Clinical Trials Register

Summary
EudraCT Number:	2021-003453-28
Sponsor's Protocol Code Number:	SOMCT03
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:	2021-12-30
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

2021-003453-28

A.3

Full title of the trial

Phase IIb, randomized, double-blind, placebo-controlled study in parallel groups assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington’s Disease with choreic movements.

Estudio de fase IIb, aleatorizado, doble ciego y controlado con placebo en grupos paralelos para evaluar la eficacia y la seguridad de dos dosis de SOM3355 en pacientes con enfermedad de Huntington con movimientos coreicos.

A.3.1

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

Phase IIb study assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington’s Disease with choreic movements.

Estudio de Fase IIb para evaluar la eficacia y la seguridad de dos dosis de SOM3355 en pacientes con Enfermedad de Huntington con movimientos coreicos.

A.4.1

Sponsor's protocol code number

SOMCT03

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	SOM Innovation Biotech SA (SOM Biotech)
B.1.3.4	Country	Spain
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	SOM Innovation Biotech SA (SOM Biotech)
B.4.2	Country	Spain
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	SOM Innovation Biotech SA (SOM Biotech)
B.5.2	Functional name of contact point	Clinical Trial Information Desk
B.5.3	Address:
B.5.3.1	Street Address	Baldiri Reixac, 4
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08028
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34934 020 150
B.5.6	E-mail	info@sombiotech.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	Calvan (bevantolol hydrochloride)
D.2.1.1.2	Name of the Marketing Authorisation holder	Nippon Chemiphar
D.2.1.2	Country which granted the Marketing Authorisation	Japan
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	SOM3355 (bevantolol hydrochloride)
D.3.2	Product code	SOM3355
D.3.4	Pharmaceutical form	Capsule
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	BEVANTOLOL HYDROCHLORIDE
D.3.9.1	CAS number	59170-23-9
D.3.9.2	Current sponsor code	SOM3355
D.3.9.4	EV Substance Code	SUB00793MIG
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	200
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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	Calvan (bevantolol hydrochloride)
D.2.1.1.2	Name of the Marketing Authorisation holder	Nippon Chemiphar
D.2.1.2	Country which granted the Marketing Authorisation	Japan
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	SOM3355 (bevantolol hydrochloride)
D.3.2	Product code	SOM3355
D.3.4	Pharmaceutical form	Capsule
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	BEVANTOLOL HYDROCHLORIDE
D.3.9.1	CAS number	59170-23-9
D.3.9.4	EV Substance Code	SUB00793MIG
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	300
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	Capsule
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

Huntington’s Disease with choreic movements.

Enfermedad de Huntington con movimientos coreicos.

E.1.1.1

Medical condition in easily understood language

Huntington’s Disease with choreic movements.

Enfermedad de Huntington con movimientos coreicos.

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

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	10020469
E.1.2	Term	Huntington's chorea
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

The primary objective of the study is to assess the efficacy of two doses of SOM3355 (400 mg/day and 600 mg/day taken twice daily [BID] over at least 8 weeks at maintenance dose) compared to placebo to reduce chorea in HD patients measured by the change in TMC score (primary efficacy endpoint).

El objetivo principal del estudio es evaluar la eficacia de dos dosis de SOM3355 (400 mg/día y 600 mg/día administrados dos veces al día [2 v/d] durante al menos 8 semanas a la dosis de mantenimiento) en comparación con placebo para reducir la corea en pacientes con EH, medida por el cambio en la puntuación de TMC (criterio principal de valoración de la eficacia).

E.2.2

Secondary objectives of the trial

To evaluate the safety and tolerability of two doses of SOM3355 (400 mg/day and 600 mg/day taken) compared with placebo in HD patients, with particular attention to depression and suicidality and to the cardiovascular hemodynamic parameters.
And to evaluate the efficacy of the two doses of SOM3355 (400 mg/day and 600 mg/day taken BID) compared with placebo on:
the change of Clinical Global Impression (CGI C),
the change of Patient Global Impression (PGI-C),
the percentage of responders based on the improvement ≥2 in TMC score,
the percentage of change of TMC score.

