Clinical Trials Register

Summary
EudraCT Number:	2014-005544-18
Sponsor's Protocol Code Number:	PB-102-F03
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2015-02-16
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

Spain - AEMPS

A.2

EudraCT number

2014-005544-18

A.3

Full title of the trial

A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients

Estudio de extensión multicéntrico de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 24 meses a pacientes adultos con enfermedad de Fabry

A.3.1

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

Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients

Estudio de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 24 meses a pacientes adultos con enfermedad de Fabry

A.4.1

Sponsor's protocol code number

PB-102-F03

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	Protalix Ltd.
B.1.3.4	Country	Israel
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	Protalix Ltd.
B.4.2	Country	Israel
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	SERMES PLANIFICACION S.L
B.5.2	Functional name of contact point	Unidad de Puesta en Marcha
B.5.3	Address:
B.5.3.1	Street Address	C/Rufino González 14, Esc.1ª-2ºD
B.5.3.2	Town/ city	Madrid
B.5.3.3	Post code	28027
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34913275025
B.5.5	Fax number	+34917542721
B.5.6	E-mail	start-up@sermescro.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	PRX102
D.3.2	Product code	PRX102
D.3.4	Pharmaceutical form	Infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PRX102
D.3.9.1	CAS number	1333358-30-7
D.3.9.2	Current sponsor code	PRX102
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	PRX102
D.3.2	Product code	PRX102
D.3.4	Pharmaceutical form	Infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PRX102
D.3.9.1	CAS number	1333358-30-7
D.3.9.2	Current sponsor code	PRX102
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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: 3
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	PRX102
D.3.2	Product code	PRX102
D.3.4	Pharmaceutical form	Infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PRX102
D.3.9.1	CAS number	1333358-30-7
D.3.9.2	Current sponsor code	PRX102
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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

Fabry disease

Enfermedad de Fabry

E.1.1.1

Medical condition in easily understood language

Fabry disease is an inherited condition caused by a deficiency of an enzyme, leading to a range of systemic symptoms.

La enfermedad de Fabry es un trastorno genético heredado provocado por la deficiencia de una enzima que conduce a una amplia gama de síntomas.

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	18.0
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 evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02- and are continuing to receive treatment at the dose each patient reeceived in study PB-102-F02.

Evaluar los parámetros de seguridad, tolerabilidad y eficacia de PRX-102 en pacientes adultos con enfermedad de Fabry que completaron de manera exitosa el tratamiento con PRX-102 en los estudios PB-102-F01 y PB-102-F02 y continúan recibiendo tratamiento con la dosis que cada paciente recibió en el estudio PB-102-F02.

E.2.2

Secondary objectives of the trial

Not applicable

No aplica

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Completion of study PB-102-F02
2. The patient signs informed consent
3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 1 month after termination of treatment.

1. Que se haya completado el estudio PB-102-F02
2. Que el paciente firme el consentimiento informado
3. Que las pacientes y los pacientes cuyas parejas tengan capacidad para concebir acepten usar un método anticonceptivo aceptable desde el punto de vista médico; se excluye el método del ritmo. Los métodos anticonceptivos aceptables incluyen productos hormonales, dispositivos intrauterinos o condones masculinos o femeninos. Deben utilizarse durante 1 mes después de la finalización del tratamiento.

E.4

Principal exclusion criteria

1. Pregnant or nursing
2. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient’s compliance with the requirements of the study

1. Embarazo o lactancia
2. Presencia de cualquier afección médica, emocional, conductual o psicológica que, según el criterio del Investigador y/o Director Médico, interfiera en el cumplimiento de los requisitos del estudio por parte del paciente

E.5 End points

E.5.1

Primary end point(s)

Efficacy Variables
The efficacy endpoints evaluated in Study PB-102-F02 will continue to be measured in this extension study. Baseline values for this extension study will be the values from the last infusion of study PB-102-F02.
Safety Variables: Safety will be assessed by the frequency, severity, and duration of treatment-emergent adverse events (TEAEs), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings, assessment of the injection site reactions and existence of Anti PRX-102 antibodies after administration of the study drug.

Variables de eficacia
Los criterios de valoración de la eficacia evaluados en el estudio PB-102-F02 se continuarán midiendo en este estudio de extensión. Los valores basales para este estudio de extensión serán los valores de la última infusión del estudio PB-102-F02.
Variables de seguridad: La seguridad se evaluará mediante la frecuencia, la gravedad y la duración de los eventos adversos emergentes del tratamiento (EAET), incluidas las anomalías de laboratorio significativas desde el punto de vista clínico, los cambios en el ECG respecto del nivel basal, los hallazgos del examen físico, la evaluación del lugar de la inyección y la existencia de anticuerpos contra PRX-102 después de la administración del fármaco del estudio.

E.5.1.1

Timepoint(s) of evaluation of this end point

Efficacy Variables
Gb3 and Lyso-Gb3 concentrations in plasma: baseline/every 3 months
Gastrointestinal symptoms, Kidney functions, BPI: baseline/every 3 months
Gb3 concentration in kidney and in skin: last treatment visit
Left ventricular mass and myocardial fibrosis: baseline/ every 12 months
Cardiac function and stress test: baseline/every 12 months
Cerebrovascular disease: baseline/last treatment of study
MSSI: baseline/every 6 months
Safety Variables
Laboratory values and physical examination: screening and visits 7, 14, 20, 27, 33, 40, 46, 53
ECG: screening and visits 14, 40, 53
Anti PRX-102 antibodies: screening and visits 5, 7, 14, 20, 27, 33, 40,46,53 and 3 months after last infusion
AE: every study visit
Injection site reactions: Every study treatment visit

Variables de eficacia
Concentraciones de Gb3 y Liso-Gb3 en plasma: nivel basal/cada 3 meses
Sínt. gastrointestinales, Fun. renales, BPI: nivel basal/cada 3 meses
Concentración Gb3 en riñón y piel: última visita del trat.
LVM y fibrosis miocárdica: nivel basal/cada 12 meses
Función cardíaca y prueba de esfuerzo: nivel basal/cada 12 meses
Trastorno cerebrovascular: nivel basal y última visita del trat.
MSSI: nivel basal/cada 6 meses
Variables de seguridad
Valores de lab. Y examen físico: screening y visitas 7, 14, 20, 27, 33, 40, 46, 53
ECG: screening y visitas 14, 40, 53
Anticuerpos PRX-102: screening y visitas 5, 7, 14, 20, 27, 33, 40,46,53 y 3 meses después de la última infusión
AE: todas las visitas
Reacciones lugar de la inyección: Todas las visitas de trat.

E.5.2

Secondary end point(s)

Not applicable

No aplica

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

No aplica

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

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)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

Extension of the study PB-102-F01 and PB-102-F02

Extensión del estudio PB-102-F01 y PB-102-F02

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

Paraguay

Spain

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

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:

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 tolerate the infusions well and complete the study are eligible to enroll in the next extension study.

Los pacientes que toleren bien las dosis y completen este estudio, se les dará la opción de participar en la siguiente extensión del estudio.

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

2015-04-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-04-15

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-08-26

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