Clinical Trials Register

Summary
EudraCT Number:	2018-003273-10
Sponsor's Protocol Code Number:	JBT101-DM-002
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	GB - no longer in EU/EEA
Date on which this record was first entered in the EudraCT database:	2018-11-20
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

UK - MHRA

A.2

EudraCT number

2018-003273-10

A.3

Full title of the trial

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis

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 dermatomyositis patients

A.3.2

Name or abbreviated title of the trial where available

A Phase 3 safety and efficacy study of lenabasum in dermatomyositis subjects

A.4.1

Sponsor's protocol code number

JBT101-DM-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	103 Háros Str
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1222
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/3/18/2070
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	SUB193005
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/3/18/2070
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	SUB193005
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

Dermatomyositis (DM)

E.1.1.1

Medical condition in easily understood language

Dermatomyositis is a rare and serious autoimmune disease.An over-active immune response causes chronic inflammation,which results in growth of scar tissue in the skin,muscles,and many internal organs.

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

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	10012503
E.1.2	Term	Dermatomyositis
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue 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 compared to placebo in subjects with dermatomyositis (DM) as measured by Total Improvement Score (TIS)

E.2.2

Secondary objectives of the trial

Secondary Objectives:
1. To evaluate the efficacy of lenabasum compared to placebo in subjects with DM, as measured by secondary endpoints;
2. To evaluate the safety of lenabasum in subjects with DM;
3. To evaluate the tolerability of lenabasum in subjects with DM.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Able to understand and voluntarily sign the informed consent
2. Male or female ≥ 18 years of age at the time of signing the informed consent
3. Fulfill one of the following criteria for DM:
a. Bohan and Peter’s criteria
b. ACR/EULAR criteria
4. Subject has active DM as determined by the investigator
5. Disease activity/severity fulfils one of the following three criteria:
i. MDGA ≥ 3 cm (0-10 cm scale) and MMT-8 score ≤ 142 (out of 150 total possible)
ii. Sum of MDGA, PtGA and EMGA VAS scores is ≥ 10 cm (all scales individually on 0-10 cm scale)
iii. MDGA ≥ 3 cm and CDASI activity score of >14
6. Stable doses of immunosuppressive medications for DM as defined by:
a. Unchanged dose of oral corticosteroids ≤ 20 mg per day prednisone or equivalent for ≥ 4 weeks before Visit 1
b. Unchanged dose of immunosuppressive medications other than oral corticosteroids for ≥ 8 weeks before Screening
7. Willing to not start or stop any immunosuppressive medications for DM from Screening through end of study, unless a change is part of the protocol or considered in the subject’s best medical interest by the site investigator or another physician who has primary responsibility for treating the subject’s DM.
8. Willing to not use any cannabinoids, including recreational marijuana, medical marijuana and other prescription cannabinoids from Screening through end of study.
9. Able to adhere to the study visit schedule and other protocol requirements and follow study restrictions.
10. Women of childbearing potential (WOCBP) must not be pregnant or breastfeeding and they or their male sexual partner must be using at least one acceptable method of contraception (Appendix 1) for at least 4 weeks before Visit 1 and be willing to continue its use for at least 4 weeks after discontinuation of study product.
11. Male subjects must be willing to follow acceptable contraceptive requirements (Appendix 1) and should not get anyone pregnant while they are taking the study product or within 4 weeks after taking the last dose of the study product, during which time period they or their female sexual partner must be willing to use at least one recommended method of contraception.

