Clinical Trials Register

Summary
EudraCT Number:	2020-005648-52
Sponsor's Protocol Code Number:	D1346C00015
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2023-12-21
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

2020-005648-52

A.3

Full title of the trial

A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Tolerability and Pharmacokinetics of Selumetinib after Multiple Doses in Adolescent Children with Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)

Ensayo fase I, de un solo brazo, secuencial, para evaluar el efecto de los alimentos sobre la tolerabilidad y la farmacocinética de selumetinib después de dosis múltiples en niños adolescentes con neurofibromas plexiformes (NP) asociados a neurofibromatosis tipo 1 (NF1)

A.3.1

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

Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children with Neurofibromatosis Type 1 (NF1) Related Plexiform
Neurofibromas (PN)

Ensayo en fase I para evaluar el efecto de los alimentos sobre la FC y la tolerabilidad gastrointestinal de selumetinib en adolescentes con neurofibromas plexiformes (NP) asociados a neurofibromatosis de tipo 1 (NF1)

A.3.2

Name or abbreviated title of the trial where available

Selumetinib Gastrointestinal Tolerability Study (Selumetinib GI Tolerability and Food Study)

Ensayo de tolerabilidad gastrointestinal de selumetinib (ensayo de tox. GI de selumetinib)

A.4.1

Sponsor's protocol code number

D1346C00015

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	AstraZeneca AB
B.1.3.4	Country	Sweden
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	AstraZeneca AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AstraZeneca Farmacéutica Spain, S.A.
B.5.2	Functional name of contact point	Unidad de Investigación Clínica
B.5.3	Address:
B.5.3.1	Street Address	C/ Serrano Galvache, 56; Parque Norte, Edificio Álamo
B.5.3.2	Town/ city	Madrid
B.5.3.3	Post code	28033
B.5.3.4	Country	Spain
B.5.4	Telephone number	0034900200444
B.5.6	E-mail	informacionEECC-Spain@astrazeneca.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	Yes
D.2.1.1.1	Trade name	Koselugo 10mg
D.2.1.1.2	Name of the Marketing Authorisation holder	AstraZeneca Pharmaceuticals LP
D.2.1.2	Country which granted the Marketing Authorisation	United States
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/3/18/2050
D.3 Description of the IMP
D.3.1	Product name	Selumetinib 10mg capsule
D.3.2	Product code	AZD6244
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	Yes
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	Selumetinib sulfate
D.3.9.1	CAS number	943332-08-9
D.3.9.2	Current sponsor code	AZD6244
D.3.9.3	Other descriptive name	selumetinib sulfate
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	12.10
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	Yes
D.2.1.1.1	Trade name	Koselugo 25mg
D.2.1.1.2	Name of the Marketing Authorisation holder	AstraZeneca Pharmaceuticals LP
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/3/18/2050
D.3 Description of the IMP
D.3.1	Product name	Selumetinib 25mg capsule
D.3.2	Product code	AZD6244
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	Yes
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	Selumetinib sulfate
D.3.9.1	CAS number	943332-08-9
D.3.9.2	Current sponsor code	AZD6244
D.3.9.3	Other descriptive name	selumetinib sulfate
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	30.25
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

Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)

Neurofibromas plexiformes (NP) asociados a neurofibromatosis de tipo 1 (NF1)

E.1.1.1

Medical condition in easily understood language

Adolescent Children with Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)

Adolescentes con neurofibromas plexiformes (NP) asociados a neurofibromatosis de tipo 1 (NF1)

E.1.1.2

Therapeutic area

Not possible to specify

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	10029270
E.1.2	Term	Neurofibromatosis, type 1 (von Recklinghausen's disease)
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 investigate the effect of a low fat meal on the PK of selumetinib capsules after multiple doses at 25 mg/m2

[If T3 is conducted] To investigate the effect of a low fat meal on the PK of selumetinib capsules after multiple doses at the adjusted dose

To investigate the GI toxicities of selumetinib capsules after multiple doses under fed conditions (T1 and T3) compared to fasted conditions (T2)

Investigar el efecto de alimentos bajos en grasas sobre la FC de las cápsulas de selumetinib tras la administración de dosis múltiples de 25 mg/m2.

