a study to evaluate the efficacy and safety of cc-90001 in subjects with idiopathic pulmonary fibrosis

Conditions: Idiopathic Pulmonary Fibrosis; Fibrosis; Idiopathic Interstitial Pneumonias; Pathologic Processes; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Summary: this is a phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, pharmacokinetics (pk), quality of life and exploratory pharmacodynamics (pd) of two treatment doses of cc-90001, 200 mg and 400 mg, compared with placebo, when delivered once daily per os (po) in subjects with idiopathic pulmonary fibrosis (ipf). this study is designed to assess response to treatment by using measures of lung function, disease progression, fibrosis on radiography, and patient-reported outcomes. it will also assess dose response.

approximately 165 adult male and female subjects with a confirmed diagnosis of idiopathic pulmonary fibrosis (ipf) (according to the most recent ipf guideline for diagnosis and management) will be randomized 1:1:1 (55 subjects per arm) to treatment with oral cc-90001or matching placebo for an initial 24 weeks. the randomization will be stratified based on the concurrent administration of soc (yes/no). subjects completing the 24-week double-blind treatment phase will continue onto the 80-week active treatment extension phase. at week 24, all subjects originally randomized to receive placebo will be re-randomized 1:1 to blinded cc-90001 (200 mg or 400 mg po qd). during the 80-week active treatment extension phase, all subjects not on concurrent soc therapy will have the opportunity, if deemed appropriate by the investigator, to receive allowed standard of care (soc). the exploratory progressive pulmonary fibrosis (ppf) sub study will evaluate the efficacy, safety, pk, quality of life and exploratory pd of one po treatment dose regimen of cc-90001, compared with placebo, for an initial 24 weeks of treatment, in subjects with ppf and long-term safety in the 80-week active treatment extension phase when all ppf subjects will receive cc-90001. approximately 45 non-soc subjects will be randomized in this sub study. all subjects who complete the study treatment phases and those subjects who discontinue investigational product (ip) prior to the completion of the study will participate in the 4-week post-treatment observational follow-up phase. the study will be conducted in compliance with the international council harmonisation (ich) of technical requirements for registration of pharmaceuticals for human use/good clinical practice (gcp) and applicable regulatory requirements. an external dmc, comprised of independent physician experts and a statistician who are not affiliated with the sponsor and for whom there is no identified conflict of interest will be responsible for safeguarding study participants' interests and for monitoring the overall conduct of the study.

Eligibility criteria:
inclusion criteria: subject understands and has voluntarily signed and dated an informed consent form 1. subject is male or female ≥ 40 years of age 2. diagnosis of ipf is supported by hrct and historical lung biopsy (surgical lung biopsy \[slb\] or cryobiopsy) if available according to guidelines. 3. no features supporting an alternative diagnosis on transbronchial biopsy, bronchoalveolar lavage (bal), or slb, if performed. 4. percent predicted forced vital capacity (% fvc) ≥ 45% and ≤ 95% at screening 5. percent predicted diffusion capacity of the lung for carbon monoxide (dlco) ≥ 25% and ≤ 90% predicted at screening. 6. able to walk ≥ 150 meters during the 6-minute walk test (6mwt) at screening 7. females of childbearing potential (fcbp) must commit to true abstinence or agree to use two effective birth control methods. 8. male subjects must practice true abstinence or use a barrier method of contraception. 9. additional inclusion criteria apply. progressive pulmonary fibrosis (ppf) sub-study: 1. met all inclusion criteria described for ipf subjects other than inclusion criterion 5. 2. features of diffuse fibrosing lung disease of \> 10% on hrct by central reading. 3. investigator-documented ≥ 5% annualized relative decline in fvc in past 24 months from screening visit 1 exclusion criteria: the presence of any of the following will exclude a subject from enrollment: 1. subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study. 2. subject with a qtcf \> 450 msec. 3. evidence of clinically relevant airways obstruction at screening. 4. subjects using therapy targeted to treat ipf. 5. history of latent or active tb, unless there is medical record documentation of successful completion of a standard course of treatment 6. history of hepatitis b and/or hepatitis c, including those considered successfully treated/cured 7. pregnancy or lactation. 8. additional exclusion criteria apply.

Keywords: idiopathic pulmonary fibrosis (ipf) pulmonary fibrosis cc-90001 safety efficacy ipf idiopathic pulmonary fibrosis