Clinical Trials Register

Summary
EudraCT Number:	2021-003791-13
Sponsor's Protocol Code Number:	BP42992
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-10-08
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

2021-003791-13

A.3

Full title of the trial

A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN CHILDREN WITH DUP15Q SYNDROME.

ESTUDIO DE FASE II, ALEATORIZADO, DOBLE CIEGO,CONTROLADO CON PLACEBO Y DE GRUPOS PARALELOS PARA EVALUAR LA SEGURIDAD, LA EFICACIA Y LA FARMACODINÁMICA DEL TRATAMIENTO CON BASMISANIL DURANTE 52 SEMANAS EN NIÑOS CON SÍNDROME DUP15Q

A.3.1

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

A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of 52 weeks of Treatment with Basmisanil in Children with Dup15q Syndrome.

A.4.1

Sponsor's protocol code number

BP42992

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	Roche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
B.1.3.4	Country	Switzerland
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	F.Hoffmann-La Roche Ltd
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	F. Hoffmann-La Roche Ltd
B.5.2	Functional name of contact point	Trial Information Support Line-TISL
B.5.3	Address:
B.5.3.1	Street Address	Grenzacherstrasse 124
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	4070
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+349132557300
B.5.5	Fax number	+34913248196
B.5.6	E-mail	spain.start_up_unit@roche.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	basmisanil
D.3.2	Product code	Ro 518-6582/F13-01
D.3.4	Pharmaceutical form	Granules in sachet
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	basmisanil
D.3.9.1	CAS number	1159600-41-5
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	GABAA a5 negative allosteric modulator, previously referred to as GABAA a5 inverse agonist
D.3.9.4	EV Substance Code	SUB31464
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	40
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Granules in sachet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Dup15q Syndrome

SÍNDROME DUP15Q

E.1.1.1

Medical condition in easily understood language

Dup15q Syndrome is a neurodevelopmental disorder, caused by the partial duplication of Chromosome 15, that confers a strong risk for autism spectrum disorder, epilepsy, and intellectual disability.

Dup15q es un trastorno del neurodesarrollo, causado por la duplicación parcial del cromosoma 15,que confiere un fuerte riesgo de trastorno del espectro autista, epilepsia y discapacidad intelectual.

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	23.0
E.1.2	Level	LLT
E.1.2	Classification code	10083952
E.1.2	Term	Dup15q syndrome
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the effects of basmisanil treatment on core symptom domains of Dup15q syndrome (language and social skills) and daily functioning.

E.2.2

Secondary objectives of the trial

• To evaluate the effects of basmisanil treatment on Motor function, Cognition, Language, Social skills, Clinical impression of global functioning, Challenging behaviors
• To evaluate the tolerability and safety of 52 weeks treatment of basmisanil
• To characterize the PK of basmisanil and its metabolite M1 and determine the dose that will deliver target exposure in participants aged 2-11 years
• To evaluate potential relationships between selected covariates and exposure to basmisanil
• To evaluate the effects of basmisanil treatment on the characteristic Dup15q EEG phenotype acutely and at steady state.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Participants aged 2 to 11 years inclusive at the time the caregiver signs the informed consent (for Stage 1, participants aged 6 to 11 years inclusive; for Stage 2, participants aged 2 to 5 years inclusive; and for Stage 3, participants aged 2 to 11 years inclusive)
• Documented maternal duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2-q13.1 region that includes the Prader-Willi/Angelman critical region defined as [BP2-BP3] segment
• Dup15q syndrome Clinical Global Impression Severity scale (Dup15q CGI-S) overall severity score ≥ 4 (at least moderately ill)
• Stages 1 and 2: Participants with epilepsy only
• Body weight equal to or above the third percentile for age
• Male and female participants: Some of the provisions that follow may have limited applicability based on the age range of study participants (i.e., up to the age of 11) and the nature of the disease under study
• Female Participants: A female participant is eligible to participate if she is not pregnant, not breastfeeding
• Male Participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse
• The participant has a parent, caregiver, or legally authorized representative (hereinafter “caregiver”) of at least 18 years of age, who is fluent in the local language at the site, and capable and willing to provide written informed consent for the participant according to International Council on Harmonisation and local regulations
• The participant’s caregiver must be living with the participant and, in the opinion of the Investigator, able and willing to reliably assess the participant’s ongoing condition, to accompany the participant to all clinic visits, and ensure compliance to study treatment throughout the study. The same caregiver is able and willing to complete the caregiver assessments and is available to the Investigational Site by telephone or email if needed
• The participant’s caregiver is able and willing to use electronic devices to record information on the participant’s condition and to complete assessments at home and agrees to home nursing visits, if local regulations allow for it and if home nursing service is available in the country/region.