[Si se realiza T3] Investigar el efecto de alimentos bajos en grasas sobre la FC de las cápsulas de selumetinib tras la administración de dosis múltiples a la dosis ajustada.

Investigar la toxicidad gastrointestinal (GI) de las cápsulas de selumetinib tras la administración de dosis múltiples en situación posprandial (T1 y T3) en comparación con en ayunas (T2).

E.2.2

Secondary objectives of the trial

To further assess the safety and tolerability of selumetinib capsules by assessment of all AEs, laboratory variables and vital signs

To further evaluate the PK of selumetinib and N-desmethyl selumetinib metabolite after multiple doses under fed conditions, compared to fasted conditions

Evaluar en mayor profundidad la seguridad y tolerabilidad de las cápsulas de selumetinib mediante la evaluación de todos los AA y todas las variables analíticas y constantes vitales.

Evaluar en mayor profundidad la FC de selumetinib y del metabolito N-desmetil-selumetinib tras la administración de dosis múltiples en situación posprandial en comparación con en ayunas.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male and female participants aged ≥ 12 to < 18 years at the time of signing the informed consent.

2. All study participants must be diagnosed with (i) NF1 per NIH Consensus Development Conference Statement and (ii) inoperable PN. In addition to PN, participants must have at least 1 other diagnostic criterion for NF1 as defined in protocol.

3. Participants must require treatment for NF1 and inoperable PN due to actual symptoms or because of the potential to develop significant clinical complications, as judged by the Investigator, as defined in the protocol.

4. Participants who have had prior treatment with any MEKi (including selumetinib) may be considered for inclusion in this study.

5. Participants must have a BSA ≥ 1.3 and ≤ 2.5 m2

1. Participantes de ambos sexos de ≥12 y <18 años en el momento de la firma del consentimiento informado.

2. Todos los participantes del estudio deben tener un diagnóstico de (i) NF1 según la Declaración de las Conferencias de Desarrollo de Consenso de los Institutos Nacionales de Salud (NIH) y (ii) NP inoperable. Además del NP, los participantes deben tener al menos otro de los criterios diagnósticos de NF1 según se define en el protocolo.

3. Los participantes deben necesitar tratamiento para la NF1 y el NP inoperable debido a la presencia de síntomas o a la posibilidad de desarrollar complicaciones clínicas importantes, de acuerdo con el criterio del investigador, según se define en el protocolo.

4. A los participantes que hayan recibido tratamiento previo con algún inhibidor de MEK (incluido selumetinib) se les puede considerar para su inclusión en este estudio.

5. Los participantes deben tener una SC ≥1,3 y ≤2,5 m2.

E.4

Principal exclusion criteria

1. Evidence or suspicion of optic glioma, malignant glioma, MPNST, or other cancer requiring treatment with chemotherapy or radiation therapy.

2. Prior malignancy requiring active treatment (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the participant had been disease free for ≥ 2 years or which would not have limited survival to < 2 years).

3. A life-threatening illness, medical condition, organ system dysfunction of laboratory finding which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of selumetinib, or put the study outcomes at undue risk.

4. Participants with clinically significant cardiovascular disease as listed in the protocol.

5. Liver function tests: bilirubin > 1.5 × the ULN for age (with the exception of those with Gilbert syndrome) or AST/ALT > 2 × upper limit of normal.

6. Renal Function: Creatinine clearance or radioisotope glomerular filtration rate < 30 mL/min/1.73 m2 or a serum creatinine > 1.2 mg/dL (for participants aged between 12 and 15 years) or > 1.5 mg/dL for participants aged > 15 years).

