A Study of Oral Vismodegib for the Treatment of Idiopathic Pulmonary Fibrosis (IPF)

• Register: ClinicalTrials.gov
• Last refreshed on: 14 November 2016
• Main ID: NCT02168530
• Date of registration: 18/06/2014
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: A Study of Oral Vismodegib for the Treatment of Idiopathic Pulmonary Fibrosis (IPF)
• Date of first enrolment: October 2014
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02168530
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Australia, France, Germany, Israel, Korea, Republic of, Mexico, Peru, United States

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion criteria

Inclusion Criteria:

- Adult patients aged >/=40 years at Visit 1

- Diagnosis of IPF within the previous 5 years from time of screening and confirmed at
baseline

- Patients from countries where a treatment is licensed/approved for IPF must
additionally meet at least one of the following criteria to be eligible: (1) be
unable to access a licensed therapy for IPF; (2) treatment with a licensed
therapy/therapies has been stopped for lack of efficacy or because of
safety/tolerability reasons (a washout period will be required); (3) be unwilling to
be treated with a licensed therapy and study enrollment considered appropriate.

- Forced vital capacity (FVC) >/=40% and
- Stable baseline lung function as evidenced by a difference of <10% in absolute FVC
measurements (in liters) between screening and Day 1/Visit 2 prior to randomization

- Diffusion capacity of the lung for carbon dioxide (DLCO) >/=25% of predicted at
screening

- Adequate hematopoietic capacity, liver and renal function

- Female patients of childbearing potential must use two methods of acceptable
contraception, including one highly effective method and a barrier method, during
treatment and for 7 months after completion of study treatment (or as per local
requirement)

- Male patients must agree to remain abstinent or use a condom, even after a vasectomy,
during sexual intercourse with female partners while being treated with
vismodegib/placebo, and for 2 months after completion of study treatment

- Agreement not to donate blood or blood products during the study and for at least 7
months (or as per local requirements) after the last dose of study treatment

Exclusion Criteria:

- Pregnant or lactating

- Known hypersensitivity to any of the study drug excipients or the drug itself

- Prior treatment with vismodegib or any Hh-pathway inhibitor

- Evidence of other known causes of interstitial lung disease

- Hospitalization due to an exacerbation of IPF within 4 weeks prior to, or during,
screening

- Lung transplant expected within 12 months of screening

- Evidence of clinically significant lung disease other than IPF

- Substantial emphysema on high resolution computed tomography (HRCT) with degree of
emphysema greater than fibrosis

- Post bronchodilator forced expiratory volume in 1 second/FVC ratio <0.7 at screening

- Class IV New York Heart Association chronic heart failure or historical evidence of
left ventricular ejection fraction <35%

- Known current malignancy or current evaluation for a potential malignancy

- Known immunodeficiency, including but not limited to HIV infection

- Any clinically significant medical disease (other than IPF) that is associated with
an expected survival of <12 months, likely to require a change in therapy during the
study, or likely to impact the ability of the patient to participate in the study in
the opinion of the investigator, or impact the study efficacy or safety assessments



Age minimum: 40 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Idiopathic Pulmonary Fibrosis

Intervention(s)

Drug: vismodegib

Drug: placebo

Primary Outcome(s)

Change in forced vital capacity (FVC) percent predicted [Time Frame: From baseline to Week 52]

Secondary Outcome(s)

Change in Quality of Life measurements [Time Frame: From baseline to Week 52]

Time from randomization to first event of acute IPF exacerbation [Time Frame: Up to 52 weeks]

Annualized rate of change in FVC [Time Frame: Week 52]

Progression-free survival [Time Frame: Week 52]

Change in diffusion capacity of the lung for carbon dioxide (DLCO) [Time Frame: From baseline to Week 52]

Safety: Incidence of adverse events (AEs) [Time Frame: Approximately 60 weeks]

Secondary ID(s)

2014-000963-42

GB29298

Source(s) of Monetary Support

Please refer to primary and secondary sponsors