1. Participantes de 2 a 11 años, ambos inclusive, en el momento en que el cuidador firme el
consentimiento informado (en la etapa 1, participantes de 6 a 11 años, ambos inclusive; en la
etapa 2, participantes de 2 a 5 años, ambos inclusive; y en la etapa 3, participantes de 2 a
11 años, ambos inclusive).
2. Duplicación (3 copias) o triplicación (4 copias) materna documentada de la región
cromosómica 15q11.2-q13.1 que incluye la región crítica de Prader-Willi/Angelman definida
como el segmento [BP2-BP3].
3. Puntuación de gravedad global en la escala Dup15q CGI-S (Escala de impresión clínica
global de la gravedad para el síndrome Dup15q) ≥ 4 (al menos moderadamente enfermo).
4. Etapas 1 y 2: Participantes con epilepsia exclusivamente
5. Peso corporal igual o superior al percentil 3 para la edad.
6. Participantes de cualquier sexo: Algunas de las disposiciones siguientes pueden tener una aplicabilidad limitada en función del intervalo de edad de los participantes en el estudio (es decir, hasta los 11 años) y la naturaleza de la enfermedad en estudio.

7. Niñas participantes: En el estudio podrán participar niñas que no estén embarazadas ni en período de lactancia

8. Niños participantes: En este estudio no se exige anticoncepción masculina debido a la dosis seminal mínima que se transmite por el coito.

9. El participante dispone de un progenitor, cuidador o representante legal (en adelante,
“cuidador”) mayor de 18 años que domina el idioma local utilizado en el centro y que es
capaz y está dispuesto a otorgar su consentimiento informado por escrito en nombre del
participante, de conformidad con las normas del Consejo Internacional de Armonización y la
normativa local.

10. El cuidador del participante debe estar viviendo con este último y, en opinión del investigador,
ser capaz y estar dispuesto a evaluar de manera fiable su enfermedad presente,
acompañarle a todas las visitas al centro y garantizar el cumplimiento del tratamiento del
estudio durante todo el estudio. El mismo cuidador es capaz y está dispuesto a realizar las
evaluaciones por parte del cuidador y estará disponible por teléfono o correo electrónico para
el centro de investigación, en caso necesario.

11. El cuidador del participante es capaz y está dispuesto a utilizar dispositivos electrónicos para
registrar información sobre la enfermedad del participante y realizar evaluaciones en el
domicilio y accede a las visitas de enfermería domiciliaria, siempre que lo permita la
normativa local y se disponga de un servicio de enfermería domiciliaria en el país o región.

E.4

Principal exclusion criteria

• Individuals with known pathogenic mutations or copy number variants, of established neurodevelopmental significance, other than duplication or triplication of the chromosome 15q11.2-q13.1
• Uncontrolled epilepsy at Screening as indicated by: Use of rescue medication more than once per month on average in the past 6 months or concomitant use of more than 4 anti-epileptic medications or status epilepticus within the past 6 months requiring urgent hospitalization or any implanted devices to treat drug-resistant epilepsy
• Lymphoma, leukemia, or any malignancy within the past 5 years, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years
• Clinically significant ECG abnormalities at Screening, including an average triplicate QTcF > 450 ms for participants > 10 years or QTcB > 450 ms for children up to and including age 10 years
• Clinically significant abnormalities in laboratory test results at screening (including positive results for HIV, hepatitis B and/or hepatitis C). ALT values > 1.5 × the upper limit of normal
• Allowed prior existing medication should be on a stable regimen (or frequency of intervention) for at least 6 weeks, and at least 8 weeks for anti-epileptic treatment, prior to Screening
• Non-pharmacological / behavioral therapies should not be stopped or newly started at least 6 weeks prior to Screening and are expected to remain stable for the entire study duration (excluding changes related to standard age and educational interventional programs and minor interruptions such as illness or vacation
• Concomitant use of prohibited medications
• Participation in an investigational drug study within one month or within 6 × the elimination half-life, whichever is longer, prior to dosing in the study
• Significant risk for suicidal behavior, in the opinion of the Investigator as assessed by suicidality questions adapted from the Columbia Classification Algorithm for Suicide (C-CASA)
• Known sensitivity to any of the study treatments or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates the participation in the study, including severe lactose intolerance
• Concomitant clinically relevant disease or condition or any clinically significant finding at screening that could interfere with, or for which, the treatment might interfere with, the conduct of the study or that would pose an unacceptable risk to the participants in this study
• Known active or uncontrolled bacterial, viral, or other infection or any major clinically significant episode of infection or hospitalization (relating to the completion of the course of antibiotics) within 6 weeks prior to the start of drug administration.

