Clinical Trials Register

Summary
EudraCT Number:	2020-003376-40
Sponsor's Protocol Code Number:	FAB122-CT-2001
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:	2021-06-23
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

2020-003376-40

A.3

Full title of the trial

A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis

Estudio multicéntrico, aleatorizado, doble ciego y controlado con placebo para investigar la eficacia y seguridad de FAB122 en pacientes con esclerosis lateral amiotrófica

A.3.1

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

A study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis

Un estudio para investigar la eficacia y seguridad de FAB122 en pacientes con esclerosis lateral amiotrófica

A.3.2

Name or abbreviated title of the trial where available

ADORE (ALS Deceleration with ORal Edaravone) study

Estudio ADORE (desaceleración ALS con Edaravone oral)

A.4.1

Sponsor's protocol code number

FAB122-CT-2001

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	Ferrer Internacional, S.A.
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	Ferrer Internacional, S.A.
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	Julius Clinical
B.5.2	Functional name of contact point	Project Manager
B.5.3	Address:
B.5.3.1	Street Address	Broederplein 41-43
B.5.3.2	Town/ city	Zeist
B.5.3.3	Post code	3703 CD
B.5.3.4	Country	Netherlands
B.5.6	E-mail	regulatory@juliusclinical.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EMA/OD/184/14
D.3 Description of the IMP
D.3.1	Product name	Edaravone
D.3.2	Product code	FAB122
D.3.4	Pharmaceutical form	Granules for oral solution in sachet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	EDARAVONE
D.3.9.1	CAS number	89-25-8
D.3.9.2	Current sponsor code	FAB122
D.3.9.4	EV Substance Code	SUB06453MIG
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.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 for oral solution 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

Amyotrophic Lateral Sclerosis

Esclerosis lateral amiotrófica

E.1.1.1

Medical condition in easily understood language

ALS

ELA

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	21.1
E.1.2	Level	PT
E.1.2	Classification code	10002026
E.1.2	Term	Amyotrophic lateral sclerosis
E.1.2	System Organ Class	10029205 - Nervous system 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 assess the effect of treatment with 100 mg of FAB122 on disease progression in patients with ALS

Evaluar el efecto del tratamiento con 100 mg de FAB122 en la evolución de la enfermedad en pacientes con ELA.

E.2.2

Secondary objectives of the trial

1. To evaluate the effect of treatment with FAB122 on survival
2. To evaluate the safety and tolerability of FAB122
3. To evaluate the effect of treatment with FAB122 on quality of life (QoL)
4. To evaluate the effect of treatment with FAB122 on cognitive functioning
5. To evaluate the pharmacokinetics (PK) of FAB122

1. Evaluar el efecto del tratamiento con FAB122 en la supervivencia.
2. Evaluar la seguridad y tolerabilidad de FAB122.
3. Evaluar el efecto del tratamiento con FAB122 en la calidad de vida.
4. Evaluar el efecto del tratamiento con FAB122 en el funcionamiento cognitivo.
5. Para evaluar la farmacocinética (PK) de FAB122.

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

There will be an optional PK sub-study, included in the main protocol. The objective is to evaluate the pharmacokinetics (PK) of FAB122.

Habrá un sub- estudio PK opcional, incluido en el protocolo principal. El objetivo es evaluar la farmacocinética (PK) de FAB122.

E.3

Principal inclusion criteria

1. Age 18 – 80 years (both inclusive), male or female;
2. Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS;
3. Onset of first symptoms* no longer than 24 months prior to randomization;
*Date of onset is the date the patient reported one or more of the following symptoms: Muscle weakness in limbs; speech/swallowing difficulties; respiratory symptoms: dyspnea was noticed
4. SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit;
5. Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;
6. Patients on riluzole should be on stable doses ≥30 days prior to the baseline visit and this dose should be maintained during the entire trial.
7. A female subject should not be able to become pregnant and needs to meet at least one of the following criteria:
• female subject who is not of reproductive potential is eligible without requiring the use of contraception. A female subject who is not of reproductive potential is defined as one who: (1) has reached natural menopause (defined as 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone [FSH] levels in the postmenopausal range as determined prior to study initiation and reported in patient’s medical records , or 12 months of spontaneous amenorrhea); (2) is 6 weeks post-surgical bilateral oophorectomy with or without hysterectomy; or; (3) has undergone bilateral tubal ligation. Spontaneous amenorrhea does not include cases for which there is an underlying disease that causes amenorrhea (e.g. anorexia nervosa) • female who is of reproductive potential and has a negative pregnancy test at screening and at baseline and is non-lactating. A female subject who is of reproductive potential agrees to true abstinence or to use (or have their partner use) or practicing adequate birth control methods starting from the time of consent through 30 days after the last dose of study therapy. Longer periods of birth control maybe required per local requirements. Acceptable methods of birth control include hormonal contraception (oral, implanted, injected or other hormonal (e.g., patch or contraceptive ring) contraception), intrauterine device in place for ≥3 months, barrier method in conjunction with spermicide OR use of appropriate double barrier contraception as per local regulations or guidelines;
8. A male patient must: • agree he will not donate sperm during the study and until 104 days after the last dose, AND • use a condom during sexual intercourse with pregnant or non-pregnant women of childbearing potential (WOCBP) partner even if he is vasectomized • in addition WOCBP partner of the male patient must use the following acceptable methods of birth control during the study and until 104 days after the last dose: hormonal contraception (oral, implanted, injected or other hormonal (e.g., patch or contraceptive ring) contraception), intrauterine device in place for ≥3 months, barrier method in conjunction with spermicide OR use of appropriate double barrier contraception as per local regulations or guidelines;
9. Capable of providing informed consent and complying with trial procedures.

