Clinical Trials Register

Summary
EudraCT Number:	2021-000497-28
Sponsor's Protocol Code Number:	PTC923-MD-004-PKU
National Competent Authority:	Netherlands - Competent Authority
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2022-09-15
Trial 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

Netherlands - Competent Authority

A.2

EudraCT number

2021-000497-28

A.3

Full title of the trial

Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria

Fase 3 open-label uitbreidingsonderzoek van PTC923 bij fenylketonurie

A.3.1

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

A Phase 3 Extension Study of PTC923 in Subjects with Phenylketonuria

Een fase 3 open-label uitbreidingsonderzoek van PTC923 bij patiënten met fenylketonurie

A.4.1

Sponsor's protocol code number

PTC923-MD-004-PKU

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	PTC Therapeutics, 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	PTC Therapeutics, 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	PTC Therapeutics Inc.
B.5.2	Functional name of contact point	Patient Advocacy
B.5.3	Address:
B.5.3.1	Street Address	100 Corporate Court
B.5.3.2	Town/ city	South Plainfield
B.5.3.3	Post code	NJ07080
B.5.3.4	Country	United States
B.5.6	E-mail	Medinfo@ptcbio.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/3/21/2435
D.3 Description of the IMP
D.3.2	Product code	PTC923
D.3.4	Pharmaceutical form	Powder for oral suspension in sachet
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	not available
D.3.9.1	CAS number	17094-01-8
D.3.9.2	Current sponsor code	PTC923
D.3.9.3	Other descriptive name	(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
D.3.9.4	EV Substance Code	SUB193410
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.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/3/21/2435
D.3 Description of the IMP
D.3.2	Product code	PTC923
D.3.4	Pharmaceutical form	Powder for oral suspension in sachet
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	not available
D.3.9.1	CAS number	17094-01-8
D.3.9.2	Current sponsor code	PTC923
D.3.9.3	Other descriptive name	(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
D.3.9.4	EV Substance Code	SUB193410
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	1000
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Metabolic Disorders - Phenylketonuria

Stofwisselingsstoornissen - Fenylketonurie

E.1.1.1

Medical condition in easily understood language

Phenylketonuria (PKU) is an autosomal-recessive inborn error of metabolism characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH), which metabolizes phenylalanine (Phe)

Fenylketonurie (PKU) is een autosomaal recessieve aangeboren stofwisselingsziekte die wordt gekenmerkt door een tekort aan enzym fenylalaninehydroxylase (PAH), dat fenylalanine (Phe) metaboliseert.

E.1.1.2

Therapeutic area

Body processes [G] - Metabolic Phenomena [G03]

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	10034873
E.1.2	Term	Phenylketonuria (PKU)
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 evaluate the long-term safety of PTC923 in subjects with PKU
• To evaluate changes from baseline in dietary Phe/protein consumption

• Evaluaren van de veiligheid van PTC923 op de lange termijn bij proefpersoenen met PKU
• Evaluaren van veranderingen in Phe/eiwitconsumptie ten opzichte van baseline, via de voeding

E.2.2

Secondary objectives of the trial

• To evaluate PTC923 effect on quality of life (QOL) using the
Phenylketonuria-quality of life (PKU-QOL) questionnaire in the subset of subjects that are able to complete the PKU-QOL (ie, subjects whose primary language is English [British or American], Turkish, Dutch, German, Spanish, Italian, Portuguese,
or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages ≥18 years Adult PKU-QOL)
• To evaluate PTC923 effect of QOL using the European Quality of Life 5 Dimensions (EQ-5D) (EQ-5D for Youth [EQ-5D-Y] Proxy Version 1 [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([≥16 years])
•To evaluate the pharmacokinetics (PK) of sepiapterin and tetrahydrobiopterin (BH4) following repeated doses of PTC923

