Clinical Trials Register

Summary
EudraCT Number:	2022-002301-24
Sponsor's Protocol Code Number:	NMD670-02-0001
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:	2023-01-12
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

2022-002301-24

A.3

Full title of the trial

A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of
NMD670 in ambulatory adults with Type 3 spinal muscular atrophy

Estudio de fase 2, aleatorizado, en doble ciego, controlado con placebo y cruzado de dos vías, para evaluar la eficacia, seguridad y tolerabilidad de NMD670 en adultos deambulantes con atrofia espinal muscular de tipo 3

A.3.1

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

A study to evaluate the efficacy, safety, and tolerability of oral NMD670 compared with placebo in ambulatory adults with spinal muscular atrophy

Estudio para evaluar la eficacia, seguridad y tolerabilidad de NMD670 oral en comparación con placebo en adultos deambulantes con atrofia espinal muscular

A.4.1

Sponsor's protocol code number

NMD670-02-0001

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	NMD Pharma A/S
B.1.3.4	Country	Denmark
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	NMD Pharma A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Parexel International
B.5.2	Functional name of contact point	Clinical Trials Department
B.5.3	Address:
B.5.3.1	Street Address	70 Sir John Rogerson's Quay
B.5.3.2	Town/ city	Dublin
B.5.3.3	Post code	2
B.5.3.4	Country	Ireland
B.5.4	Telephone number	+32 10 817-132
B.5.6	E-mail	clinicaltrial.enquiries@parexel.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.3.2	Product code	NMD670
D.3.4	Pharmaceutical form	Film-coated tablet
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	N/A
D.3.9.1	CAS number	2354321-33-6
D.3.9.2	Current sponsor code	NMD670
D.3.9.3	Other descriptive name	NMD670
D.3.9.4	EV Substance Code	SUB208279
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	100
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	NMD670
D.3.4	Pharmaceutical form	Film-coated tablet
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	N/A
D.3.9.1	CAS number	2354321-33-6
D.3.9.2	Current sponsor code	NMD670
D.3.9.3	Other descriptive name	NMD670
D.3.9.4	EV Substance Code	SUB208279
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	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
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

Type 3 spinal muscular atrophy

Atrofia espinal muscular de tipo 3

E.1.1.1

Medical condition in easily understood language

spinal muscular atrophy - an inherited condition that makes the muscles weaker and causes problems with movement.

atrofia muscular espinal - una condición hereditaria que debilita los músculos y causa problemas con el movimiento.

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

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	10079415
E.1.2	Term	Spinal muscular atrophy type III
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 assess changes in muscle strength and function after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA

Evaluar los cambios en la fuerza y función musculares tras la administración de NMD670 400 mg bid (2 veces al día) durante 21 días, en comparación con placebo, en participantes con SMA

E.2.2

Secondary objectives of the trial

- To assess changes in muscle strength after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To further assess changes in muscle strength and function after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To assess changes in neuromuscular junction transmission after NDM670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To assess the safety and tolerability of NMD670, compared with placebo, over 21-day dosing, in participants with SMA

Exploratory objectives:
- To assess changes in patient-reported quality of life and fatigue severity after 21-day dosing of NMD670, compared with placebo, in participants with SMA
- To explore drug exposure after NMD670 400 mg bid for 21 days in participants with SMA

- Evaluar cambios en la fuerza muscular tras la administración de NMD670 400 mg bid durante 21 días, en comparación con placebo, en participantes con SMA
- Evaluar adicionalmente cambios en la fuerza y función musculares tras la administración de NMD670 400 mg bid durante 21 días, comparado con placebo, en participantes con SMA
- Evaluar cambios en la transmisión en la unión neuromuscular tras la administración de NDM670 400 mg bid durante 21 días, comparado con placebo, en participantes con SMA
- Evaluar la seguridad y la tolerabilidad de NMD670, en comparación con placebo, a lo largo de su administración durante 21 días, en participantes con SMA
Objetivos exploratorios:
- Evaluar cambios en la calidad de vida y la severidad de la fatiga comunicados por el paciente tras 21 días de tratamiento con NMD670, en comparación con placebo, en participantes con SMA
- Explorar la exposición al fármaco tras la administración de NMD670 400 mg bid durante 21 días en participantes con SMA

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Participant must be 18 to 60 years of age inclusive, at the time of signing the informed consent.
2. Participants who are with a clinical diagnosis of Type 3 SMA.
3. Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids.
4. Participant with genetic confirmation of diagnosis (i.e., homozygous deletion of survival of motor neuron 1 gene [SMN1]).
5. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
6. Participants with a MFM-32 dimension 1 score <80% at screening.
7. Participants with >/=7% CMAP amplitude decrement at screening (recorded from the trapezius muscle during repeated nerve stimulation [RNS]).
8. Participant has a body mass index (BMI) within the range 19-30 kg/m2 (inclusive).
9. Participant is male or female.
10. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Male Participants:
• A male participant must agree to use a highly effective contraception as detailed in Appendix 4 of this protocol during the intervention period and for at least 14 days after the last dose of study intervention (corresponding to time needed to eliminate study intervention) and refrain from donating sperm during this period.
Female Participants:
• A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies:
o Not a woman of childbearing potential (WOCBP) as defined in Appendix 4.
OR
o A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the intervention period and for at least 14 days after the last dose of study intervention (corresponding to time needed to eliminate study intervention).
11. Participant is capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