7. Participants with abnormal ophthalmological findings/conditions as listed in the protocol.

8. Have any unresolved chronic toxicity, associated with previous therapy for NF1-PN:

- Gastrointestinal toxicity of CTCAE Grade 1 or higher.
- Have any other unresolved chronic toxicity with CTCAE Grade ≥ 2, except hair changes (such as alopecia or high lightening).

9. Participants who have previously been treated with a MEKi (including selumetinib) and either discontinued treatment or required a dose reduction due to toxicity.

10. Have had recent major surgery within a minimum of 4 weeks prior to starting study intervention, with the exception of surgical placement for vascular access. Have planned major surgery during the treatment period.

11. Any multivitamin containing vitamin E must be stopped at least 7 days prior to initiation of selumetinib.

1. Signos o sospecha de glioma óptico, glioma maligno, tumor maligno de la vaina de los nervios periféricos (TMVNP) u otro cáncer que requiera quimioterapia o radioterapia.

2. Neoplasia maligna previa que requiera tratamiento activo (excepto carcinoma basocelular o epidermoide, cáncer cervicouterino in situ o de otro tipo debidamente tratados y ausentes durante un período ≥2 años, o que no vayan a limitar la supervivencia a un período <2 años).

3. Enfermedad potencialmente mortal, afección médica, insuficiencia orgánica descubierta en el laboratorio que, en opinión del investigador, podría poner en peligro la seguridad del participante, interferir en la absorción o el metabolismo de selumetinib o poner los resultados del estudio en riesgo indebido.

4. Participantes con enfermedad cardiovascular clínicamente significativa, tal como se indica en el protocolo.

5. Pruebas funcionales hepáticas: bilirrubina >1,5 × límite superior de la normalidad (LSN) para la edad (excepto pacientes con síndrome de Gilbert) o AST/ALT >2 × LSN.

6. Función renal: aclaramiento de creatinina o tasa de filtración glomerular radioisótopo <30 ml/min/1,73 m2 o creatinina sérica >1,2 mg/dl (para participantes de 12 a 15 años) o >1,5 mg/dl para participantes de >15 años).

7. Participantes con hallazgos/afecciones oftalmológicos anómalos, tal como se indica en el protocolo.

8. Alguna toxicidad crónica no resuelta asociada al tratamiento previo para la NF1-NP:

- Toxicidad gastrointestinal de grado 1 o superior según los criterios terminológicos comunes para acontecimientos adversos (CTCAE).
- Cualquier otra toxicidad crónica no resuelta de grado ≥2 según los CTCAE, excepto alteraciones capilares (como alopecia o gran aclaramiento del cabello).

9. Participantes tratados previamente con un inhibidor de MEK (incluido selumetinib) y que interrumpieron el tratamiento o necesitaron una reducción de la dosis por toxicidad.

10. Intervención de cirugía mayor reciente, en un plazo mínimo de 4 semanas antes del inicio de la intervención del estudio, excepto colocación quirúrgica para acceso vascular. Intervención de cirugía mayor programada que coincida con el periodo de tratamiento.

11. La toma de suplementos multivitamínicos que contengan vitamina E debe interrumpirse al menos 7 días antes del inicio de selumetinib.

E.5 End points

E.5.1

Primary end point(s)

1. Geometric mean ratio and 90% CI of selumetinib AUC0-12, SS for T1 (fed) versus T2 (fasted).

2. Geometric mean ratio and 90% CI of selumetinib AUC0-12, SS for T3 (fed) versus T2 (fasted).

3. Descriptive statistics for GI AEs graded by CTCAE Version 5.0; Gastrointestinal toxicity diary incorporating the mBSFS-C and Nausea and Vomiting Symptom Rating Scale (adapted from the Children’s Cancer and Leukaemia Group); Usage of GI concomitant medication.

1. Cociente de media geométrica e IC del 90 % del ABC0-12 de selumetinib, EE para T1 (situación posprandial) en comparación con T2 (en ayunas).

2. Cociente de media geométrica e IC del 90 % del ABC0-12 de selumetinib, EE para T3 (situación posprandial) en comparación con T2 (en ayunas).

