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Completed

NCT Number: NCT03451045

Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older. Approximately 415 subjects will be enrolled in this study at about 100 sites in North America, and Europe. The planned duration of treatment with study drug is 28 weeks.

Study drug will be lenabasum 20 mg BID, lenabasum 5 mg BID, and placebo in a 2:1:2 ratio.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Cystic Fibrosis Centre Innsbruck Medical University of Innsbruck, Dept. for Child and Adolescent Health, University Clinic for Paediatrics, Cardiology, Pneumology, Allergology, Cystic Fibrosis, Innsbruck, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • ≥ 12 years of age at the time Informed Consent/ Assent is signed.
  • Weight ≥ 40 kg.
  • FEV1 ≥ 40% predicted and < 100% predicted in the last 12 months.
  • Physician-initiated treatment with an IV antibiotic 2 or 3 times in the last 12 months for a new PEx or physician-initiated treatment with an IV antibiotic 1 time in the last 12 months plus physician-initiated treatment with oral antibiotic(s) 1 or more times in the past 12 months for a new PEX.

Exclusion criteria

  • Severe or unstable CF at screening or Visit 1.
  • Any of the following values for laboratory tests at screening:
  • A positive pregnancy test.
  • Hemoglobin < 10 g/dL in males and < 9 g/dL in females.
  • Neutrophils < 1.0 x 10^9 /L.
  • Platelets < 75 x 10^9/L.
  • Creatinine clearance < 50 mL/min according to Modification of Diet in Renal Disease (MDRD) Study equation.
  • Serum transaminases > 2.5 x upper limit of normal.
  • Any medical condition or concurrent medical therapies at screening or Visit 1 that may put the subject at greater safety risk, influence response to study drug or interfere with study assessments.

Treatment and study plan

Lenabasum 20 mg

Drug

Subjects will receive lenabasum 20 mg twice daily.

Lenabasum 5 mg

Drug

Subjects will receive lenabasum 5 mg twice daily.

Placebo

Other

Subjects will receive placebo twice daily.

Primary outcomes

  1. Pulmonary Exacerbation (PEx) Rate Over 28 Weeks

    Time frame: 28 weeks (Baseline Day 0 to Week 28)

    Rate of PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo, during the treatment period. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx.

Secondary outcomes

  1. Pulmonary Exacerbation (PEx) Rate

    Time frame: 28 weeks (Baseline Day 0 to Week 28)

    Event rate of PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx. The PEx rate is calculated as the number of PEx/28 weeks

  2. Time to First New Pulmonary Exacerbation (PEx)

    Time frame: 28 weeks (Baseline Day 0 to Week 28)

    Time to first new PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx. The rate is calculate over a 28 week period from visit 1 to week 28 visit

  3. Pulmonary Exacerbation (PEx)

    Time frame: 28 weeks (Baseline Day 0 to Week 28)

    Time to first PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx.

  4. CFQ-R Respiratory Symptom Domain

    Time frame: 28 weeks (Change from Baseline Day 0 to Week 28)

    Cystic Fibrosis Questionnaire - Revised measures change from baseline in CFQ-R respiratory symptom domain with lenabasum compared to placebo. Subjects >/= 14 years of age. 5 distinct 4-point Likert scales (e.g., always/often/ sometime/never) Scores for each HRQoL domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health.

  5. FEV1 % Predicted

    Time frame: 28 weeks (Change from Baseline Day 0 to Week 28)

    Change from baseline to week 28 in Forced Expiratory Volume in 1 second (FEV1) expressed as a percentage of a normal range. A lower percentage FEV1 is indicative of decrease in lung functionality. The changes observed from baseline to week 28 for lenabasum will be compared with those observed for placebo treated participants.

Sponsors and collaborators

Lead sponsor

Corbus Pharmaceuticals Inc.

Industry

Collaborators

  • Cystic Fibrosis Foundation

Registry information

Official study title

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

Important dates

Study start
2017
Primary completion
2020
Study completion
2020
First posted
Mar 1, 2018
Registry last updated
Jan 18, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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