Clinical Trials Register

Summary
EudraCT Number:	2017-003723-29
Sponsor's Protocol Code Number:	JBT101-CF-002
National Competent Authority:	Romania - National Agency for Medicines and Medical Devices
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2022-05-24
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Romania - National Agency for Medicines and Medical Devices

A.2

EudraCT number

2017-003723-29

A.3

Full title of the trial

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

Studiu de Fază 2, Contolat cu Placebo, Multicentric, Randomizat, Dublu-Orb, de Evaluare a Eficacității și Siguranței Lenabasum în Fibroza Chistică

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A study of safety and efficacy of lenabasum in Cystic Fibrosis

A.3.2

Name or abbreviated title of the trial where available

A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis

A.4.1

Sponsor's protocol code number

JBT101-CF-002

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Corbus Pharmaceuticals, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Corbus Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Accelsiors CRO and Consultancy Services Ltd
B.5.2	Functional name of contact point	Regulatory Unit
B.5.3	Address:
B.5.3.1	Street Address	50 Miskolci Str
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1147
B.5.3.4	Country	Hungary
B.5.4	Telephone number	+361299 00 91
B.5.5	Fax number	+361299 00 96
B.5.6	E-mail	regulatory@accelsiors.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/16/1736
D.3 Description of the IMP
D.3.1	Product name	Lenabasum
D.3.2	Product code	JBT-101
D.3.4	Pharmaceutical form	Capsule
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Lenabasum
D.3.9.1	CAS number	137945-48-3
D.3.9.2	Current sponsor code	JBT-101
D.3.9.3	Other descriptive name	resunab, ajulemic acid, anabasum
D.3.9.4	EV Substance Code	SUB178399
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/16/1736
D.3 Description of the IMP
D.3.1	Product name	Lenabasum
D.3.2	Product code	JBT-101
D.3.4	Pharmaceutical form	Capsule
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Lenabasum
D.3.9.1	CAS number	137945-48-3
D.3.9.2	Current sponsor code	JBT-101
D.3.9.3	Other descriptive name	resunab, ajulemic acid, anabasum
D.3.9.4	EV Substance Code	SUB178399
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Cystic Fibrosis (CF)

E.1.1.1

Medical condition in easily understood language

Cystic Fibrosis is a genetic disorder which results in thick mucus formation on the airways leading to increased lung infections,fibrosis of the lungs and digestive tract and abnormal immune function.

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10011762
E.1.2	Term	Cystic fibrosis
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the efficacy of lenabasum 20 mg twice per day (BID) compared to placebo in the treatment of cystic fibrosis (CF) by assessing the rate of pulmonary exacerbations (PEx) using primary definition of PEx

E.2.2

Secondary objectives of the trial

Efficacy:
1. To evaluate the efficacy of lenabasum 20 mg BID compared to placebo in the treatment of CF by assessing other efficacy endpoints
2. To evaluate the efficacy of lenabasum 5 mg BID compared to placebo in the treatment of CF
Pharmacokinetic:
1. To evaluate steady state plasma concentrations of
lenabasum 20 mg and 5 mg BID at the estimated time of trough
concentration after a dose of lenabasum
2. To evaluate plasma concentrations of lenabasum 20 mg and 5 mg at
the estimated time of peak concentration after the first dose
3. To evaluate metabolites of lenabasum
4. To develop population pharmacokinetic models of lenabasum
exposure in CF subjects
Safety:
1. To evaluate safety of lenabasum 20 mg BID and lenabasum 5 mg BID
treatment and placebo treatment
2. To evaluate tolerability of lenabasum 20 mg BID and lenabasum 5 mg BID treatment

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Documentation of a CF diagnosis as evidenced by 1 or more clinical features consistent with the CF phenotype and 1 or more of the following criteria:
a. Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test
b. Two known disease-causing mutations in the CFTR gene.
2. Twelve years of age or older at the time Informed Consent/Assent is signed.
3. Weight ≥ 40 kg.
4. FEV1 ≥ 40% predicted and < 100 % predicted within the last 12 months.
5. Physician-initiated treatment with an IV antibiotic 2 or 3 times in the last 12 months for a new PEx.
6. As an alternative to inclusion criterion 5, 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.
7. Completion of the last course of antibiotics prescribed for any PEx ≥ 28 days before Visit 1.
8. Able to perform pulmonary function tests. Optional use of a bronchodilator before testing is allowed to facilitate testing if the bronchodilator is used consistently starting with Visit 1.
9. Willing to provide repeat sputum specimens. If a subject is unable to reliably spontaneously expectorate sputum, induced sputum collection is acceptable. Optional collection of induced sputum specimens is allowed if induced sputum specimens are consistently collected starting with Visit 1. Adolescents should try to produce sputum spontaneously and can opt out of sputum induction.
10. Willing not to use any cannabinoids or any illegal substance of abuse from screening through Visit 9.
11. Women of childbearing potential must not be pregnant or breastfeeding at Visit 1 and must be using at least one highly effective method of contraception (failure rate < 1% per year) for at least 28 days before Visit 1 and be willing to continue to use at least one highly effective method of contraception throughout the study and for at least 28 days after discontinuation of study drug .
12. Able to adhere to the study visit schedule and other protocol requirements.