1. Edad entre 18 y 80 años (ambos inclusive), hombre o mujer.
2. Diagnóstico de ELA definitivo, probable, probable con respaldo de laboratorio o posible, según los criterios de diagnóstico de la ELA de El Escorial y los revisados por Airlie House.
3. Inicio de los primeros síntomas* no más de 24 meses antes de la aleatorización.
*La fecha de inicio es la fecha en que el paciente informó de uno o más de los siguientes síntomas:
 Debilidad muscular en las extremidades.
 Dificultades para hablar/tragar.
 Síntomas respiratorios: notó disnea.
4. CVL igual o superior al 70 % del valor normal previsto para el sexo, la altura y la edad en la visita de cribado.
5. Cambios en la puntuación de la ALSFRS-R entre 0,35 puntos y 1,5 puntos por mes (ambos inclusive) en el período comprendido entre el inicio de los primeros síntomas y la visita de cribado.
6. Los pacientes que tomen riluzol deben estar tomando dosis estables ≥30 días antes de la visita inicial, además, esta dosis debe mantenerse durante todo el ensayo.
7. El sujeto mujer no debe ser capaz de quedarse embarazada y tiene que cumplir al menos uno de los siguientes criterios:
• El sujeto mujer que no tiene capacidad de reproducción es elegible sin ser necesario el uso de anticonceptivos. Se define a un sujeto mujer sin capacidad de reproducción como aquel que: (1) Ha alcanzado la menopausia natural (definida como 6 meses de amenorrea espontánea con niveles séricos de la hormona foliculoestimulante [FSH] en el rango posmenopáusico determinado antes del inicio del estudio y recogido en los registros médicos de la paciente, o 12 meses de amenorrea espontánea). (2) Se encuentra en las 6 semanas posteriores a la ooforectomía bilateral quirúrgica con o sin histerectomía. O (3) se ha sometido a una ligadura de trompas bilateral. La amenorrea espontánea no incluye los casos en los que hay una enfermedad subyacente que causa amenorrea (por ejemplo: anorexia nerviosa).
• Mujeres con capacidad de reproducción que tengan una prueba de embarazo negativa en el momento del cribado y la visita inicial y que no estén lactando. Un sujeto mujer con capacidad de reproducción que se comprometa a una abstinencia real o a utilizar (o hacer que su pareja utilice) métodos anticonceptivos adecuados desde el momento del consentimiento hasta 30 días después de la última dosis del tratamiento del estudio. Es posible que se requieran períodos más largos de uso de anticonceptivos según los requisitos locales. Los métodos anticonceptivos aceptados incluyen la anticoncepción hormonal (oral, implantada, inyectada u otra anticoncepción hormonal, por ejemplo: parche o anillo anticonceptivo), el dispositivo intrauterino colocado durante ≥3 meses, método de barrera junto con espermicida O el uso de anticonceptivos de doble barrera apropiados según las regulaciones o normativas locales.
8. Un paciente varón debe:
• Aceptar que no donará esperma durante el estudio y hasta 104 días después de la última dosis, Y
• utilizar preservativo durante las relaciones sexuales con una pareja embarazada o con una mujer en edad fértil (WOCBP), incluso si está vasectomizado.
• Asimismo, la pareja en edad fértil del paciente varón debe utilizar los siguientes métodos anticonceptivos aceptados durante el estudio y hasta 104 días después de la última dosis: anticoncepción hormonal (oral, implantada, inyectada u otra anticoncepción hormonal, por ejemplo: parche o anillo anticonceptivo), dispositivo intrauterino colocado durante ≥3 meses, método de barrera junto con espermicida O el uso de anticonceptivos de doble barrera apropiados según las regulaciones o normativas locales.
9. Ser capaz de aceptar el consentimiento informado y cumplir con los procedimientos del ensayo.