1. Sujetos con mutaciones patógenas conocidas o variantes en el número de copias, con
importancia confirmada para el desarrollo neurológico, distintas de una duplicación o
triplicación del cromosoma 15q11.2-q13.1.
2. Epilepsia no controlada en el período de selección, indicada por: Uso de medicación de rescate más de una vez al mes, por término medio, en los últimos 6 meses o Uso concomitante de más de cuatro antiepilépticos o Estado epiléptico en los 6 últimos meses con necesidad de hospitalización urgente o presencia de cualquier dispositivo implantado para tratar la epilepsia farmacorresistente.
3. Linfoma, leucemia o cualquier neoplasia maligna en los últimos 5 años, excepto carcinomas basocelulares o espinocelulares de piel que se hayan resecado sin signos de metástasis durante 3 años.

4. Anomalías electrocardiográficas clínicamente significativas en el período de selección, incluido un QTcF (promedio de tres) >450 ms en los participantes > 10 años o un QTcB >450 ms en los niños de hasta 10 años, inclusive.

5. Anomalías clínicamente significativas en los resultados de los análisis clínicos de selección (incluidos resultados positivos para VIH, hepatitis B o hepatitis C). Valores de ALT > 1.5 el límite superior de la normalidad.

6. Los medicamentos previos permitidos deben mantenerse en una pauta (o frecuencia de intervención) estable durante al menos 6 semanas (8 semanas en el caso de tratamientos antiepilépticos) antes de la selección.
7. Las terapias no farmacológicas o conductuales no deben interrumpirse ni iniciarse al menos 6 semanas antes de la selección y cabe esperar que se mantengan estables durante todo el estudio (excluidos los cambios relacionados con la edad normal y los programas de intervención educativa, así como las interrupciones poco importantes, por ejemplo, por enfermedad o vacaciones).
8. Uso concomitante de medicamentos prohibidos.
9. Participación en un estudio con un fármaco en investigación en el mes anterior o en el plazo equivalente a 6 veces la semivida de eliminación, lo que suponga más tiempo, antes de la administración en el estudio
10. Riesgo significativo de conducta suicida, en opinión del investigador, evaluado mediante preguntas sobre tendencias suicidas adaptadas de la escala C-CASA.
11. Sensibilidad conocida a cualquiera de los tratamientos del estudio o sus componentes o alergias a fármacos u otras sustancias que, en opinión del investigador, contraindiquen la participación en el estudio, incluida la intolerancia grave a la lactosa.

12. Enfermedad o trastorno concomitante clínicamente relevante o cualquier hallazgo clínicamente significativo en el período de selección que pueda interferir, o cuyo tratamiento podría interferir, en la realización del estudio o que pueda suponer un riesgo inaceptable para los participantes en este estudio.
13. Infección conocida activa o no controlada por bacterias, virus u otros microorganismos (excepto micosis de los lechos ungueales) o cualquier episodio importante y clínicamente significativo de infección u hospitalización (en relación con la finalización del ciclo de antibióticos) en las 6 semanas previas al inicio de la administración del fármaco

E.5 End points

E.5.1

Primary end point(s)

1. Vineland Adaptive Behavior Scale–Third Edition (Vineland-3): Adaptive Behavior Composite.

1. Escala de comportamiento adaptativo de Vineland - Tercera edición (Vineland-3): Compuesto de comportamiento adaptativo.

E.5.1.1

Timepoint(s) of evaluation of this end point

1. Baseline (Day -7 to Day -1), Day 183, Day 365.

1. Visita basal (día -7 a día -1), día 183, día 365.

E.5.2

Secondary end point(s)

1. Vineland-3: gross and fine motor, expressive and receptive language, play and leisure time and interpersonal relationships subdomains
2. Mullen Scales of Early Learning (MSEL): gross and fine motor domains, visual reception domain, expressive and receptive language subdomains domains
3. Dup15q syndrome Clinical Global Impression Severity scale (Dup15q CGI-S)
4. Dup15q syndrome Clinical Global Impression Change scale (Dup15q CGI-C)
5. Aberrant Behavior Checklist - Second Edition – Community Version (ABC-2-C) domain scores
6. Incidence, nature, and severity of AEs and SAEs
7. Incidence of treatment discontinuations due to AEs
8. Incidence of laboratory abnormalities based on hematology, clinical chemistry, and urinalysis test results
9. ECG changes from baseline; incidence of abnormal ECG assessments
10. Change from baseline in all seizure frequency, duration, and type of seizure as reported in a seizure diary by caregivers
11. Abnormal changes in EEG recordings compared to baseline with a focus on treatment-emergent epileptiform abnormalities
12. Systolic and diastolic blood pressure and heart rate measurements
13. Suicidality as assessed through questions from selected items adapted from the Columbia Classification Algorithm for Suicide (C-CASA) in children aged 6-11 years
14. Height, weight, head circumference
15. Tanner staging over time (in children aged 9-11 years)
16. Plasma concentration of basmisanil and its major metabolite M1 at specified timepoints
17. Plasma concentration ratio of M1 to basmisanil at trough
18. qEEG beta-band power.