Evaluar la seguridad y tolerabilidad de dos dosis de SOM3355 (400 mg/día y 600 mg/día) comparado con placebo en pacientes con EH, prestando especial atención a la depresión y a las tendencias suicidas y a los parámetros hemodinámicos cardiovasculares. Y evaluar la eficacia de las dos dosis de SOM3355 (400 mg/día y 600 mg/día dos veces al día) en comparación con placebo en:
el cambio de la Impresión clínica global (Clinical Global Impression, CGI-C),
el cambio de la Impresión global del paciente (Patient Global Impression, PGI-C),
el porcentaje de pacientes con respuesta en función de la mejoría ≥2 en la puntuación de TMC,
el porcentaje de cambio de la puntuación de TMC.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

A PK sub-study will be conducted to characterize the PK profile of repeated doses of SOM3355 at 400 and 600 mg/day. This analysis will be performed by collecting 12-hour PK samples (H0, H0.5, H1, H1.5, H2, H3, H5, H8, and H12) in 8 patients per arm (24 patients in total) that will be hospitalized for 24 hours at Visit 2. In addition, cardiac and hemodynamic parameters will be collected in parallel, with 12-hour cardiac Holter and blood pressure measurements before each PK sample, to allow PK-PD modeling assessments. These patients will be recruited by 3 or 4 sites able to manage the logistics of PK sampling.

Se realizará un subestudio de PK para caracterizar el perfil de PK de dosis repetidas de SOM3355 a 400 y 600 mg/día. Este análisis se realizará recogiendo muestras de PK de 12 horas (H0, H0,5, H1, H1,5, H2, H3, H5, H8 y H12) en 8 pacientes por brazo (24 pacientes en total) que serán hospitalizados durante 24 horas en la Visita 2. Además, se recogerán parámetros cardíacos y hemodinámicos en paralelo, con Holter cardíaco de 12 horas y mediciones de la presión arterial antes de cada muestra de PK, para permitir las evaluaciones de modelado PK-PD. Estos pacientes serán reclutados por 3 o 4 centros capaces de gestionar la logística de la toma de muestras PK.

E.3

Principal inclusion criteria

1.Males or females ≥21 years old.
2.Patients with a diagnosis of Huntington’s Disease determined by a movement disorders expert and confirmed by a number of HTT gene cytosine-adenosine-guanine (CAG) repeats ≥36.
3.UHDRS Total maximal chorea (TMC) score ≥10.
4.UHDRS Total Functional Capacity (TFC) ≥7 (corresponding to mildly to moderately impaired patients).
5.Able to walk independently or with minimal assistance.
6.Females of child-bearing potential must use a medically accepted effective method of birth control, agree to continue this method for the duration of the study, and be negative to serum pregnancy test performed at the screening visit. Female patients should not be breast-feeding.
7.In the opinion of the Investigator, the patient must have adequate support to comply with the entire study requirements as described in this protocol (e.g. transportation to and from the trial site, self-rating scales, drug compliance, scheduled visits, etc.).
8.Able and willing to provide written informed consent prior to any study-related procedure being performed at the screening visit.

Los criterios de elegibilidad para participar en este estudio son:
1.Hombres o mujeres ≥21 años.
2.Pacientes con un diagnóstico de enfermedad de Huntington determinada por un experto en trastornos del movimiento y confirmado por una serie de detección del número de repeticiones del trinucleótido citosina-adenosina-guanina (CAG) en el gen HTT ≥36.
3.Puntuación de corea máxima total (TMC) de UHDRS® ≥10.
4.Capacidad funcional total (Total Functional Capacity, TFC) de UHDRS® ≥7 (correspondiente a pacientes con deterioro leve o moderado).
5.Capacidad para caminar de forma independiente o con una asistencia mínima.
6.Las mujeres con capacidad de procrear deben utilizar un método anticonceptivo eficaz médicamente aceptado, aceptar continuar con este método durante todo el estudio y tener un resultado negativo en la prueba de embarazo en suero realizada en la visita de selección. Las mujeres no deben estar en periodo de lactancia.
7.En opinión del investigador, el paciente debe tener apoyo adecuado para cumplir todos los requisitos del estudio según se describe en este protocolo (p. ej., transporte hasta y desde el centro del estudio clínico, escalas de autoevaluación, cumplimiento terapéutico, visitas programadas, etc.).
8.Capacidad y voluntad de proporcionar el consentimiento informado por escrito antes de que se realice cualquier procedimiento relacionado con el estudio en la visita de selección.

