Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

• Sponsor: Corbus Pharmaceuticals Inc.
• Collaborator: Cystic Fibrosis Foundation
• Information provided by (Responsible Party): Corbus Pharmaceuticals Inc.

Study Description

Brief Summary:

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, Europe, and Australia. 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.

Condition or disease	Intervention/treatment	Phase
Cystic Fibrosis	Drug: Lenabasum 20 mg; Drug: Lenabasum 5 mg; Other: Placebo	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	415 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Intervention Model Description:	Multicenter, double-blind, randomized, placebo-controlled, parallel group study.
Masking:	Double (Participant, Investigator)
Primary Purpose:	Treatment
Official Title:	A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
Actual Study Start Date :	December 22, 2017
Estimated Primary Completion Date :	September 2019
Estimated Study Completion Date :	November 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Lenabasum 20 mg BID	Drug: Lenabasum 20 mg; Subjects will receive lenabasum 20 mg twice daily.
Experimental: Lenabasum 5 mg BID	Drug: Lenabasum 5 mg; Subjects will receive lenabasum 5 mg twice daily.
Placebo Comparator: Placebo BID	Other: Placebo; Subjects will receive placebo twice daily.

Outcome Measures

Primary Outcome Measures :

1. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Rate of PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo, during the treatment period.

Secondary Outcome Measures :

1. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Event rate of PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo.
2. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Time to first new PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo.
3. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Time to first PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo.
4. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Event rate of PEx using the primary PEx definition with lenabasum 5 mg BID compared to placebo, during the treatment period.
5. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Event rate of PEx using the secondary PEx definition with lenabasum 5 mg BID compared to placebo.
6. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Time to first new PEx using the primary PEx definition with lenabasum 5 mg BID compared to placebo.
7. Pulmonary Exacerbation (PEx) [ Time Frame: Visit 1 through Week 28. ]
   Time to first PEx using the secondary PEx definition with lenabasum 5 mg BID compared to placebo.
8. CFQ-R respiratory symptom domain [ Time Frame: Visit 1 through Week 28. ]
   Change from baseline in CFQ-R respiratory symptom domain with lenabasum compared to placebo.
9. FEV1 % predicted [ Time Frame: Visit 1 through Week 28. ]
   Change from baseline in FEV1 % predicted with lenabasum compared to placebo.
10. Adverse Events [ Time Frame: Visit 1 through Week 28. ]
    Incidence of treatment emergent adverse events with lenabasum treatment.

Eligibility Criteria

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Ages Eligible for Study:	12 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

1. ≥ 12 years of age at the time Informed Consent/ Assent is signed.
2. Weight ≥ 40 kg.
3. FEV1 ≥ 40% predicted and < 100% predicted in the last 12 months.
4. 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:

1. Severe or unstable CF at screening or Visit 1.

2. Any of the following values for laboratory tests at screening:

   1. A positive pregnancy test.
   2. Hemoglobin < 10 g/dL in males and < 9 g/dL in females.
   3. Neutrophils < 1.0 x 10^9 /L.
   4. Platelets < 75 x 10^9/L.
   5. Creatinine clearance < 50 mL/min according to Modification of Diet in Renal Disease (MDRD) Study equation.
   6. Serum transaminases > 2.5 x upper limit of normal.
3. 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.

Contacts and Locations

Contacts

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Contact: Lindsey Smith	617-977-5030	CFpatients@corbuspharma.com
Contact: Scott Constantine, MS	617-977-5030	CFpatients@corbuspharma.com

  Show 65 Study Locations

Sponsors and Collaborators

Corbus Pharmaceuticals Inc.

Cystic Fibrosis Foundation

Investigators

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Principal Investigator:	James Chmiel, MD	Rainbow Baby and Children's Hospital
Principal Investigator:	J. Stuart Elborn, MD	National Heart and Lung Institute, Imperial College

More Information

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Responsible Party:	Corbus Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:	NCT03451045 History of Changes
Other Study ID Numbers:	JBT101-CF-002
First Posted:	March 1, 2018
Last Update Posted:	March 13, 2019
Last Verified:	March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Undecided

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Studies a U.S. FDA-regulated Drug Product:		Yes
Studies a U.S. FDA-regulated Device Product:		No

Keywords provided by Corbus Pharmaceuticals Inc.:

Pulmonary exacerbation, lenabasum, JBT-101

Additional relevant MeSH terms:

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Fibrosis; Cystic Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases	Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases