Clinical Trials Register

Summary
EudraCT Number:	2015-003681-87
Sponsor's Protocol Code Number:	BMN-044-201
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2021-01-19
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2015-003681-87

A.3

Full title of the trial

A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

Studio di estensione, in aperto, multicentrico, multinazionale, volto a valutare l'efficacia a lungo termine e la sicurezza di BMN 044 (PRO044) in soggetti affetti da Distrofia Muscolare di Duchenne (DMD).

A.3.1

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

A study to assess the effect of BMN 044 in subjects with Duchenne
muscular dystrophy (Extension study)

Studio per valutare l'effetto di BMN 044 in soggetti affetti da Distrofia Muscolare di Duchenne ( studio di estensione)

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

BMN-044-201

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 Pharmaceutical Inc.
B.5.2	Functional name of contact point	Clinical Trials Information
B.5.3	Address:
B.5.3.1	Street Address	105 Digital Drive
B.5.3.2	Town/ city	Novato
B.5.3.3	Post code	CA 94949
B.5.3.4	Country	United States
B.5.4	Telephone number	000000000000000000
B.5.5	Fax number	000000000000000000
B.5.6	E-mail	clinicaltrials@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	Yes
D.2.5.1	Orphan drug designation number	EU/3/08/598
D.3 Description of the IMP
D.3.1	Product name	BMN 044
D.3.2	Product code	BMN 044
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	1802402-63-6
D.3.9.2	Current sponsor code	PS188
D.3.9.3	Other descriptive name	PS188
D.3.9.4	EV Substance Code	SUB179970
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	200
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	Yes
D.3.11.13.1	Other medicinal product type	Antisense oligonucleotide
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/08/598
D.3 Description of the IMP
D.3.1	Product name	BMN 044
D.3.2	Product code	BMN 044
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PS188
D.3.9.1	CAS number	1802402-63-6
D.3.9.2	Current sponsor code	PS188
D.3.9.3	Other descriptive name	PS188
D.3.9.4	EV Substance Code	SUB179970
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	100
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	Yes
D.3.11.13.1	Other medicinal product type	Oligonucleotide antisenso

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

Duchenne muscular dystrophy

Distrofia Muscolare di Duchenne

E.1.1.1

Medical condition in easily understood language

Duchenne's disease

malattia di Duchenne

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10013801
E.1.2	Term	Duchenne muscular dystrophy
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 long-term safety of BMN 044 in subjects with DMD
correctable by BMN 044 induced DMD exon 44 skipping who have
previously participated in an eligible study

¿ valutare la sicurezza a lungo termine di BMN 044 in soggetti affetti da DMD trattabile con skipping BMN 044-indotto dell'esone DMD 44, che hanno partecipato precedentemente a uno studio idoneo

E.2.2

Secondary objectives of the trial

Treatment Period 1:
¿ To evaluate the long-term efficacy of the dosing regimens used in prior
BMN 044 studies.
¿ To evaluate the long-term safety and tolerability of the dosing
regimens used in prior BMN 044 studies.
Treatment Period 2:
¿ To evaluate the long-term efficacy of a selected dosing regimen(s) for
BMN 044.
¿ To evaluate the long-term safety and tolerability of a selected dosing
regimen(s) for BMN 044.
¿ To evaluate the long-term impact on patient reported outcomes of continued treatment with BMN 044.

Periodo di trattamento 1:
¿ valutare l'efficacia a lungo termine dei regimi posologici impiegati negli studi precedenti su BMN 044.
¿ valutare la sicurezza e la tollerabilit¿ a lungo termine dei regimi posologici impiegati negli studi precedenti su BMN 044.
Periodo di trattamento 2:
¿ valutare l'efficacia a lungo termine di un/alcuni regime(i) posologico(i) selezionato(i) di BMN 044.
¿ valutare la sicurezza e la tollerabilit¿ a lungo termine di un/alcuni regime(i) posologico(i) selezionato(i) di BMN 044.
¿ valutare l'influenza a lungo termine del trattamento continuo con BMN 044 sui risultati riferiti dai pazienti.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subjects previously treated with BMN 044 or a comparator treatment
in a BMN 044 Sponsored Study or Investigator Initiated Trial and who
are not eligible for another ongoing BMN 044 study. Subjects who
withdrew from any previous BMN 044 study due to meeting laboratory safety
stopping criteria may be eligible to enroll if the applicable laboratory
parameter has resolved to be within normal limits or parent study baseline value, and benefit of further treatment with BMN 044
outweighs the risk to the individual subject, as agreed in consultation
with the Medical Monitor.
2. Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain
on glucocorticosteroids for the duration of this study. Changes to or
cessation of glucocorticosteroids will be at the discretion of the
Investigator conducting this study in consultation with the
subject/parent and Medical Monitor. Subjects who have discontinued
glucocorticosteroids in their previous BMN 044 study, following
consultation with the Medical Monitor may be enrolled.
3. Willing and able to comply with all study requirements and
procedures.
4. Willing and able to provide written, signed informed consent, or in the
case of subjects under the age of 18 years(or 16 years, depending on the
region), provide written assent (if required) and written informed
consent by a legally authorized representative after the nature of the
study has been explained, and prior to the conduct of any researchrelated
procedures.

1. Soggetti trattati precedentemente con BMN 044 o con un comparatore in uno Studio Sponsorizzato su BMN 044 o intrapreso dallo Sperimentatore e non idonei per un altro studio su BMN 044 in corso. I soggetti che si sono ritirati da qualsiasi studio precedente BMN 044 per via di criteri di esclusione relativi alla sicurezza delle analisi di laboratorio possono essere idonei per l'arruolamento, se il parametro di laboratorio in questione è rientrato nei limiti dela norma o al valore del basale dello studio primario e il beneficio dell'ulteriore trattamento con BMN 044 è superiore al rischio individuale del soggetto, come stabilito dalla consultazione con il Monitor Medico.
2. Uso continuo di glucocorticosteroidi per un minimo di 60 giorni prima dell'ingresso nello studio, con ragionevole aspettativa che il soggetto continui l'assunzione per la durata dello studio. Modifiche o sospensioni del trattamento con glucocorticosteroidi saranno a discrezione dello Sperimentatore che guida lo studio, consultandosi con il soggetto/familiare e il Monitor Medico. I soggetti che hanno sospeso il trattamento con glucocorticosteroidi nel loro studio precedente su BMN 044, previa consultazione con il Monitor Medico, possono essere arruolati.
3. Soggetti capaci e disposti a rispettare tutti i requisiti e le procedure dello studio.
4. Soggetti capaci e disposti a fornire un consenso informato scritto e firmato, o nel caso di soggetti di età inferiore a 18 anni (o 16 anni, a seconda della regione), a fornire consenso scritto (se necessario) e consenso informato scritto da parte di un rappresentante legalmente autorizzato, dopo spiegazione della natura dello studio e prima dell'esecuzione di qualunque procedura legata alla ricerca.

E.4

Principal exclusion criteria

1. Subjects who have previously been treated with BMN 044 who
had a serious adverse experience or met safety stopping criteria
that remains unresolved, which in the opinion of the Investigator could
have been attributable to BMN 044. Once resolved, subject may be
eligible to enter the study following Investigator consultation with the
Medical Monitor.
2. History of significant medical disorder which may confound the
interpretation of safety data (e.g. current or history of renal or liver
disease/impairment, history of inflammatory illness, bleeding
complications, mental retardation and/or behavioral problems).
3. Acute illness within 4 weeks prior to the first dose of BMN 044 (Week
1) which may interfere with the measurements.
4. Symptomatic cardiomyopathy. If subject has a left ventricular ejection
fraction <45% at start of this study, the Investigator should discuss
inclusion of subject in this study with the Medical Monitor.
5. Baseline aPTT above the upper limit of normal (ULN). A re test is
possible at a later stage, and if within normal range, the subject may
enter the study.
6. Baseline platelet count below the lower limit of normal (LLN). A re
test is possible at a later stage, and if within normal range, the subject
may enter the study.
7. Use of anti coagulants, anti thrombotics or anti platelet agents within
28 days of the baseline visit. Chronic use of anti coagulants, anti
thrombotics or anti platelet agents is prohibited during the study. As
needed dosing (pro re nata – PRN) may be acceptable (except for
aspirin) following discussion with the Medical Monitor.
8. Prior use of any investigational product (other than BMN 044) or
investigational medical device must be discussed with the Medical
Monitor prior to screening.
9. Current or history of drug and/or alcohol abuse.

1. Soggetti trattati precedentemente con BMN 044 che hanno riportato una reazione avversa grave o in cui non si è verificata risoluzione di uno dei criteri di esclusione di sicurezza, che in base all'opinione dello Sperimentatore potrebbe essere attribuibile a BMN 044. Una volta risolta la questione, il soggetto può essere considerato idoneo all'ingresso nello studio previa consultazione dello Sperimentatore con il Monitor Medico.
2.Anamnesi di disturbi medici significativi che potrebbero alterare l'interpretazione dei dati sulla sicurezza (ad es. anamnesi o attuale malattia/danno renale o epatico, anamnesi di malattie a carattere infiammatorio, complicanze emorragiche, ritardo mentale e/o problemi comportamentali).
3. Patologia in fase acuta a 4 settimane dalla prima dose di BMN 044 (Settimana 1) che può interferire con le misurazioni.
4. Cardiomiopatia sintomatica. Se il soggetto ha una frazione dell'eiezione ventricolare sinistra <45% all'inizio dello studio, lo Sperimentatore deve discutere della sua inclusione con il Monitor Medico.
5. Tempo di tromboplastina parziale attivata (aPTT) basale al di sopra del limite superiore della norma (ULN). È possibile eseguire un secondo test in una fase successiva; se i valori rientrano nella norma il soggetto può entrare a far parte dello studio.
6. Conta delle piastrine basale al di sotto del limite inferiore della norma (LLN). È possibile eseguire un secondo test in una fase successiva; se i valori rientrano nella norma il soggetto può entrare a far parte dello studio.
7. Uso di anticoagulanti, agenti antitrombotici o antiaggreganti nei 28 giorni precedenti la visita basale. L'uso cronico di anticoagulanti, agenti antitrombotici o antiaggreganti non è consentito durante lo studio. Le somministrazioni in caso di necessità (pro re nata - PRN) possono essere accettate (ad eccezione dell'aspirina) previa discussione con il Monitor Medico.
8.L'uso precedente di qualsiasi prodotto (diverso da BMN 044) o dispositivo medico sperimentale deve essere discusso con il Monitor Medico prima dello screening.
9.Anamnesi o storia attuale di abuso di droghe e/o alcol.

E.5 End points

E.5.1

Primary end point(s)

Efficacy:
Ambulant subjects:
• Muscle function using:
- 6 minute walking distance (6MWD) test
- Timed function tests (4 stair climb, Rise from floor, 10 meter walk/run)North Star Ambulatory Assessment
• Patient Reported Outcomes Measure (PROM), Patient Outcomes Data
Collection Instrument (PODCI), EQ 5D 5L
• Pulmonary function (forced expiratory volume in the 1st second of
exhalation [FEV1], forced vital capacity [FVC], Maximum Inspiratory
Pressure [MIP], Maximum Expiratory Pressure [MEP], Peak Cough Flow
[PCF], and Peak Flow [PF])
• Time to major disease milestones (e.g. loss of ambulation, night time
ventilation)
• Performance Upper Limb (PUL)
Non-ambulant subjects:
• Pulmonary function (FEV1, FVC, MIP, MEP, PCF, and PF)
• Time to major disease milestones (e.g. night time ventilation)
• PUL
• Egen Klassification
• PROM, PODCI, EQ 5D 5L
Safety:
• Incidence and severity of adverse events
• Vital signs
• ECG parameters
• Injection Site Reactions
• Safety hematology and biochemistry parameters including nonstandard
parameters such as coagulation parameters (in particular
aPTT), serum cystatin C, Complement Factor C3/C4/H, haptoglobin,
fibrinogen, high sensitivity C-reactive protein (hsCRP)
• Urinalysis (including quantitative protein and creatinine and their ratio)• Anti-dystrophin antibodies and anti-BMN 044 antibodies
Pharmacodynamic:
• Serum Creatine Kinase (CK), lactate dehydrogenase (LDH) and other
biomarkers to be defined
Pharmacokinetic:
• Plasma PK pre-dose every 24 weeks

Efficacia:
Soggetti ambulanti:
Uso della funzionalità muscolare:
- Test del cammino dei 6 minuti (6MWD)
- Test della funzionalità a tempo (salita 4 gradini, sollevarsi da terra, 10 metri cammino/corsa)
• North Star Ambulatory Assessment
• Patient Reported Outcomes Measure (PROM), Patient Outcomes Data Collection Instrument (PODCI), EDEQ-5D-5L
• Funzionalità polmonare (Flusso espiratorio forzato nel primo secondo di espirazione [FEV1], Capacità vitale forzata [FVC],
Massima Pressione Inspiratoria [MIP], Massima Pressione Espiratoria [MEP], Picco di Flusso della Tosse [PCF] e Picco di Flusso [PF])
• Tempo delle fasi principali della malattia (ad es. perdita della deambulazione, ventilazione notturna)
• Prestazioni degli arti superiori (Performance Upper Limb-PUL).
Soggetti non ambulanti:
Funzionalità polmonare (FEV1, FVC, MIP, MEP, PCF e PF)
• Tempo delle fasi principali della malattia (ad es. ventilazione notturna)
• PUL
• Egen Klassification
• PROM, PODCI, EDEQ-5D-5L
Sicurezza
• Incidenza e gravità degli eventi avversi
• Segni vitali
• Parametri ECG
• Reazioni in sede di iniezione
• Parametri di sicurezza ematologici e biochimici inclusi parametri non standard come quelli relativi a coagulazione (in particolare
aPTT), cistatina C sierica, Fattore del Complemento C3/C4/H, aptoglobina, fibrinogeno, Proteina C reattiva ad alta sensibilità
(hsCRP)
• Esame delle urine (inclusa la valutazione quantitativa di proteine e creatinina e il loro rapporto)
• Anticorpi antidistrofina e anticorpi anti-BMN044
Farmacodinamica:
• Creatinchinasi sierica (CK), lattato deidrogenasi (LDH) ed altri biomarcatori da definire
Farmacocinetica:
• Farmacocinetica plasmatica pre-dose ogni 24 settimane

E.5.1.1

Timepoint(s) of evaluation of this end point

Please refer to Section 12.3.2 Study period procedures and assessments in the Protocol

Si prega di far riferimento alla Sez. 12.3.2 del protocollo, procedure e test del periodo di studio

E.5.2

Secondary end point(s)

E.5.2.1

Timepoint(s) of evaluation of this end point

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

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

dose exploration

esplorazione della dose

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)

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

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

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

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

Information not present in EudraCT

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

United States

Belgium

Italy

Netherlands

Sweden

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
A subject will continue in BMN-044-201 until one of the following
occurs:
¿ The subject withdraws consent and discontinues from the study.
¿ The subject is discontinued from the study at the discretion of the
Investigator or BioMarin.
¿ The study is terminated.
¿ The study drug receives marketing authorization in their country of
residence

LVLS
I soggetti continueranno il trattamento con BMN-044-201 fino a quando non si verifica una delle seguenti condizioni:

¿ Il soggetto ritira il consenso e interrompe la partecipazione allo studio.
¿ Il soggetto viene escluso dallo studio a discrezione dello Sperimentatore o di BioMarin.
¿ Lo studio viene terminato.
¿ Il farmaco dello studio riceve l'autorizzazione all'immissione in commercio nel proprio paese di residenza.

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

Yes

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)

F.2 Gender

F.2.1

Female

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

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

Children under the age of 18

Bambini di et¿ inferiore ai 18

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)

This study will be active until marketing authorization or termination.
Once BMN044 is commercially available, subjects can keep on their
treatment with commercial supplies.

Questo studio sar¿ attivo fino all'autorizzazione in commercio oppure fino alla cessazione. Una volta che BMN044 sar¿ disponibile in commercio, i soggetti potranno continuare il loro trattamento con il farmaco commerciale.

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

2016-03-08

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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