• Evalueren van het effect van PTC923 op de kwaliteit van leven (QOL) met behulp van de fenylketonurie-kwaliteit van leven (PKU-QOL) vragenlijst in de subset van proefpersonen die de PKU-QOL kunnen invullen (ie. proefpersonen waarvan de primaire taal Engels [Brits of Amerikaans], Turks, Nederlands, Duits, Spaans, Italiaans, Portugees of Frans is) (leeftijd 6 tot 8 jaar: Ouders PKU-QOL; leeftijd 9 tot 11 jaar: Kind PKU-QOL; leeftijd 12 tot 17 jaar: Adolescent PKU-QOL; leeftijd ≥18 jaar: Volwassen PKU-QOL)
• Evalueren van het PTC923 effect van QOL met behulp van de European Quality of Life 5 Dimensions (EQ-5D) (EQ-5D voor jongeren [EQ-5D-Y] Proxy Version 1 [3 tot 7 jaar]; EQ-5D-Y [8 tot 15 jaar]; EQ-5D-5L ([≥16 jaar])
• Evalueren van de farmacokinetiek (PK) van sepiapterine en tetrahydrobiopterine (BH4), na herhaalde doses PTC923

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children
and/or subjects that are mentally impaired secondary to disease]) with parental/legal
guardian consent
2. Completed a PTC-sponsored Phase 3 PKU clinical study
3. Women of childbearing potential, as defined in (CTFG 2020), must have a negative pregnancy test at study entry and agree to abstinence or the use of at least one highly effective form of contraception (with a failure rate of <1% per year when used consistently and correctly):
• Combined (estrogen and progestogen containing) hormonal contraception associated
with inhibition of ovulation:
− Oral
− Intravaginal
− Transdermal
• Progestogen-only hormonal contraception associated with inhibition of ovulation:
− Oral
− Injectable
− Implantable
• Intrauterine device
• Intrauterine hormone-releasing system
• Vasectomized partner with confirmed azoospermia
Highly effective contraception or abstinence must be continued for the duration of the
study, and for up to 90 days after the last dose of the study drug.
All females will be considered of childbearing potential unless they are postmenopausal
(at least 12 months consecutive amenorrhea in the appropriate age group without other
known or suspected cause) or have been sterilized surgically (eg, hysterectomy, bilateral
tubal ligation, bilateral oophorectomy).
4. Males who are sexually active with women of childbearing potential who have not had a
vasectomy must agree to use a barrier method of birth control during the study and for up
to 90 days after the last dose of study drug. Males must also refrain from sperm donations
during this time period.
Males who are abstinent will not be required to use a contraceptive method unless they
become sexually active. Males who have undergone a vasectomy are not required to use a
contraceptive method if at least 16 weeks post procedure.
5. Willing and able to comply with the protocol and study procedure
6. Willing to continue current diet unchanged while participating in the study (unless
specifically instructed to change diet during the study by the investigator)

1. Geïnformeerde toestemming en instemming (indien nodig, naar goeddunken van de onderzoeker [dwz voor kinderen
en/of personen met een verstandelijke beperking secundair aan ziekte]) met ouderlijke/juridische toestemming van de voogd.
2. Een door PTC gesponsorde klinische fase 3 PKU-studie afgerond.
3. Vrouwen die zwanger kunnen worden, zoals gedefinieerd in (CTFG 2020), moeten een negatieve zwangerschapstest hebben bij deelname aan het onderzoek en akkoord gaan met onthouding of het gebruik van ten minste één zeer effectieve vorm van anticonceptie (met een mislukkingspercentage van <1% per jaar bij consequent en correct gebruik):
• Gecombineerde (oestrogeen- en progestageen bevattende) hormonale anticonceptie geassocieerd met remming van de ovulatie:
- Oraal
- Intravaginaal
- Transdermaal
• Hormonale anticonceptie met alleen progestageen geassocieerd met remming van de ovulatie:
- Oraal
- Injecteerbaar
- Implanteerbaar
• Spiraaltje
• Intra-uterien hormoonafgevend systeem
• Gevasectomiseerde partner met bevestigde azoöspermie
Zeer effectieve anticonceptie of onthouding moet worden voortgezet voor de duur van de studie, en tot 90 dagen na de laatste dosis van het studiegeneesmiddel.
Alle vrouwen worden beschouwd als vruchtbaar, tenzij ze postmenopauzaal zijn (minstens 12 maanden opeenvolgende amenorroe in de juiste leeftijdsgroep zonder andere bekende of vermoedelijke oorzaak) of operatief zijn gesteriliseerd (bijv. hysterectomie, bilateraal afbinden van de eileiders, bilaterale ovariëctomie).
4. Mannen die seksueel actief zijn met vrouwen in de vruchtbare leeftijd die geen vasectomie hebben gehad, moet instemmen met het gebruik van een barrièremethode voor anticonceptie tijdens het onderzoek en voor maximaal tot 90 dagen na de laatste dosis van het onderzoeksgeneesmiddel. Mannen moeten zich ook onthouden van spermadonaties tijdens deze periode.
Mannen die zich onthouden, hoeven geen anticonceptiemethode te gebruiken, tenzij ze seksueel actief worden. Mannen die een vasectomie hebben ondergaan, hoeven geen anticonceptiemethode te gebruiken indien ten minste 16 weken na de procedure.
5. Bereid en in staat om het protocol en de studieprocedure na te leven.
6. Bereid om het huidige dieet onveranderd voort te zetten tijdens deelname aan het onderzoek (tenzij specifiek geïnstrueerd om tijdens het onderzoek van dieet te veranderen door de onderzoeker).

