ICH GCP - EU Clinical trials Registry

Summary
EudraCT Number:	2017-003757-41
Sponsor's Protocol Code Number:	CCP-020-302
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-11-13
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

A. Protocol Information

A.1

Member State Concerned

UK - MHRA

A.2

EudraCT number

2017-003757-41

A.3

Full title of the trial

An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)

A.3.1

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

An International Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)

A.4.1

Sponsor's protocol code number

CCP-020-302

A.5.4

Other Identifiers

Name:

IND

Number:

131 384

A.7

Trial is part of a Paediatric Investigation Plan

No

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Castle Creek Pharmaceuticals, LLC
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	Castle Creek Pharmaceuticals, LLC
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	Medpace Germany GmbH
B.5.2	Functional name of contact point	Vasiliki Iassonidou
B.5.3	Address:
B.5.3.1	Street Address	Theresienhöhe 30
B.5.3.2	Town/ city	München
B.5.3.3	Post code	80339
B.5.3.4	Country	Germany
B.5.4	Telephone number	+498989 55 718 99 ex24960
B.5.5	Fax number	+442033183827
B.5.6	E-mail	V.Iassonidou@medpace.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/3/14/1236
D.3 Description of the IMP
D.3.1	Product name	Diacerein
D.3.2	Product code	Diacerein
D.3.4	Pharmaceutical form	Ointment
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Topical use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DIACEREIN
D.3.9.1	CAS number	13739-02-1
D.3.9.2	Current sponsor code	CCP-020
D.3.9.3	Other descriptive name	Diacerein
D.3.9.4	EV Substance Code	SUB07060MIG
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/W) percent weight/weight
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Epidermolysis Bullosa Simplex (EBS)

E.1.1.1

Medical condition in easily understood language

Epidermolysis Bullosa Simplex (EBS)

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	10014989
E.1.2	Term	Epidermolysis bullosa
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

The primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101.

E.2.2

Secondary objectives of the trial

None

E.2.3

Trial contains a sub-study

No

E.3

Principal inclusion criteria

1. In the opinion of the Investigator, the subject is capable of understanding and complying with protocol requirements.
2. The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
3. Subject has a documented genetic mutation consistent with EBS.
4. Subject who participated in the CCP-020-301 or the CCP-020-101 study are eligible to be rolled into the CCP-020-302 open label extension study, regardless of their completion status on the feeder study.
5. Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions (e.g. medicated cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.).
6. If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, as defined by this protocol, for the duration of the study.
7. Subject is non-lactating and is not planning for pregnancy during the study period.
8. Subject is willing and able to follow all study instructions and to attend all study visits.

E.4

Principal exclusion criteria

1. Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require topical antibiotic therapy to treat an infection).
2. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline.
3. The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication.
4. Subject has experienced a change in clinical status from the feeder study that, in the investigator's opinion, puts the subject at undue risk to participate.

E.5 End points

E.5.1

Primary end point(s)

Safety will be evaluated in terms of the occurrence of AEs and changes in clinical laboratory parameters, clinical examination findings, vital signs, weight, and urine measurements.

E.5.1.1

Timepoint(s) of evaluation of this end point

For each cycle: start day; start day + 8 weeks; start day + 16 weeks

E.5.2

Secondary end point(s)

none

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

No

E.6.2

Prophylaxis

No

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

No

E.6.6

Pharmacokinetic

No

E.6.7

Pharmacodynamic

No

E.6.8

Bioequivalence

No

E.6.9

Dose response

No

E.6.10

Pharmacogenetic

No

E.6.11

Pharmacogenomic

No

E.6.12

Pharmacoeconomic

No

E.6.13

Others

No

E.6.13.1

Other scope of the trial description

Not applicable

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

No

E.7.1.1

First administration to humans

No

E.7.1.2

Bioequivalence study

No

E.7.1.3

Other

No

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

No

E.7.4

Therapeutic use (Phase IV)

No

E.8 Design of the trial

E.8.1

Controlled

No

E.8.1.1

Randomised

No

E.8.1.2

Open

Yes

E.8.1.3

Single blind

No

E.8.1.4

Double blind

No

E.8.1.5

Parallel group

No

E.8.1.6

Cross over

No

E.8.1.7

Other

No

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

No

E.8.2.2

Placebo

No

E.8.2.3

Other

No

E.8.3

The trial involves single site in the Member State concerned

Information not present in EudraCT

E.8.4

The trial involves multiple sites in the Member State concerned

Information not present in EudraCT

E.8.4.1

Number of sites anticipated in Member State concerned

2

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

9

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

No

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

Austria

France

Germany

Israel

Italy

Netherlands

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

No

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

LPLV

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

2

E.8.9.1

In the Member State concerned months

0

E.8.9.1

In the Member State concerned days

0

E.8.9.2

In all countries concerned by the trial years

2

E.8.9.2

In all countries concerned by the trial months

0

E.8.9.2

In all countries concerned by the trial days

0

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:

44

F.1.1.1

In Utero

No

F.1.1.2

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

No

F.1.1.3

Newborns (0-27 days)

No

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

4

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

20

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

20

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

20

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

20

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

No

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

No

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

No

F.3.3.4

Nursing women

No

F.3.3.5

Emergency situation

No

F.3.3.6

Subjects incapable of giving consent personally

No

F.3.3.7

Others

No

F.4 Planned number of subjects to be included

F.4.1

In the member state

8

F.4.2

For a multinational trial

F.4.2.1

In the EEA

30

F.4.2.2

In the whole clinical trial

80

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)

Standard of Care

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1

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

N.

Competent Authority Decision

Authorised

N.

Date of Competent Authority Decision

2017-12-29

N.

Ethics Committee Opinion of the trial application

Favourable

N.

Ethics Committee Opinion: Reason(s) for unfavourable opinion

N.

Date of Ethics Committee Opinion

2018-02-19

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-04-17

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