E.4

Principal exclusion criteria

1. Unstable DM or DM with end-stage organ involvement at Screening or Visit 1, including:
a. On an organ transplantation list or has received an organ transplant, except corneal transplant
b. Interstitial lung disease requiring constant oxygen therapy. This excludes oxygen used to aid sleep or exercise
c. Pulmonary hypertension requiring constant oxygen therapy. This excludes oxygen used to aid sleep or exercise
d. Subjects requiring supplemental tube feeding or parenteral nutrition
2. Certain medications at Visit 1, including:
a. Treatment with intravenous corticosteroids within 4 weeks before Visit 1 (Note: treatment with intra-articular corticosteroids within 4 weeks before Visit 1 is allowed)
b. Treatment with oral or intravenous antibiotics or antiviral treatments for new bacterial or viral infections within 4 weeks of Visit 1. This does not include prophylactic antibiotics or antiviral treatments
c. Any investigational agent within 30 days or 5 therapeutic half-lives of that agent whichever is longer, before Visit 1
3. Significant diseases or conditions other than DM that may influence response to the study product or safety, such as:
a. Acute or chronic hepatitis B or C infection (HBsAg or HCV RNA positivity)
b. Human immunodeficiency virus infection
c. History of active tuberculosis or positive tuberculosis test without a completed course of appropriate treatment or having already completed at least 1 month of ongoing appropriate treatment
d. Evidence of cancer (except for treated basal or squamous cell carcinoma of the skin or cervical carcinoma in situ) within 3 years of Visit 1
Note: Overlap with features of SSc, systemic lupus erythematosus, Sjogren’s syndrome, or rheumatoid arthritis is allowed if the dominant clinical disease is DM.
4. Any of the following values for laboratory tests at Screening:
a. A positive pregnancy test in women of child-bearing potential (WOCBP) - also at Visit 1
b. Hemoglobin < 9 g/dL in males and < 8 g/dL in females
c. Neutrophils < 1.0 × 10^9/L
d. Platelets < 75 × 10^9/L
e. Creatinine clearance < 50 mL/min on screening blood test, per the Modification of Diet in Renal Disease Study or in 24 hour urine creatine clearance measurement
5. Any known hypersensitivity to lenabasum.
6. Any medical, psychiatric or substance abuse condition, concurrent medical therapies, or abnormal laboratory values that in the opinion of the site investigator may put the subject at greater safety risk, influence response to study drug or interfere with study assessments.

E.5 End points

E.5.1

Primary end point(s)

The Total Improvement Score (TIS) by the 2016 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) Myositis Response Criteria compared to placebo at Week 52
TIS score is comprised of 6 core set measures:
1. Physician global activity (MDGA)
2. Patient global activity (PtGA)
3. Manual Muscle Testing (MMT)
4. Health Assessment Questionnaire (HAQ)
5. Muscle enzymes
6. Extramuscular global activity (EMGA)

E.5.1.1

Timepoint(s) of evaluation of this end point

Muscle Enzymes: from Baseline through Week 52;
For others: Day 1, Week 4, Week 16, Week 28, Week 40, Week 52

E.5.2

Secondary end point(s)

1. Change from Baseline compared to placebo at Week 52 in:
1. Mean MMT-8 score
2. Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score
3. Proportion of subjects who achieve clear or almost clear skin on the Investigator Global Assessment (IGA) scale of skin activity
4. Short Form – 36 (SF-36) physical functioning domain score
5. Corticosteroid dose
6. Forced Vital Capacity (FVC) % predicted
2. Adverse events (AEs) and changes in: vital signs, physical examinations, blood and urine laboratory safety tests, and ECGs associated with lenabasum treatment.
3. Number of subjects who permanently discontinue study product due to AEs probably- or definitely-related to treatment.

E.5.2.1

Timepoint(s) of evaluation of this end point

MMT-8 score, CDASI, IGA, SF-36: From Baseline through Day 1, Week 4, Week 16, Week 28, Week 40, Week 52.
Concomitant medications (Corticosteroid dose), Safety monitoring, blood lab safety testing: From Baseline through Week 52.
Spiromarty (FVC%): From Baseline through Day 1, Week 4, Week 16, Week 28, Week 52.
ECGs: From Baseline through Day 1, Week 4, Week 28, Week 10.
Urine dipstick: Baseline through Day 1, Week 4, Week 28, Week 52.

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

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

Bulgaria

Canada

Czech Republic

Germany

Hungary

Italy

Japan

Korea, Republic of

Poland

Romania

Serbia

Spain

Sweden

Switzerland

United Kingdom

United States

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

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

130

F.1.3

Elderly (>=65 years)

Yes

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

F.4.2.2

In the whole clinical trial

150

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 DM from their treating physician. Subjects will remain on their baseline treatment for DM during the trial to reduce the risk of disease flare where study product is discontinued. It is the intent of the Sponsor to offer participation in a separate open-label trial with lenabasum for subjects who complete this trial through to visit 11 (Day 365/Week 52).

G. Investigator Networks to be involved in the Trial

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-01-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-05-21

P. End of Trial
P.	End of Trial Status	GB - no longer in EU/EEA

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