Clinical Trials Register

Summary
EudraCT Number:	2016-000942-77
Sponsor's Protocol Code Number:	EFC14028
National Competent Authority:	Hungary - National Institute of Pharmacy
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-12-04
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

Hungary - National Institute of Pharmacy

A.2

EudraCT number

2016-000942-77

A.3

Full title of the trial

A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease

III. fázisú, randomizált, multicentrikus, nemzetközi, kettős vak vizsgálat a kéthetente ismételt neoGAA (GZ402666) és alfa-alglükozidáz infúzió hatásosságának és biztonságosságának összehasonlítására korábban nem kezelt, késői megjelenésű Pompe-kórban szenvedő betegeknél

A.3.1

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

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Vizsgálat a kéthetente ismételt adagolású neoGAA (GZ402666) és alfa-alglükozidáz infúzió hatásosságának és biztonságosságának összehasonlítására korábban nem kezelt, késői megjelenésű Pompe-kórban szenvedő betegekben

A.4.1

Sponsor's protocol code number

EFC14028

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1178-4806

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	Genzyme Corporation
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	Genzyme Corporation
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	Sanofi-Aventis Zrt.
B.5.2	Functional name of contact point	NA
B.5.3	Address:
B.5.3.1	Street Address	Tó u. 1-5.
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1045
B.5.3.4	Country	Hungary
B.5.6	E-mail	kapcsolat@sanofi.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/3/14/1251
D.3 Description of the IMP
D.3.1	Product name	neoGAA
D.3.2	Product code	GZ402666
D.3.4	Pharmaceutical form	Powder for concentrate for solution for 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.3.9.1	CAS number	1802558-87-7
D.3.9.2	Current sponsor code	GZ402666
D.3.9.3	Other descriptive name	Recombinant human α-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
D.3.9.4	EV Substance Code	SUB120294
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	100
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	Comparator
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	Yes
D.2.1.1.1	Trade name	MYOZYME®
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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/3/00/018
D.3 Description of the IMP
D.3.1	Product name	Myozyme®
D.3.4	Pharmaceutical form	Powder for concentrate for solution for 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	ALGLUCOSIDASE ALFA
D.3.9.1	CAS number	420784-05-0
D.3.9.4	EV Substance Code	SUB21275
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	50
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

Pompe disease (acid alpha-glucosidase deficiency)

Pompe-kór (savanyú alfa-glükozidáz enzim hiány)

E.1.1.1

Medical condition in easily understood language

Treatment of enzyme deficiency in glycogen storage disease type II (Pompe disease)

2-es típusú glikogén tárolási betegség (Pompe-kór) kezelése

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	LLT
E.1.2	Classification code	10036143
E.1.2	Term	Pompe's disease
E.1.2	System Organ Class	100000004850

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 neoGAA (GZ402666) treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.

A neoGAA-kezelés légzőizomerőre kifejtett hatásának meghatározása a függőleges testhelyzetben mért forszírozott vitálkapacitás (Forced Vital Capacity, FVC) %-os értékén keresztül, az alfa-alglükozidáz-kezeléssel összehasonlítva.

E.2.2

Secondary objectives of the trial

To determine the safety and effect of neoGAA treatment on functional endurance (6-minute walk test [6MWT], inspiratory muscle strength (maximum inspiratory pressure [MIP]), expiratory muscle strength (maximum expiratory pressure [MEP]), lower extremity muscle strength (hand-held dynamometry [HHD]), motor function (Quick Motor Function Test [QMFT]), and health-related quality of life (SF-12).

A neoGAA-kezelés biztonságosságának és a funkcionális terhelhetőségre (6 perces járásteszt [6-minute walk test, 6MWT]), légzőizomerőre (maximális belégzési nyomás [maximum inspiratory pressure, MIP]), kilégzési izomerőre (maximális kilégzési nyomás [maximum expiratory pressure, MEP]), alsó végtagi izomerőre (kézi dinanométeres mérés [hand-held dynamometry, HHD]), motoros funkcióra (gyors motoros funkciós teszt [Quick Motor Function Test, QMFT]) és az egészséggel összefüggő életminőségre (Short Form-12 kérdőív [SF-12]) kifejtett hatásának meghatározása.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

-The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
-The patient must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from patients, if applicable.

- A beteg bármely szövetforrásból kimutatott és/vagy 2 GAA génmutációval igazolt savas alfa-glükozidáz (GAA) enzimhiányban szenved.
- Aláírt beleegyezési nyilatkozat a betegtől, illetve adott esetben szülőtől, gondviselőtől.

E.4

Principal exclusion criteria

-The patient is <3 years of age.
-The patient has known Pompe specific cardiac hypertrophy.
-The patient is wheelchair dependent.
-The patient is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device.
-The patient requires invasive-ventilation (non-invasive ventilation is allowed).
-The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of ≥30% predicted and ≤85% predicted.
-The patient (and patient’s legal guardian if patient is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol.
-The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
-The patient has prior or current use of immune tolerance induction therapy
- The patient, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline.

- A beteg nem töltötte be a 3 éves életkort
- A beteg ismert Pompe-kór-specifikus cardialis hypertrophiában szenved
- A beteg A beteg nem képes 40 méter megtételére gyalogosan megállás és segédeszköz nélkül
- A beteg invazív lélegeztetésre szorul (nem invazív lélegeztetés megengedett)
- A beteg nem képes sikeresen végrehajtani a függőleges testhelyzetben végzett ismételt FVC méréseket ≥30%-a és ≤85%-a közötti várható eredménnyel,
- A beteg korábban immunmodulátor-, alfa-alglükozidáz, vagy bármilyen Pompe-kór elleni vizsgálati kezelésben részesült
- A beteg korábban vagy beválasztáskor immuntolerancia növelő kezelésben részesül
- A beteg pozitív terhességi teszteredménnyel rendelkezik a kiinduláskor

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in percent predicted forced vital capacity (%FVC) in upright position

A függőleges testhelyzetben mért várható FVC %-os változása a kiindulási időponttól.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline to 12 months

A kiindulási időponttól a 12. hónapig

E.5.2

Secondary end point(s)

- Change from baseline in six-minute walk test scores

- Change from baseline in maximal inspiratory pressure in upright position

- Change from baseline in maximal expiratory pressure in upright position

- Change from baseline in hand-held dynamometry measurement

- Change from baseline in Quick Motor Function Test scores

- Change from baseline in 12- Item Short-form health survey scores

- Number of participants with adverse events

- 6 perces járásteszt eredményének változása a kiindulási értéktől;
- maximális belégzési nyomás függőleges testhelyzetben mért értékének változása a kiindulási értéktől;
- kézi dinanométeres mérés eredményének változása a kiindulási értéktől;
- Motoros funkció teszt (Quick Motor Function Test eredményének változása a kiindulási értéktől;
- Egészséggel összefüggő 12-egységes életminőségi teszt eredményének változása a kiindulási értéktől;
- Nem kívánatos eseményt észlelő betegek száma

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline to 12 months

A kiindulási időponttól a 12. hónapig

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

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

Yes

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)

Yes

E.8.2.2

Placebo

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

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

Argentina

Australia

Austria

Belgium

Brazil

Bulgaria

Canada

Colombia

Czech Republic

Denmark

France

Germany

Hungary

Italy

Japan

Korea, Republic of

Mexico

Netherlands

Poland

Portugal

Russian Federation

Spain

Sweden

Switzerland

Taiwan

Turkey

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

Utolsó beteg utolsó vizit

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

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

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:

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Legally minor patients, as defined by local regulation

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)

None

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-01-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-01-24

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-05-31

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