E.4

Principal exclusion criteria

1. The individual, in the opinion of the investigator, is unwilling or unable to adhere to the
requirements of the study
2. Inability to tolerate oral medication
3. A female who is pregnant or breastfeeding, or considering pregnancy
4. Serious neuropsychiatric illness (eg, major depression) not currently under medical
control, that in the opinion of the investigator or sponsor, would interfere with the
subject’s ability to participate in the study or increase the risk of participation for that
subject
5. Past medical history and/or evidence of renal impairment and/or condition including
moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 mL/min)
as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist
6. Any other condition that in the opinion of the investigator or sponsor, would interfere
with the subject’s ability to participate in the study or increase the risk of participation for
that subject
7. Requirement for concomitant treatment with any drug known to inhibit folate synthesis
(eg, methotrexate)
8. Concomitant treatment with BH4 supplementation (eg, sapropterin dihydrochloride,
KUVAN) or pegvaliase-pqpz (PALYNZIQ)

1. De betrokkene is naar het oordeel van de onderzoeker niet bereid of niet in staat zich te houden aan de eisen van de studie.
2. Onvermogen om orale medicatie te verdragen.
3. Een vrouw die zwanger is of borstvoeding geeft, of zwangerschap overweegt.
4. Ernstige neuropsychiatrische ziekte (bijv. ernstige depressie) die momenteel niet onder medische behandeling valt, die naar de mening van de onderzoeker of sponsor zou interfereren met het vermogen van de proefpersoon om deel te nemen aan het onderzoek of het risico op deelname daarvoor te vergroten.
5. Medische voorgeschiedenis en/of bewijs van nierinsufficiëntie en/of aandoening, waaronder matige/ernstige nierinsufficiëntie (glomerulaire filtratiesnelheid [GFR] <60 ml/min) zoals meest recent geschat tijdens kwalificerende deelname aan een feederonderzoek) en/of onder behandeling van een nefroloog.
6. Elke andere voorwaarde die naar de mening van de onderzoeker of sponsor zou interfereren met het vermogen van de proefpersoon om aan het onderzoek deel te nemen of het risico op deelname voor de proefpersoon.
7. Vereiste voor gelijktijdige behandeling met een geneesmiddel waarvan bekend is dat het de folaatsynthese remt (bijv. methotrexaat).
8. Gelijktijdige behandeling met BH4-suppletie (bijv. sapropterinedihydrochloride, KUVAN) of pegvaliase-pqpz (PALYNZIQ).

E.5 End points

E.5.1

Primary end point(s)

Safety is a primary endpoint: Safety of long-term treatment with PTC923 will be measured by
number of treatment-emergent adverse events (TEAEs), including assessment of severity of
TEAEs, clinical laboratory tests, vital signs, and physical examinations.

The primary efficacy endpoint is the change from baseline in dietary Phe/protein consumption
measured during Phe Tolerance Assessment period.