3. Estadísticos descriptivos de los AA GI clasificados según la versión 5.0 de los CTCAE; diario de toxicidad gastrointestinal que incorpora la escala mBSFS-C y la escala de valoración de los síntomas de náuseas y vómitos (adaptada del Grupo de Cáncer y Leucemia Infantil); uso de medicamentos concomitantes GI.

E.5.1.1

Timepoint(s) of evaluation of this end point

PK: Day 8 of each treatment period;
Safety: from screening until 30 days after last dose

FC: día 8 de cada periodo de tratamiento.
Seguridad: desde la selección hasta 30 días después de la última dosis.

E.5.2

Secondary end point(s)

1. Safety and tolerability will be evaluated in terms of AEs, clinical safety laboratory assessments (clinical chemistry, haematology, urinalysis), physical examination, weight, vital signs, ECG, ECHO or cardiac MRI, ophthalmologic assessment and performance status; Assessments related to AEs will include: occurrence/frequency, relationship to study intervention, CTCAE grade, seriousness, death, AEs leading to discontinuation of study intervention, AEs of special interest.

2. Plasma concentrations and PK parameters of selumetinib and N-desmethyl selumetinib after multiple dose administration, including, but not limited to: Cmax, AUClast, tmax, tlast.

1. La seguridad y la tolerabilidad se evaluarán en función de los AA, evaluaciones analíticas clínicas de la seguridad (bioquímica clínica, hematología, análisis de orina), exploración física, peso, constantes vitales, ECG, ECO o RM cardiaca, evaluación oftalmológica y estado funcional; las evaluaciones relacionadas con los AA incluirán: incidencia/frecuencia, relación con la intervención del estudio, grado según los CTCAE, gravedad, muerte, AA que provoquen la interrupción de la intervención del estudio, AA de especial interés.

2. Concentraciones plasmáticas y parámetros FC de selumetinib y N-desmetil-selumetinib tras la administración de dosis múltiples, entre otros: Cmáx, ABCúltima, tmáx, túltima.

E.5.2.1

Timepoint(s) of evaluation of this end point

PK: Day 8 of each treatment period;
Safety: from screening until 30 days after last dose

FC: día 8 de cada periodo de tratamiento.
Seguridad: desde la selección hasta 30 días después de la última dosis.

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

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)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

The trial evaluating the effect of food on the safety and pharmacokinetics of selumetinib

Ensayo para evaluar el efecto de los alimentos sobre la seguridad y farmacocinética de selumetinib

E.7.2

Therapeutic exploratory (Phase II)

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

Yes

E.8.1.7.1

Other trial design description

Estudio de un solo grupo, secuencial, de dos o tres periodos

Single-arm, Sequential, two or three period 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

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

Russian Federation

United States

Poland

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

LSLV

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)

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)

F.1.3

Elderly (>=65 years)

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

Subjects may have learning difficulties.

Los pacientes pueden tener dificultades de aprendizaje.

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)

If in the PI or the participant's treating physician's opinion, the participant continues to benefit from treatment with selumetinib, participants may (whichever is available earliest):
- Transition to marketed drug available to them by prescription, if selumetinib is approved locally and reimbursed for use in paediatric patients with NF1 who have symptomatic, inoperable PN; or
- Continue to receive selumetinib through PTAP under the same protocol

Si a juicio del IP o médico que trata al paciente,el paciente continua beneficiándose del tratamiento con selumetinib,los pacientes pueden (lo que esté disponible antes):
-Pasar a usar el producto comercializado disponible para los pacientes por prescripción,si selumetinib se aprueba localmente y se reembolsa para el uso en pacientes pediátricos con NF1 que tengan síntomas,PN inoperable; o
-Continuar recibiendo selumetinib a través de Post Trial Access Program (PTAP) bajo el mismo protocolo.

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

2021-05-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-05-19

P. End of Trial
P.	End of Trial Status	Completed

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Clinical trials