1. Edad entre 18 y 60 años, ambos inclusive, en el momento de firmar el consentimiento informado.
2. Diagnóstico clínico de atrofia muscular espinal (SMA, spinal muscular atrophy) de tipo 3.
3. Deambulación, definida como la capacidad de andar un mínimo de 50 metros sin ayuda para caminar.
4. Confirmación genética del diagnóstico (a saber, pérdida homocigótica del gen de supervivencia de la neurona motora 1 [SMN1, survival of motor neuron 1]).
5. De 3 a 5 copias del gen de supervivencia de la neurona motora 2 [SMN2, survival of motor neuron 2].
6. Puntuación de la dimensión 1 en la Medida de la Función Motora (MFM-32, Motor Function Mesure) <80% en la selección.
7. Disminución de la amplitud del potencial de acción muscular compuesto (CMAP, compound muscle action potencial) >/=7%, en la selección (registrada en el trapecio, durante la estimulación nerviosa repetida [RNS, repeated nerve stimulation]).
8. Índice de masa muscular (BMI, body mass index) de 19-30 kg/m2 (ambos inclusive).
9. Pacientes de ambos sexos.
10. El uso de anticonceptivos por los varones y las mujeres debe estar de acuerdo con la legislación local en cuanto a métodos anticonceptivos en los participantes en estudios clínicos.
Varones: • El participante debe estar de acuerdo en utilizar un anticonceptivo de alta eficacia, tal como se detalla en el Apéndice 4 de este protocolo, durante el periodo de tratamiento y durante al menos 14 días después de la última dosis del tratamiento del estudio (tiempo que corresponde a la eliminación del medicamento del estudio) y abstenerse de donar semen durante este mismo periodo.
Mujeres: • Una mujer es elegible para participar si no está embarazada (véase el Apéndice 4), no está en fase de lactancia y cumple una o más de las siguientes condiciones:
. No es una mujer potencialmente fértil (WOCBP, woman of childbearing potential), según la definición en el Apéndice 4.
O BIEN
. Es una mujer potencialmente fértil pero está de acuerdo en seguir las normas de anticoncepción del Apéndice 4, durante el periodo de tratamiento y durante al menos 14 días después de la última dosis del tratamiento del estudio (tiempo que corresponde a la eliminación del medicamento del estudio).
11. Capacidad de otorgar y firmar el consentimiento informado, tal como se describe en el Apéndice 1, Sección 10.1.3, lo que incluye el cumplimiento de los requisitos y limitaciones que figuran en el documento de consentimiento informado (ICF, informed consent form) y en este protocolo.

E.4

Principal exclusion criteria

1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
3. Participant with a clinical diagnosis of gout, or with serum uric acid >6.5 mg/dL at screening.
4. Participant with clinically significant ECG abnormalities at screening including PR interval >/=220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare supraventricular or rare ventricular ectopic beats) in the judgement of the Investigator, or T-wave configurations are not of sufficient quality for assessing QT interval duration.
5. Participant with any of the following abnormalities in QT interval corrected for heart rate using Fridericia’s correction (QTcF) at screening:
a. QTcF >450 msec for male participants without bundle branch block.
b. QTcF >470 msec for female participants without bundle branch block.
c. QTcF >480 msec in participants with bundle branch block.
6. Participant with any of the following:
a. Abnormal liver function test defined as total bilirubin >1.5×ULN (participants with Gilbert's syndrome can be included with total bilirubin >1.5×ULN if direct bilirubin is < = 1.5×ULN and < = 35% of total bilirubin).
b. Current or chronic history of liver disease including (but is not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non-alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson's disease, alpha-1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease
considered clinically significant by the Investigator.
c. Known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).
7. Participant with clinically significant laboratory test abnormalities at screening.
8. Participant with breast cancer within the past 10 years or lymphoma, leukaemia, or any malignancy within the past 5 years. An exception of this 5-year requirement is basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 5 years.
9. Participants with ongoing significant psychiatric disorder (e.g., uncontrolled depression, anxiety).
10. Participants with positive drug screen (cocaine, heroin, ketamine [unless medically prescribed], opiates) at screening.
11. Participants with positive human immunodeficiency virus (HIV) antibody test.
12. Participants with presence of hepatitis B surface antigen (HBsAg) [or hepatitis B core antibody (HBcAb)] at screening or within 3 months prior to first dose of investigational intervention.
13. Participants with positive hepatitis C antibody or ribonucleic acid (RNA) test result at screening or within 3 months prior to Day 1.