E.4

Principal exclusion criteria

1.Onset of HD symptoms prior to age of 21 years, corresponding to juvenile forms of HD.
2.HD patients presenting rigid akinesia.
3.Use of other vesicular monoamine transporter type 2 (VMAT2) inhibitors such as tetrabenazine, deutetrabenazine, or valbenazine within 3 months before enrollment, and at any time during the study period; and use of other antichoreic treatment such as any neuroleptic within 2 months or amantadine, memantine, riluzole within 1 month before enrollment and along the study.
4.Patients who experienced severe depression or suicide attempt in the last 5 years.
5.Severe untreated or under-treated psychiatric illness such as active suicidal ideation or behavior (BDI-21 item #9 >0 and active suicidal ideation in C-SSRS) or depression at screening and/or initiation visit (BDI-21 items total score >30); although patients taking authorized antidepressant therapy at a stable dose for at least 2 months and stabilized can be enrolled.
6.Patients with a history of, or current, hypotension (SBP <110 mmHg), bradycardia (HR <50 bpm), or orthostatic hypotension as defined by the European Society of Hypertension (reduction in SBP ≥20 mmHg or in DBP ≥10 mmHg).
7.Patients with hypertension already treated with more than 2 antihypertensive drugs.
8.Other active clinically significant illness, including unstable cardiovascular disease, angina pectoris, congestive heart failure, pulmonary hypertension, peripheral arterial disease, history of pheochromocytoma, asthma, COPD, diabetic ketoacidosis or metabolic acidosis, or neoplastic pathology, which could interfere with the study conduct, or counter-indicate the study treatment, or to place the patient at risk during the trial, or compromise their study participation.
9.Any significant serious abnormality in the electrocardiogram (ECG), e.g. recent myocardial infarction, significant sinus bradycardia (<50 bpm), atrioventricular block (grades I to III with PR >240msec), sinoatrial block, atrial sinus disease or prolonged QTc interval at screening (ECG Bazett’s corrected QT interval (QT /√ [HR/60] >450 msec for males or >470 msec for females), or a known history of long QTc syndrome.
10.Patients with severe hepatic impairment, or with severe renal impairment, or with any other significant abnormality in the physical examination or clinical laboratory results that, in the Investigator’s opinion, would not be compatible with study participation or represent a risk for the patient while in the study.
11.Females who are pregnant or lactating, or who intend to become pregnant during the study period.
12.Patients with allergy under desensitization, with known psoriasis, or a known allergy / hypersensitivity to any ingredients of the trial medication or placebo.
13.History of alcohol or substance abuse in the previous 12 months.
14.Patients participating in any other study, and the use of any investigational therapy, within 1 month prior to entry in this study.

