Clinical Trials Register

Summary
EudraCT Number:	2017-003369-85
Sponsor's Protocol Code Number:	ID-069A301
National Competent Authority:	Austria - BASG
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-04-27
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

Austria - BASG

A.2

EudraCT number

2017-003369-85

A.3

Full title of the trial

A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

A.3.1

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

A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease

A.3.2

Name or abbreviated title of the trial where available

MODIFY

A.4.1

Sponsor's protocol code number

ID-069A301

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	Idorsia Pharmaceuticals Ltd
B.1.3.4	Country	Switzerland
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	Idorsia Pharmaceuticals Ltd
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Idorsia Pharmaceuticals Ltd
B.5.2	Functional name of contact point	Clinical Trial Disclosure Desk
B.5.3	Address:
B.5.3.1	Street Address	Hegenheimermattweg 91
B.5.3.2	Town/ city	Allschwil
B.5.3.3	Post code	4123
B.5.3.4	Country	Switzerland
B.5.6	E-mail	clinical-trials-disclosure@idorsia.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/3/12/1033; EMA/OD/042/12
D.3 Description of the IMP
D.3.1	Product name	Lucerastat
D.3.2	Product code	ACT-434964
D.3.4	Pharmaceutical form	Capsule, hard
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	Lucerastat
D.3.9.2	Current sponsor code	ACT-434964
D.3.9.3	Other descriptive name	OGT923
D.3.9.4	EV Substance Code	SUB189593
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	250
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, hard
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

Fabry disease

E.1.1.1

Medical condition in easily understood language

Fabry disease is a rare, inherited disease caused by a genetic defect which leads to an accumulation of fatty substance in the body. Symptoms include stomach pain and damage to vital organs.

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

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	10016016
E.1.2	Term	Fabry's disease
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 determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).

E.2.2

Secondary objectives of the trial

• To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (abdominal pain and diarrhea) in subjects with FD and GI symptom(s) at baseline.
• To confirm the effect of lucerastat on biomarkers of FD.
• To determine the safety and tolerability of lucerastat in subjects with FD.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

PK profile sub-study (all sites): A sub-study will be conducted at all sites to determine the PK profile of lucerastat at steady state in a subgroup of approximately 36 subjects with FD with either normal or subnormal renal function (i.e., screening eGFR ≥ 60 mL/min/1.73 m2) or impaired renal function (i.e., screening eGFR < 60 mL/min/1.73 m2). If necessary, blood samples collected for PK may be used for the identification of potential metabolites of lucerastat in this population. Subjects consenting to this sub study will have a 12-hour PK profile performed at the Month 1 visit.

E.3

Principal inclusion criteria

1. Signed and dated ICF prior to any study-mandated procedure.
2. Male or female subjects; 18-years old and above.
3. FD diagnosis confirmed with local genetic test results (i.e., presence
of at least 1 mutation in GLA, the gene coding for α-
galactosidase A).
4. Fabry-associated neuropathic pain, as defined by the subject, in the
last 3 months prior to screening.

E.4

Principal exclusion criteria

1. Pregnant, planning to be become pregnant up to 30 days after study
treatment discontinuation or lactating subject;
2. Severe renal insufficiency defined as an estimated glomerular
filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology
Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening;
3. Subject on regular dialysis for the treatment of chronic kidney
disease;
4. Subject has undergone, or is on a waiting list for, or is scheduled to
undergo kidney or other organ transplantation;
5. Known and documented transient ischemic attack, stroke, unstable
angina or myocardial infarction within 6 months prior to screening;
6. Clinically significant unstable cardiac disease in the opinion of the
investigator (e.g., uncontrolled symptomatic arrhythmia, New York
Heart Association class III or IV congestive heart failure);
7. Any other subject at high risk for developing clinical signs of organ
involvement within the time period of the study, as per investigator
judgment;
8. Subject planned for imminent initiation of treatment with ERT.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is change from baseline to Month 6 in the "modified" Brief Pain Inventory-Short Form item 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours".

E.5.1.1

Timepoint(s) of evaluation of this end point

From baseline to Month 6

E.5.2

Secondary end point(s)

• Change from baseline to Month 6 in the 11-point Numerical Rating Scale (NRS-11) score of “abdominal pain at its worst in the last 24 hours” in subjects with GI symptoms at baseline.
• Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline.
• Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).

E.5.2.1

Timepoint(s) of evaluation of this end point

From baseline to Month 6

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

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

Yes

E.6.13.1

Other scope of the trial description

tolerability

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

Yes

E.8.4

The trial involves multiple sites in the 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

Austria

Belgium

Canada

Germany

Ireland

Italy

Netherlands

Norway

Poland

Spain

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:
If the last subject enters the OLE study, the end of the trial corresponds to the EOT visit. If the last subject does not enter the OLE study, the end of the trial corresponds to the FU1 visit, FU2 visit, or to the last visit of the PTOP, whichever is last.

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:

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

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)

Subjects who completed the Month 6 visit will be proposed to enroll in a separate OLE study.

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

2018-05-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-06-29

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-09-02

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