Veiligheid is een primair eindpunt: de veiligheid van langdurige behandeling met PTC923 zal worden gemeten door:
- aantal op de behandeling optredende bijwerkingen (TEAE's), inclusief beoordeling van de ernst van TEAE's
- klinische laboratoriumtests
- vitale functies
- lichamelijk onderzoek

Het primaire werkzaamheidseindpunt is de verandering ten opzichte van baseline in Phe/eiwitconsumptie via de voeding gemeten tijdens de Phe Tolerance Assessment periode.

E.5.1.1

Timepoint(s) of evaluation of this end point

Safety of long-term treatment with PTC923 will be measured by
number of treatment-emergent adverse events (TEAEs), including assessment of severity of
TEAEs, clinical laboratory tests, vital signs, and physical examinations.

Phe tolerance evaluated at Baseline and every 2 weeks over the course of 26 weeks assessment period

Veiligheid is een primair eindpunt: de veiligheid van langdurige behandeling met PTC923 zal worden gemeten door:
- aantal op de behandeling optredende bijwerkingen (TEAE's), inclusief beoordeling van de ernst van TEAE's
- klinische laboratoriumtests
- vitale functies
- lichamelijk onderzoek

Phe-tolerantie geëvalueerd bij baseline en elke 2 weken gedurende een beoordelingsperiode van 26 weken.

E.5.2

Secondary end point(s)

• Changes from baseline in QOL using PKU-QOL questionnaire in the subset of
subjects that are able to complete the PKU-QOL (ie, subjects whose primary
language is English [British or American], Turkish, Dutch, German, Spanish,
Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to
11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL;
ages ≥18 years Adult PKU-QOL)
• Changes from baseline in QOL using the EQ-5D (EQ-5D-Y Proxy Version 1
[3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([≥16 years])
•PK assessment of sepiapterin and BH4 concentrations in plasma using sparse sampling

• Veranderingen ten opzichte van baseline in QOL met behulp van PKU-QOL-vragenlijst in de subset van proefpersonen die de PKU-QOL kunnen voltooien (dwz proefpersonen waarvan de primaire taal Engels [Brits of Amerikaans], Turks, Nederlands, Duits, Spaans, Italiaans, Portugees of Frans) (leeftijd 6 tot 8 jaar: Ouder PKU-QOL; leeftijd 9 tot 11 jaar: Kind PKU-QOL; 12 tot 17 jaar: Adolescent PKU-QOL; leeftijden ≥18 jaar: volwassen PKU-QOL)
• Veranderingen ten opzichte van baseline in QOL met behulp van de EQ-5D (EQ-5D-Y Proxy Version 1, [3 tot 7 jaar]; EQ-5D-Y [8 tot 15 jaar]; EQ-5D-5L ([≥16 jaar])
•PK-beoordeling van sepiapterine- en BH4-concentraties in plasma met behulp van steekproeven

E.5.2.1

Timepoint(s) of evaluation of this end point

Secondary measures will be evaluated at 6-month intervals starting at M8D1.

Secundaire metingen worden geëvalueerd met tussenpozen van 6 maanden vanaf M8D1.

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

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

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)

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.8.2.4

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

Brazil

Canada

Mexico

United States

France

Netherlands

Spain

Germany

Italy

Denmark

Georgia

Portugal

Turkey

United Kingdom

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

LBLP (laatste bezoek laatste patiënt)

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 days

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)

Yes

F.1.1.3.1

Number of subjects for this age range:

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

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:

100

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

Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children and/or subjects who are mentally impaired secondary to disease]) with parental/legal guardian consent

Geïnformeerde toestemming en instemming (indien nodig, naar goeddunken van de onderzoeker [dwz voor kinderen en/of personen met een verstandelijke beperking als gevolg van ziekte]) met toestemming van de ouder/wettelijke voogd

F.3.3.7

Others

Yes

F.3.3.7.1

Details of other specific vulnerable populations

0-18 years

0-18 jaar

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

200

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

geen

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

2022-09-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-04-26

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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IMP with orphan designation in the indication
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