Clinical Trials Register

Summary
EudraCT Number:	2010-023561-22
Sponsor's Protocol Code Number:	POM-001
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-02-07
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

Germany - BfArM

A.2

EudraCT number

2010-023561-22

A.3

Full title of the trial

A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease

A.3.1

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

A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease

A.3.2

Name or abbreviated title of the trial where available

A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease

A.4.1

Sponsor's protocol code number

POM-001

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01230801

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	BioMarin Pharmaceutical 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	BioMarin Pharmaceutical, 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	BioMarin Europe Ltd.
B.5.2	Functional name of contact point	BMN701 Clinical Program Management
B.5.3	Address:
B.5.3.1	Street Address	164 Shaftesbury Avenue
B.5.3.2	Town/ city	London
B.5.3.3	Post code	WC2H 8HL
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	+440782455 2081
B.5.5	Fax number	+440207420 0829
B.5.6	E-mail	smccarthy@bmrn.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	BMN 701
D.3.2	Product code	BMN 701
D.3.4	Pharmaceutical form	Powder 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.3.9.2	Current sponsor code	BMN 701
D.3.9.3	Other descriptive name	GILT-rhGAA
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	24
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

E.1.1.1

Medical condition in easily understood language

Pompe disease

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	16.0
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

The primary objectives of the study are:
• To evaluate the safety and tolerability of BMN 701
• To determine the anti-BMN 701 antibody response to BMN 701; and
• To determine the anti-IGF-I and anti-IGF-II antibody response to BMN 701.

E.2.2

Secondary objectives of the trial

Secondary objectives of the study are,:
• To determine the single-dose and multiple-dose PK of BMN 701; and
• To determine mobility and functional exercise capacity, as measured by the Six-Minute Walk Test (6MWT).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

•Patient has provided written informed consent after the nature of the study has been explained, and prior to any study-related procedures;
• Patient has been diagnosed with Pompe disease, either prior to, or during the screening period based on 2 GAA gene mutations and either o endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblasts,
o or,
o endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay;
• Patient is male or female and 18 years of age or older at the time of enrollment in the study;
• The patient, if sexually active, must be willing to use an acceptable method of contraception while participating in the study and for at least 4 months following the last dose of BMN 701;
• The patient, if female, is not considered to be of childbearing potential: that is she is at least 2 years post-menopausal or had tubal ligation at least 1 year prior to screening, or has had a total hysterectomy;
• The patient, if female of childbearing potential, has negative urine pregnancy tests during the Screening Period and at the Baseline visit and is willing to have additional pregnancy tests during the study;
• If patient is female, she is not lactating;
• Patient has ≥30% predicted upright FVC and either <80% predicted upright FVC or > 10% reduction in supine FVC compared to upright FVC during the Screening Period;
• Patient is naïve to ERT with rhGAA;
• Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted at the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted); and
• Patient has the ability to comply with the protocol requirements, in the opinion of the Investigator.

E.4

Principal exclusion criteria

•Patient has a history of diabetes or other disease known to cause hypoglycemia and is currently receiving, or might anticipate receiving, hypoglycemic agents during the course of the study;
• Patient has been on any immunosuppressive medication other than glucocorticosteroids within 1 year prior to enrollment into this study;
• Patient requires invasive ventilatory assistance at the time of enrollment into the study;
• Patient has received any investigational medication within 30 days prior to the first dose of study drug or is scheduled to receive any investigational drug other than BMN 701 during the course of the study;
• Patient has previously been admitted to the study;
• Patient is breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
• Patient has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient’s ability to comply with the protocol requirements or compromise the patient’s well being or safety;
• Patient has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.

E.5 End points

E.5.1

Primary end point(s)

The primary objectives of the study are:
• To evaluate the safety and tolerability of BMN 701;
• To determine the anti-BMN 701 antibody response to BMN 701

E.5.1.1

Timepoint(s) of evaluation of this end point

Screening
Baseline
Day 1
Week 2-24 qow
30 Days after Week 24

E.5.2

Secondary end point(s)

Secondary objectives of the study are:
• To determine the single-dose and multiple-dose PK of BMN 701; and
• To determine mobility and functional exercise capacity, as measured by the Six-Minute Walk Test (6MWT).

E.5.2.1

Timepoint(s) of evaluation of this end point

Screening
Baseline
Day 1
Week 2-24 qow
30 Days after Week 24

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

Yes

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

Yes

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)

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

Yes

E.8.1.1

Randomised

E.8.1.2

Open

Yes

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

Yes

E.8.1.7.1

Other trial design description

Multiple dose escalation study

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Multiple dose escalation study

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

France

Germany

Netherlands

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

The study will end after the last patient has completed the Follow-up or Termination visit.

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)

Sponsor is planning to conduct an extension study with BMN 701 for eligible patients from POM-001.

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

2012-07-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-06-13

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2013-03-05

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