Clinical Trials Register

Summary
EudraCT Number:	2021-000497-28
Sponsor's Protocol Code Number:	PTC923-MD-004-PKU
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-04-04
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

Spain - AEMPS

A.2

EudraCT number

2021-000497-28

A.3

Full title of the trial

Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria

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

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

Enfermedades Metabólicas - fenilcetunuria

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)

La fenilcetonuria (PKU) es un error congénito autosómico-recesivo del metabolismo caracterizado por la deficiencia de la enzima fenilalanina hidroxilasa (HAP), que metaboliza la fenilalanina (Phe

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

• Evaluar la seguridad a largo plazo de PTC923 en pacientes con fenilcetonuria (FCU).
• Evaluar las variaciones con respecto al valor inicial en el consumo de fenilalanina (FA)/proteínas en la dieta.

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

•Evaluar el efecto de PTC923 en la calidad de vida(CdV)mediante el cuestionario sobre la calidad de vida en la fenilcetonuria(PKU-QOL)en el subgrupo de sujetos que pueden completar el cuestionario PKUQOL(sujetos cuyo idioma principal es inglés [británico o americano],turco,neerlandés,alemán,español,italiano,portugués o francés)(pacientes con edades de 6 a 8 años,cuestionario PKU-QOL para padres;pacientes con edades de 9 a 11 años,cuestionario PKU-QOL para niños;pacientes con edades de 12 a 17 años,cuestionario PKU-QOL para adolescentes;pacientes con ≥18 años de edad,cuestionario PKU-QOL para adultos).
•Evaluar el efecto de PTC923 en la CdV utilizando el cuestionario europeo de calidad de vida de 5 dimensiones (EQ-5D)(EQ-5D para jóvenes [EQ-5D for Youth, EQ-5D-Y] respondido por sus cuidadores, V. 1 [de 3 a 7 años];EQ-5D-Y [de 8 a 15 años];EQ-5D-5L [≥16 años]).
•Evaluar la farmacocinética de la sepiapterina y la tetrahidrobiopterina tras la administración de dosis repetidas de 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. Consentimiento y asentimiento informado (si es necesario, a criterio del investigador [p. ej., para niños y/o pacientes con deterioro mental secundario a la enfermedad]) con el consentimiento de los padres/tutores legales.
2. Pacientes que hayan completado un estudio clínico de fase III sobre la FCU patrocinado por PTC.
3. Las mujeres con capacidad de procrear, según se define en (CTFG 2020), deben tener un resultado negativo en una prueba de embarazo en el momento de entrada en el estudio y aceptar la abstinencia sexual o el uso de al menos un método anticonceptivo de granPTC923-MD-004-PKU Protocolo clínico V4.0 07-02-2022
V4.0 07-02-2022 PTC Therapeutics. - Confidencial Página 7 de 9 eficacia (con una tasa de fracaso de <1 % al año cuando se usan de forma sistemática y correcta):
• Anticonceptivos hormonales combinados (con estrógenos y gestágenos) asociados a la inhibición de la ovulación:
− Orales
− Intravaginales
− Transdérmicos
• Anticonceptivos hormonales únicamente con gestágenos asociados a la inhibición de la ovulación:
− Orales
− Inyectables
− Implantables
• Dispositivo intrauterino
• Sistema intrauterino liberador de hormonas
• Pareja vasectomizada con azoospermia confirmada
Los métodos anticonceptivos de gran eficacia o la abstinencia deben mantenerse durante todo el estudio y hasta 90 días después de la última dosis del fármaco del estudio.
Todas las mujeres se considerarán fértiles a menos que sean posmenopáusicas (al menos 12 meses consecutivos de amenorrea en el grupo de edad correspondiente sin otra causa conocida o sospechada) o hayan sido esterilizadas quirúrgicamente (por ejemplo, histerectomía, ligadura de trompas bilateral, ovariectomía bilateral).
4. Los varones sexualmente activos con parejas femeninas con capacidad de procrear que no se hayan sometido a una vasectomía deben aceptar el uso de un método anticonceptivo de barrera durante el estudio y hasta 90 días después de la última dosis del medicamento del estudio. Los varones también deben abstenerse de donar semen durante este periodo de tiempo.
Los varones que practiquen la abstinencia no estarán obligados a utilizar un método
anticonceptivo a menos que sean sexualmente activos. Los varones que se hayan
sometido a una vasectomía no están obligados a utilizar un método anticonceptivo si han pasado al menos 16 semanas desde la intervención.
5. Disposición y capacidad para cumplir el protocolo y los procedimientos del estudio.
6. Disposición para seguir con la dieta actual sin cambios durante la participación en el estudio (a menos que el investigador indique específicamente un cambio en la dieta durante el estudio)

