IDIOPATHIC PULMONARY FIBROSIS MedDRA version: 20.0 Level: LLT Classification code 10067761 Term: Exacerbation of idiopathic pulmonary fibrosis System Organ Class: 100000004855
Conditions
Interventions
Product Code: CC-90001
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Not available
CAS Number: 1403859-14-2
Current Sponsor code: CC-90001
Concentration unit: mg milligram(s)
Concentrat
Sponsors
Celgene Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject is male or female = 40 years of age at the time of signing the informed consent form (ICF) 2. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted 3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements 4. Investigator has considered all available IPF treatment options with the potential subject before consenting the subject for participation in the study 5. Diagnosis of IPF is supported by HRCT as described in the Protocol 6. Extent of fibrotic changes (eg, honeycombing, reticular changes) greater than the extent of emphysema on HRCT scan, as determined by central review 7. No features supporting an alternative diagnosis on transbronchial biopsy, bronchoalveolar lavage (BAL), or SLB, if performed 8. Percent predicted forced vital capacity ( FVC) = 45% at Screening confirmed by central review 9. Change in FVC (measured in milliliters [mL]) between Screening and Day 1 less than a 10% relative difference, calculated as: the absolute value of 100% * (Screening FVC [mL] - Day 1 FVC [mL]) / Screening FVC (mL) 10.Hemoglobin-corrected percent predicted diffusion capacity of the lung for carbon monoxide (DLCO) = 25% and = 85% predicted at Screening 11. Able to walk = 150 meters during the 6-minute walk test (6MWT) at Screening 12. Investigator has considered all available IPF treatment options with the potential subject before consenting the subject for participation in the study Females of childbearing potential (FCBP) 1 must: a. Have two negative pregnancy tests as verified by the Investigator prior to starting IP. She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact. [refer to protocol] b. Either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use two effective birth control methods (one of which is highly effective) at the same time, and be able to comply with, effective contraception without interruption, 28 days prior to starting IP, during the study therapy (including dose interruptions), and for 28 days after discontinuation of IP [Refer to protocol] 13. Male subjects must: Practice true abstinence (which must be reviewed on a monthly basis) or agree to use a latex condom or nonlatex condom not made out of natural (animal) membrane (eg, polyurethane) during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 28 days following IP discontinuation, even if he has undergone a successful vasectomy. 14. For subjects stratified to the protocol-allowable standard of care therapy group (only): Subjects must be receiving and agree to maintain the same dose of protocol-allowable standard of care (SOC) therapy for at least 8 weeks prior to Screening Visit 1 and must agree to continue this dose through Visit 9/Week 24. Adjustments in pirfenidone dose after randomization (Visit 2) may be allowed for safety or tolerability reasons, according to the pirfenidone label. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 62 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 148
Exclusion criteria
Exclusion criteria: 1. Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study 2. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study 3. Subject has any condition that confounds the ability to interpret data from the study 4. Significant clinical worsening of IPF between Screening and Baseline (Visit 2), in the opinion of the Investigator 5. Subjects with any of the following laboratory criteria: • White blood cell count (WBC) 14,000/mm3 (> 14 X 109/L) • Platelet count 1.5 mg/dL (> 132.6 µmol/L) • Aspartate aminotransferase (AST/SGOT) > 1.5 X upper limit of normal (ULN) • Alanine aminotransferase (ALT/SGPT) > 1.5 X upper limit of normal (ULN) • Total bilirubin > 2 mg/dL (> 34.2 µmol/L) • Hemoglobin 450 msec 7. Any condition other than IPF that in the opinion of the Investigator is likely to result in the death of the subject within the next year 8. Inability to obtain reproducible, high-quality pulmonary function tests. 