1.Aparición de los síntomas de EH antes de los 21 años, lo que corresponde a las formas juveniles de EH.
2.Pacientes con EH que presentan acinesia con rigidez.
3.Uso de otros inhibidores del transportador de la monoamina vesicular tipo 2 (VMAT2) como tetrabenazina, deutetrabenazina o valbenazina en los 3 meses previos a la inclusión y en cualquier momento durante el periodo del estudio; y uso de otro tratamiento anticoreico como cualquier neuroléptico en los 2 meses previos o amantadina, memantina, riluzol en el mes previo a la inclusión y a lo largo del estudio.
4.Pacientes que hayan experimentado depresión grave o intento de suicidio en los últimos 5 años.
5.Enfermedad psiquiátrica grave no tratada o infratratada, como ideación o conducta suicida activa (ítem n.º 9 de BDI-21 >0 e ideación suicida activa en la escala C-SSRS) o depresión en la visita de selección y/o inicial (puntuación total de los ítems de BDI-21 >30); aunque los pacientes que toman tratamiento antidepresivo autorizado a una dosis estable durante al menos 2 meses y están estabilizados se pueden incluir.
6.Pacientes con antecedentes o presencia actual de hipotensión (PAS <110 mmHg), bradicardia (FC <50 lpm) o hipotensión ortostática definida por la Sociedad Europea de Hipertensión (reducción de la PAS ≥20 mmHg o de la PAD ≥10 mmHg).
7.Pacientes con hipertensión ya tratados con más de 2 antihipertensivos.
8.Otra enfermedad activa clínicamente significativa, como enfermedad cardiovascular inestable, angina de pecho, insuficiencia cardíaca congestiva, hipertensión pulmonar, arteriopatía periférica, antecedentes de feocromocitoma, asma, EPOC, cetoacidosis diabética o acidosis metabólica, o patología neoplásica, que podría interferir en la realización del estudio, contraindicar el tratamiento del estudio, poner en riesgo al paciente durante el ensayo o comprometer su participación en el mismo.
9.Cualquier anomalía grave significativa en el electrocardiograma (ECG), p. ej., infarto de miocardio reciente, bradicardia sinusal significativa (<50 lpm), bloqueo auriculoventricular (grados I a III con PR >240 ms), bloqueo sinoauricular, enfermedad sinusal auricular o intervalo QTc prolongado en la selección (intervalo QT corregido según Bazett en el ECG (QT/√[FC/60] >450 ms en varones o >470 ms en mujeres) o antecedentes conocidos de síndrome QTc prolongado.
10.Pacientes con insuficiencia hepática grave, o con disfunción renal grave, o con cualquier otra anomalía importante en la exploración física o los resultados analíticos clínicos que, según el criterio del investigador, no fuera compatible con la participación en el estudio o que represente un riesgo para el paciente mientras participa en el estudio.
11.Mujeres embarazadas o en periodo de lactancia, o que tengan previsto quedarse embarazadas durante el periodo del estudio.
12.Pacientes con alergia en desensibilización, con psoriasis conocida o una alergia/hipersensibilidad conocida a cualquier componente de la medicación del ensayo o placebo.
13.Antecedentes de alcoholismo o abuso de sustancias en los 12 meses anteriores.
14.Pacientes que participen en cualquier otro estudio y el uso de cualquier tratamiento en investigación en el plazo de 1 mes antes de la inclusión en este estudio.

E.5 End points

E.5.1

Primary end point(s)

Change in Total Maximal Chorea (TMC) sub-score of the Unified Huntington's Disease Rating Scale (UHDRS)

Cambio en la subpuntuación de corea máxima total (TMC) de la Escala unificada de valoración de la enfermedad de Huntington (UHDRS®)

E.5.1.1

Timepoint(s) of evaluation of this end point

Between baseline and end of maintenance dose

Entre la línea de base y el final de la dosis de mantenimiento

E.5.2

Secondary end point(s)

•Change from baseline in the Clinical Global Impression (CGI-C).
•Change from baseline in the Patient Global Impression (PGI-C).
•TMC-response defined as improvement ≥2 in TMC score between baseline and end of maintenance dose.
•Percentage of change in TMC score between baseline and the end of maintenance dose.
•Change from baseline in Total Motor Score (TMS) of the UHDRS.
•Change from baseline in Gait sub-score of the UHDRS.
•Change from baseline in Dystonia sub-score of the UHDRS.
•Change from baseline in EQ5D-5L measuring quality of life.

•Cambio respecto al inicio en la Impresión clínica global (CGI-C).
•Cambio respecto al inicio en la Impresión global del paciente (PGI-C).
•La respuesta de TMC se define como la mejoría ≥2 en la puntuación del TMC entre el inicio y el final de la dosis de mantenimiento.
•Porcentaje de cambio en la puntuación de TMC entre el inicio y el final de la dosis de mantenimiento.
•Cambio con respecto al inicio en la puntuación motora total (TMS) de la UHDRS®.
•Cambio con respecto al inicio en la subpuntuación de marcha de la UHDRS®.
•Cambio con respecto al inicio en la subpuntuación de distonía de la UHDRS®.
•Cambio con respecto al inicio en la medición de la calidad de vida del EQ5D-5L.

E.5.2.1

Timepoint(s) of evaluation of this end point

Between baseline and end of maintenance dose

Entre la línea de base y el final de la dosis de mantenimiento

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

Germany

Italy

Poland

Spain

Switzerland

United Kingdom

France

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LPLV

Ultimo paciente ultima visita

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

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

120

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

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

105

F.4.2.2

In the whole clinical trial

129

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)

Yes, an OLE study

Si, un estudio OLE

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	European Huntington’s Disease Network (EHDN)
G.4.3.4	Network Country	Germany

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-03-28

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-03-23

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

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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