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. El sujeto, en opinión del investigador, no está dispuesto a cumplir los requisitos del estudio o es incapaz de hacerlo.
2. Incapacidad para tolerar la medicación oral.
3. Mujeres embarazadas o en periodo de lactancia, o que estén pensando en quedarse embarazadas.
4. Enfermedad neuropsiquiátrica grave (p. ej., depresión mayor) que actualmente no está bajo control médico, que, en opinión del investigador o del promotor, podría interferir en la capacidad del paciente para participar en el estudio o aumentar el riesgo de la participación en ese paciente.
5. Antecedentes médicos y/o indicios de alteración y/o afección renal, incluida
insuficiencia renal moderada/grave (filtración glomerular [FG] <60 ml/min según lo
calculado más recientemente durante la cualificación para la participación en un estudio previo) y/o atendidos por un nefrólogo.
6. Cualquier otra afección que, en opinión del investigador o del promotor, pudiera
interferir en la capacidad del paciente para participar en el estudio o aumentar el riesgo de la participación en ese paciente.
7. Necesidad de tratamiento concomitante con cualquier fármaco conocido por inhibir la síntesis del folato (p. ej., metotrexato).
8. Tratamiento concomitante con un suplemento de BH4 (p. ej., dihidrocloruro de
sapropterina, KUVAN) o pegvaliasa-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.

La seguridad es un criterio principal de valoración: la seguridad del tratamiento a largo plazo con PTC923 se determinará mediante el número de acontecimientos adversos aparecidos durante el tratamiento (AAT), incluida la evaluación de la intensidad de los AAT, los análisis clínicos, las constantes vitales y las exploraciones físicas.
El criterio principal de valoración de la eficacia es la variación con respecto al inicio en el consumo de FA/proteínas en la dieta, medido durante el periodo de evaluación de la tolerancia a la FA

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

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

• Variaciones con respecto al inicio en la calidad de vida determinada mediante el
cuestionario PKU-QOL en el subgrupo de sujetos que pueden completar el cuestionario PKU-QOL (es decir, sujetos cuyo idioma principal es inglés [británico o americano], turco, neerlandés, alemán, español, italiano, portugués o francés) (en pacientes con edades de 6 a 8 años, cuestionario PKU-QOL para padres; en pacientes con edades de 9 a 11 años, cuestionario PKU-QOL para niños; en pacientes con edades de 12 a 17 años, cuestionario PKU-QOL paraPTC923-MD-004-PKU
Protocolo clínico V4.0 07-02-2022 V4.0 07-02-2022 PTC Therapeutics. - Confidencial Página 9 de 9 adolescentes; en pacientes con ≥18 años de edad, cuestionario PKU-QOL para adultos).
• Variaciones respecto al inicio en la CdV determinada mediante el cuestionario
EQ-5D (EQ-5D-Y respondido por sus cuidadores, versión 1 [de 3 a 7 años]; EQ-5D-Y [de 8 a 15 años]; EQ-5D-5L [≥16 años]).
• Evaluación de la FC de las concentraciones de sepiapterina y BH4 en plasma
mediante muestreo disperso.

E.5.2.1

Timepoint(s) of evaluation of this end point

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

Los criterios secundarios de valoración se evaluarán en intervalos de 6 meses a partir del D1M8

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

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

Australia

Brazil

Canada

Georgia

Mexico

Turkey

United States

Denmark

France

Germany

Italy

Portugal

United Kingdom

Netherlands

Spain

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

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

F.3.3.7

Others

Yes

F.3.3.7.1

Details of other specific vulnerable populations

0-18 years

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

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

2022-06-02

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-19

P. End of Trial
P.	End of Trial Status	Ongoing

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