E.4

Principal exclusion criteria

1. Severe or unstable CF at screening or Visit 1, such as:
a. Change in dose, or initiation of any new chronic therapy for CF lung disease within 28 days before Visit 1
b. Treatment with any systemic corticosteroids > 10 mg per day prednisone or equivalent within 14 days before Visit 1
c. Actively listed on an organ transplant list or have had an organ transplant other than corneal transplant.
2. Significant diseases or conditions other than CF that may influence response to the study drug or safety, such as:
a. Active hepatitis B or C infection
b. Human immunodeficiency virus infection
c. A history of cancer except basal cell carcinoma or in situ carcinoma of the cervix treated with apparent success with curative therapy ≥ one year before Visit 1.
3. Pregnant, trying to become pregnant or lactating female.
4. Current evidence of alcohol abuse (defined as 4 or more drinks per day on at least 4 days of the week) or history of abuse of illegal and/or legally prescribed drugs such as barbiturates, benzodiazepines, amphetamines, cocaine, or opioids during the 1 year before screening.
5. Any investigational agent within 30 days or five therapeutic half-lives of that agent whichever is longer, before Visit 1
6. Any of the following values for laboratory tests at screening:
a. A positive pregnancy test
b. Hemoglobin < 10 g/dL in males and < 9 g/dL in females.
c. Neutrophils < 1.0 x 1000,000,000/L
d. Platelets < 75 x 1000,000,000/L
e. Creatinine clearance < 50 ml/min according to Modification of Diet in Renal Disease (MDRD) Study equation
f. Serum transaminases > 2.5 x upper normal limit
7. Any other condition or concurrent medical therapy at screening or Visit 1 that causes the investigator to determine it is not safe for the subject to participate or that may influence response to study drug or interfere with study assessments.

E.5 End points

E.5.1

Primary end point(s)

Rate of PEx using primary definition of PEx with lenabasum 20 mg BID, compared to placebo, during the treatment period

E.5.1.1

Timepoint(s) of evaluation of this end point

Visit 1 through study completion, up to 6 months

E.5.2

Secondary end point(s)

Efficacy (lenabasum 20 mg BID):
a. Event rate of PEx using secondary definition of PEx with lenabasum 20 mg BID compared to placebo
b. Time to first new PEx using primary definition of PEx with lenabasum 20 mg BID compared to placebo
c. Time to first PEx using secondary definition of PEx with lenabasum 20 mg BID compared to placebo
d. Change from baseline in CFQ-R respiratory symptom domain with lenabasum 20 mg BID compared to placebo
e. Change from baseline in FEV1 % predicted with lenabasum 20 mg BID compared to placebo
Efficacy (lenabasum 5 mg BID):
a. Rate of pulmonary exacerbations (PEx) using primary definition of PEx with lenabasum 5 mg BID compared to placebo, during the treatment period
b. Event rate of PEx using secondary definition of PEx with lenabasum 5 mg BID compared to placebo
c. Time to first new PEx using primary definition of PEx with lenabasum 5 mg BID compared to placebo
d. Time to first PEx using secondary definition of PEx with lenabasum 5 mg BID compared to placebo
e. Change from baseline in CFQ-R respiratory symptom domain with lenabasum 5 mg BID compared to placebo
f. Change from baseline in FEV1 % predicted with lenabasum 5 mg BID compared to placebo
Pharmacokinetics:
1. Estimated trough plasma concentrations of lenabasum
2. Estimated maximum plasma concentration (Cmax) of lenabasum
3. Metabolites of lenabasum
Safety:
1. Treatment emergent adverse events (TEAEs)
2. Changes in vital signs, physical examination, blood and urine
laboratory safety tests and electrocardiograms
3. Treatment discontinuations with lenabasum treatments compared to
placebo

E.5.2.1

Timepoint(s) of evaluation of this end point

Efficacy and Safety endpoints: Visit 1 through study completion, up to 6 months;
PK endpoints: Visit 1 through Visit 8.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

Réunion

United States

Austria

France

Poland

Bulgaria

Netherlands

Romania

Spain

Czechia

Germany

Greece

Italy

Belgium

Hungary

Russian Federation

Serbia

Slovakia

United Kingdom

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

353

F.1.3

Elderly (>=65 years)

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

300

F.4.2.2

In the whole clinical trial

415

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

After the trial, subjects will continue to receive care for cystic fibrosis from their treating physician. Subjects will remain on their baseline treatment for cystic fibrosis during the trial to reduce the risk of disease flare where study product is discontinued.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	European Cystic Fibrosis Society (ECFS) - Clinical Trial Network
G.4.3.4	Network Country	Belgium

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-06-18

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-11-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-06-17

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