9. Evidence of clinically relevant airways obstruction (ie, FEV1/FVC 12.5 mg/day or equivalent) within 4 weeks prior to the Screening Visit. (Note: Refer to protocol.) 13. Use of any cytokine modulator/biologic, such as etanercept, adalimumab, efalizumab, infliximab, or rituximab within 12 weeks of randomization 14. Use of an inhaled long-acting bronchodilator within 24 hours of the Screening Visit or short-acting bronchodilator within 8 hours of the Screening Visit 15. Use of drugs that are known to cause hepatotoxicity, such as, but not limited to, acetaminophen (paracetamol) at dosages of > 3 grams/day and niacin dosage of > 2 grams/day while on study or within 2 weeks of first dose of IP 16. Use of any medications that are substrates of one or more of the transporters P-gp, BCRP, OAT3, OATP1B1, OATP1B3, and OCT2 and have a narrow therapeutic index (eg, digoxin, mycophenolate mofetil) 17. History of recent (within 6 months of Screening) deep vein thrombosis (DVT) or pulmonary embolism (PE) and/or recurrent DVT or recurrent PE 18. History of cardiac valve replacement requiring chronic anticoagulation therapy 19. History of congenital and/or acquired immunodeficiencies (eg, common variable immunodeficiency, human immunodeficiency virus [HIV], etc) 20. History of hepatitis B and/or hepatitis C, including those consider
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effect of CC-90001, 200 mg and 400 mg, when orally administered (PO) once daily (QD), compared with placebo, on percent of predicted forced vital capacity (FVC) after 24 weeks of treatment in subjects with IPF.;Secondary Objective: To evaluate the effects of CC-90001, 200 mg and 400 mg PO QD, compared to placebo, after 24 weeks of treatment in subjects with IPF, on: - FVC (milliliters [mL]) - Six-minute Walk Test (6MWT) - Disease progression - Heath-related quality of life: St. George’s Respiratory Questionnaire (SGRQ) and University of California San Diego-Shortness of Breath Questionnaire (UCSDSOBQ) - Dose response - Safety and tolerability;Primary end point(s): Forced vital capacity (FVC) - Percentage point difference in % predicted FVC;Timepoint(s) of evaluation of this end point: Baseline to Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. FVC - Absolute change and rate of decline in FVC (expressed in mL) 2. 6-minute Walk Test (6MWT) with Borg Scale: - Change in the distance walked during the 6MWT as measured in meters (m) - Change in dyspnea rating on Borg Scale 3. Disease progression: - Death from respiratory failure, or - Absolute decrease of = 10% from baseline in % predicted FVC at two consecutive evaluations at a minimum of 4 weeks between evaluations, or - Decrease from baseline of = 50 meters in 6MWT distance (in the absence of a readily explainable cause, such as injury or trauma), or - Unexplained worsening hypoxemia (an absolute decrease from baseline of 4% or more in arterial oxygen saturation by pulse oximetry [SpO2]).). 4. Quality of life : - Change from Baseline in total score and domains including cough on the Saint George’s Respiratory Questionnaire (SGRQ) - Change from Baseline in the University of California San Diego Shortness of Breath Questionnaire (UCSD- SOBQ) 5. Safety and tolerability - Type, frequency, severity, and relationship of AEs, clinical laboratory tests including urine cytology, 12-lead ECG, vital signs, and physical examination;Timepoint(s) of evaluation of this end point: 1. FVC - Baseline through Week 24 2. 6-minute Walk Test (6MWT) with Borg Scale: - Baseline to Week 24; - Baseline to Week 52; - Baseline to Week 76; - Baseline to Week 104; - Week 24 to Week 52; - Week 24 to Week 104 3. Disease progression - Baseline through Week 24 4. Quality of life - Baseline through Week 24 5. Safety and tolerability - Signing of the informed consent form through Week 108 (4-week post-treatment observational follow-up) | — |
Countries
Australia, Brazil, Canada, Colombia, France, Germany, Greece, Romania, Russian Federation, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trial Disclosure
Celgene Corporation
Outcome results
None listed