1. Vineland-3: subdominios de motricidad fina y gruesa, lenguaje expresivo y receptivo, juego y tiempo libre y relaciones interpersonales
2. Escalas de Mullen de Aprendizaje Temprano (MSEL): dominios de motricidad gruesa y fina, dominio de recepción visual, dominios de subdominios del lenguaje expresivo y receptivo
3. Escala de gravedad de la impresión clínica global del síndrome Dup15q (Dup15q CGI-S)
4. Escala de cambio de impresión clínica global del síndrome Dup15q (Dup15q CGI-C)
5. Lista de verificación de comportamiento aberrante - Segunda edición - Puntajes de dominio de la versión comunitaria (ABC-2-C)
6. Incidencia, naturaleza y gravedad de los EA y los EA
7. Incidencia de interrupciones del tratamiento debido a EA
8. Incidencia de anomalías de laboratorio basadas en hematología, química clínica y resultados de análisis de orina.
9. Cambios en el ECG desde la línea de base; incidencia de evaluaciones de ECG anormales
10. Cambio desde el valor inicial en toda la frecuencia, duración y tipo de convulsiones según lo informado en un diario de convulsiones por los cuidadores
11. Cambios anormales en los registros de EEG en comparación con la línea de base con un enfoque en las anomalías epileptiformes emergentes del tratamiento
12. Mediciones de la presión arterial sistólica y diastólica y la frecuencia cardíaca
13. El suicidio evaluado a través de preguntas de elementos seleccionados adaptados del algoritmo de clasificación de Columbia para el suicidio (C-CASA) en niños de 6 a 11 años
14. Altura, peso, circunferencia de la cabeza
15. Estadificación de Tanner a lo largo del tiempo (en niños de 9 a 11 años)
16. Concentración plasmática de basmisanil y su principal metabolito M1 en momentos específicos
17. Relación de concentración plasmática de M1 a basmisanil en el valle
18. Potencia de banda beta de qEEG.

E.5.2.1

Timepoint(s) of evaluation of this end point

1-2. Baseline, Day 183, Day 365
3. Screening, Baseline, Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up
4. Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up
5. Baseline, Day 28, Day 92, Day 183, Day 274, Day 365
6-8. Up to Day 395
9-11. Baseline to Day 395
12-15. Up to Day 395
16-17. Day 1, Day 2, Day 14, Day 92, Day 183, Day 274, Day 365
18. Baseline, Day 1, Day 2, Day 14, Day 183, Day 365.

1-2. Visita basal, día 183, día 365
3. Detección, línea de base, día 28, día 92, día 183, día 274, día 365, seguimiento
4. Día 28, día 92, día 183, día 274, día 365, seguimiento
5. Línea de base, día 28, día 92, día 183, día 274, día 365
6-8. Hasta el día 395
9-11. Línea de base al día 395
12-15. Hasta el día 395
16-17. Día 1, Día 2, Día 14, Día 92, Día 183, Día 274, Día 365
18. Visita basal, día 1, día 2, día 14, día 183, día 365.

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

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

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

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

Yes

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

Australia

China

Russian Federation

Italy

Poland

Portugal

Spain

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

A participant is considered to have completed the study if he/she has completed all phases of the study, including the last scheduled procedure per SoA or withdrawal of consent.
The end of the study is defined as the date when the last participant’s last observation occurs.
All participants who complete the 52 weeks of study treatment will be offered the option to rollover into an open-label extension study for continued drug access (separate protocol).

un participante ha completado el estudio si ha completado todas las fases del estudio,incluido el último procedimiento programado por SoA o la retirada del consentimiento.El final del estudio se define como la fecha en que se produce la última observación del último participante.
A todos los participantes que completen las 52 semanas de tto del estudio se les ofrecerá la opción de pasar a un estudio de extensión de etiqueta abierta para el acceso continuo a los medicamentos (protocolo separado)

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 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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

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

Participants aged 2 to 11 years inclusive at the time the caregiver signs the informed consent.

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)

All participants who complete the 52 weeks of study treatment will be offered the option to roll over into an open-label extension study for continued drug access under a separate study protocol.
A participant will not be eligible to receive study treatment after the end of the study if any of the conditions outlined in the study protocol are not met.

A todos los participantes que completen las 52 semanas de tratamiento del estudio se les ofrecerá la opción de pasar a un estudio de extensión de etiqueta abierta para el acceso continuo a los medicamentos bajo un protocolo de estudio separado.
Un participante no será elegible para recibir el tratamiento del estudio una vez finalizado el estudio si no se cumple alguna de las condiciones descritas en el protocolo del estudio.

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

2022-03-31

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-03-25

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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