPE-791 Dose B
  Participants will receive a daily oral dose of PIPE-791 in tablet form
• Drug Placebo
  Participants will receive a daily oral dose of matching Placebo in tablet form

Primary outcome measures

• Absolute change in forced vital capacity (FVC) (mL) [Time frame: From baseline to Week 26]

Secondary outcome measures (7)

• To investigate the safety and tolerability of PIPE-791 compared to placebo based on percentage of treatment-emergent adverse events (TEAE) [Time frame: From baseline to Week 30]
• Relative change in FVC (mL) [Time frame: From baseline to Week 26]
• Proportion of participants with a ≥10% absolute decline in percent predicted FVC (ppFVC) [Time frame: From baseline to Weeks 12 and Week 26]
• Time to first ≥10% absolute decline in ppFVC [Time frame: From baseline to time of first ≥10% absolute decline, up to Week 26]
• Absolute change in ppFVC [Time frame: From baseline to Week 26]
• Relative change in ppFVC [Time frame: From baseline to Week 26]
• Change in quantitative CT-derived lung fibrosis burden on HRCT [Time frame: From baseline to Week 26]

Eligibility criteria

Inclusion criteria

• Male or female ≥ 40 years of age at the time of Randomization.
• A diagnosis of IPF within 7 years prior to Screening, based on the 2022 ATS/ERS/JRS/ALAT practice guideline as confirmed by the Investigator, and a centrally read screening HRCT consistent with usual interstitial pneumonia (UIP) or probable UIP.
• Percent predicted (pp) FVC ≥ 40% on Screening spirometry.
• Participants may enter the study whether or not they are receiving background nintedanib or pirfenidone therapy approved for the treatment of IPF, but not both concurrently.

Exclusion criteria

• Those with a history of interstitial lung disease (ILD) other than IPF are not eligible.
• Those with pulmonary arterial hypertension (PAH) requiring multi-drug therapy are not eligible.
• Those who have experienced an IPF exacerbation within 6 weeks of Screening, or during Screening, are not eligible.
• Those with an estimated glomerular filtration rate (eGFR) ≤ 30 ml/min/1.73 m2 (Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] formula) (Inker 2021) or who have Child-Pugh Class B or C hepatic impairment are not eligible.
• Female participants must not be of childbearing potential.

Additional inclusion and exclusion criteria apply.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

Argentina · 11 centers

• Instituto de Especialidades de la Salud Rosario - Clínica del Tórax — Rosario
• Instituto Médico de la Fundación Estudios Clínicos — Rosario
• Centro de Investigaciones Médicas Tucumán - PPDS — San Miguel de Tucumán
• CIMER-Centro Integral de Medicina Respiratoria — San Miguel de Tucumán
• IPR-Investigaciones en Patologías Respiratorias — San Miguel de Tucumán
• Centro Modelo de Cardiología-Laprida 554 — San Miguel de Tucumán
• STAT Research S.A. — Buenos Aires
• Consultorios Médicos Organización del Buen Ayre SRL — Buenos Aires
• … and 3 more centers

Israel · 11 centers

• Shamir Medical Center — Be’er Ya‘aqov
• Meir Medical Center — Kafr Saba
• Rabin Medical Center - PPDS — Petah Tikva
• The Chaim Sheba Medical Center - PPDS — Ramat Gan
• Kaplan Medical Center — Rehovot
• Hillel Yaffe Medical Center — Hadera
• Hadassah Medical Center- Ein Kerem - PPDS — Jerusalem
• Assuta Ashdod University Medical Center — Ashdod
• … and 3 more centers

South Korea · 8 centers

• The Catholic University of Korea - Eunpyeong St. Mary's Hospital — Seoul
• The Catholic University of Korea, Bucheon St. Mary's Hospital — Bucheon-si
• Seoul National University Bundang Hospital — Seongnam-si
• Severance Hospital Yonsei University Health System — Seoul
• Samsung Medical Center — Seoul
• Soon Chun Hyang University Hospital — Seoul
• Gachon University Gil Medical Center — Incheon
• Korea University Anam Hospital — Seoul

Australia · 7 centers

• Royal Prince Alfred Hospital — Camperdown
• Westmead Hospital — Westmead
• The Prince Charles Hospital — Chermside
• Austin Health — Heidelberg
• The Alfred Hospital — Melbourne
• Institute for Respiratory Health — Midland
• TrialsWest Pty Ltd — Spearwood

United Kingdom · 7 centers

• NIHR Leicester Biomedical Research Centre - Glenfield Hospital — Leicester
• St Bartholomew's Hospital - PPDS — London
• Southampton General Hospital — Southampton
• Royal Infirmary of Edinburgh — Edinburgh
• Perth Royal Infirmary — Perth
• Birmingham Heartlands Hospital — Birmingham
• Royal Brompton Hospital — London

Canada · 5 centers

• Vancouver General Hospital — Vancouver
• Toronto General Hospital — Toronto
• Dynamic Drug Advancement Ltd. — Ajax
• Dr. Anil Dhar Medicine Professional Corporation — Windsor
• Centre d'investigation Clinique Mauricie — Trois-Rivières

New Zealand · 4 centers

• Greenlane Clinical Centre — Epsom
• Aotearoa Clinical Trials Trust - Middlemore Hospital - PPDS — Papatoetoe
• Canterbury Respiratory Research Group — Christchurch
• Health New Zealand - Te Whatu Ora - Waikato Hospital — Hamilton

Serbia · 4 centers

• Institute of Pulmonary Diseases of Vojvodina — Kamenitz
• University Clinical Center of Serbia - Koste Todorovica - PPDS — Belgrade
• University Clinical Center Kragujevac — Kragujevac
• University Clinical Center Nis — Niš

Chile · 3 centers

• Centro de Investigacion Curicó — Curicó
• CEC SpA — Ñuñoa
• Biocinetic Servicios Medicos Ltda — Santiago

Identifiers

NCT: NCT07284459 · CTX-791-201

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