1. Cirugía o deformidad fija (escoliosis, contracturas) previa, que pudiera limitar la capacidad para realizar las tareas relacionadas con el estudio.
2. Otra enfermedad importante que pudiera interferir en la interpretación de los datos del estudio (por ejemplo, otras enfermedades neuromusculares o musculares).
3. Diagnóstico clínico de gota, o ácido úrico en suero >6,5 mg/dl, en la selección.
4. Anomalías del ECG clínicamente significativas, tales como intervalo PR >/= 220 ms, ritmos irregulares (distintos de arritmia sinusal o extrasístoles supraventriculares o ventriculares aisladas ocasionales), según criterio del Investigador, o forma de la onda T de calidad insuficiente para valorar la duración del intervalo QT, en la selección.
5. Cualquiera de las siguientes anomalías en el intervalo QT corregido según la frecuencia cardiaca mediante corrección de Fridericia (QTcF), en la selección:
a. QTcF >450 ms en varones sin bloqueo de rama.
b. QTcF >470 ms en mujeres sin bloqueo de rama.
c. QTcF >480 ms en participantes con bloqueo de rama.
6. Presencia de alguna de las siguientes anomalías o afecciones:
a. Prueba de función hepática anómala, definida como bilirrubina total >1,5×límite superior de la normalidad (ULN, upper limit of normal) (se podrán incluir participantes con síndrome de Gilbert con bilirrubina total >1,5×ULN si la bilirrubina directa es < = 1,5×ULN y < = 35% de la bilirrubina total).
b. Hepatopatía actual o antecedentes de hepatopatía crónica, incluidas (entre otras) infecciones por virus de la hepatitis, hepatopatía por consumo de drogas o alcohol, esteatohepatitis no alcohólica, hepatitis autoinmunitaria, hemocromatosis, enfermedad de Wilson, déficit de alpha-1 antitripsina, colangitis biliar primaria, colangitis esclerosante primaria o cualquier otra hepatopatía que el Investigador considere clínicamente importante.
c. Anomalías hepáticas o biliares conocidas (con excepción del síndrome de Gilbert o cálculos biliares asintomáticos).
7. Anomalías de laboratorio clínicamente importantes, en la selección.
8. Cáncer de mama en los 10 últimos años, o linfoma, leucemia o neoplasia maligna en los 5 últimos años. Una excepción a este requisito de 5 años es el carcinoma cutáneo de células basales o de células escamosas que se haya extirpado y no presente evidencia de metástasis en 5 años.
9. Trastorno psiquiátrico importante en curso (por ejemplo, depresión o ansiedad no controladas).
10. Positividad en el control de drogas (cocaína, heroína, ketamina [salvo por prescripción médica], opiáceos), en la selección.
11. Positividad en la prueba de anticuerpos contra el virus de la inmunodeficiencia humana (HIV, human immunodeficiency virus).
12. Presencia del antígeno de superficie del virus de la hepatitis B (HBsAg, hepatitis B surface antigen) [o anticuerpo contra el antígeno central del virus de la hepatitis B (HBcAb, hepatitis B core antibody)], en la selección o en los 3 meses anteriores a la primera dosis del tratamiento en investigación.
13. Positividad en la prueba de anticuerpos o de ácido ribonucleico (RNA, ribonucleic acid) del virus de la hepatitis C, en la selección o en los 3 meses anteriores al Día 1.

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in 6MWT (total distance) after 21-day dosing

Cambio frente al basal en la 6MWT (distancia total) tras 21 días de tratamiento

E.5.1.1

Timepoint(s) of evaluation of this end point

After 21 day dosing

Tras 21 días de tratamiento

E.5.2

Secondary end point(s)

Change from baseline in individual muscle groups maximum strength (measured with a dynamometer) after 21-day dosing
Change from baseline after 21-day dosing in:
- 6MWT (fatigue index)
- revised Hammersmith scale score
- ESNHP
Change from baseline in jitter and blocking measured via sfEMG after 21-day dosing
AEs, physical examinations, clinical laboratory parameters, vital signs, ECG, C-SSRS

Exploratory:
Change from baseline after 21-day dosing in:
- INQoL score
- FSS
Plasma levels of NMD670 and its metabolite NMD1190

Cambio frente al basal en la fuerza máxima de determinados grupos musculares (medida mediante dinamómetro) tras 21 días de tratamiento
Cambio frente al basal tras 21 días de tratamiento en:
- 6MWT (índice de fatiga)
- puntuación de la escala Hammersmith revisada
- ESNHP
Cambio frente al basal en el jitter y el bloqueo en su medición mediante sfEMG tras 21 días de tratamiento
AEs, exploraciones físicas, determinaciones de laboratorio, constantes vitales, ECG, C SSRS

Exploratorios:
Cambio frente al basal tras 21 días de tratamiento en:
- Puntuación de INQoL
- FSS
Niveles plasmáticos de NMD670 y de su metabolito NMD1190

E.5.2.1

Timepoint(s) of evaluation of this end point

After 21 day dosing
During study participation

Tras 21 días de tratamiento
Durante la participación en el estudio

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)

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

E.8.1.6

Cross over

Yes

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

Canada

United States

Netherlands

Spain

Germany

Italy

Belgium

Denmark

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

Última visita del último paciente

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

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:

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

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)

Standard of care

Tratamiento estándar

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

2023-09-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

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

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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