E.4

Principal exclusion criteria

1. Diagnosis of Primary Lateral Sclerosis;
2. Diagnosis of Frontotemporal Dementia;
3. Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease);
4. Diagnosis of polyneuropathy;
5. Other causes of neuromuscular weakness;
6. Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function;
7. Use of intravenous (IV) edaravone within 6 months of the screening visit;
8. Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening;
9. Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation;
10. Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, or subject has a positive screening test for HIV, hepatitis B or C;
11. Presence of any of the following clinical conditions:
a. Unstable cardiac, pulmonary, endocrine, hematologic or active infectious disease
b. Unstable psychiatric illness defined as psychosis, untreated major depression within 90 days of the screening visit
c. Significant cognitive impairment, clinical dementia or psychiatric illness
d. Cancer that is currently under active treatment or is likely to require treatment during the trial that may alter the subject´s function and interfere with assessment of ALS disease progression.
12. Any comorbidity that may interfere with the functions as scored with the ALSFRS-R;
13. History of known sensitivity or intolerability to edaravone, to any related compound, or to any of the excipients;
14. Exposure to any investigational drug within 30 days of the screening visit;
15. Current substance or alcohol dependence.

1. Diagnóstico de esclerosis lateral primaria.
2. Diagnóstico de demencia frontotemporal.
3. Diagnóstico de otras enfermedades neurodegenerativas (por ejemplo, enfermedad de Parkinson, enfermedad de Alzheimer).
4. Diagnóstico de polineuropatía.
5. Otras causas de debilidad neuromuscular.
6. Tener un trastorno pulmonar significativo no atribuido a la ELA y/o requerir un tratamiento que interfiera con la evaluación de la ELA en la capacidad respiratoria.
7. Uso de edaravona intravenosa (IV) en los 6 meses anteriores a la visita de cribado.
8. Depender de ventilación mecánica (invasiva o no invasiva) o necesitar una traqueostomía en el momento del cribado.
9. Deterioro renal señalado en un aclaramiento de creatinina inferior a 50 ml/min calculado mediante la ecuación de Cockcroft Gault.
10. El sujeto tiene antecedentes de una enfermedad hepática clínicamente significativa (hepatitis o enfermedad del tracto biliar), o, el sujeto cuenta con una prueba de detección positiva de VIH, la hepatitis B o C.
11. Presencia de cualquiera de las siguientes condiciones clínicas:
a. Enfermedad cardíaca, pulmonar, endocrina, hematológica o infecciosa activa inestable.
b. Enfermedad psiquiátrica inestable entendida como psicosis, una depresión mayor no tratada en los 90 días anteriores a la visita de cribado.
c. Deterioro cognitivo significativo, demencia clínica o enfermedad psiquiátrica.
d. Cáncer que se encuentre actualmente en tratamiento activo o que requiera probablemente un tratamiento durante el ensayo que pueda alterar la función del sujeto e interferir con la evaluación de la evolución de la enfermedad de la ELA.
12. Cualquier comorbilidad que pueda interferir con las funciones puntuadas mediante la ALSFRS-R.
13. Antecedentes de sensibilidad o intolerancia conocida a la edaravona, a cualquier compuesto relacionado o a cualquiera de los excipientes.
14. Exposición a cualquier fármaco de una investigación en los 30 días anteriores a la visita de cribado.
15. Dependencia actual de sustancias o alcohol.

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) score after 48 weeks of treatment

Cambios con respecto al inicio en la puntuación de la escala revisada de valoración funcional de la esclerosis lateral amiotrófica (ALSFRS-R) tras 48 semanas de tratamiento.

E.5.1.1

Timepoint(s) of evaluation of this end point

After 48 weeks of treatment

Tras 48 semanas de tratamiento.

E.5.2

Secondary end point(s)

Key secondary endpoints
1. Combined assessment of function and survival (CAFS) at 48 and 72 weeks of treatment;
2. Survival time, i.e. time to death, tracheostomy or initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days, over 72 weeks of treatment

Efficacy
1. Change from baseline in ALSFRS-R score after 12, 24, 60, 72 weeks of treatment;
2. The slope of the decrease in ALSFRS-R score over time at 24, 48, 60 and 72 weeks of treatment;
3. Absolute ALSFRS-R score per assessment time point;
4. Change from baseline in ALSFRS-R score on Bulbar function (question 1-3 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment;
5. Change from baseline in ALSFRS-R score on Fine motor function (question 4-6 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment;
6. Change from baseline in ALSFRS-R score on Gross motor function (question 7-9 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment;
7. Change from baseline in ALSFRS-R score on Respiratory function (question 10-12 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment;
8. Time to a 3, 6, 9 and 12 points change from baseline in ALSFRS-R score;
9. Proportion of subjects with change from baseline in ALSFRS-R score at 24, 48, 60 and 72 weeks of treatment in categories: categories will include change ≥0, change between <0 and ≥-1, change between <-1 and ≥-2 etc.;
10. Staging of disease progression (King’s staging system and MiToS);
11. Overall survival: Proportion of subjects alive (survival rate) after 24, 48, 60 and 72 weeks of treatment;
12. Proportion of subjects alive and no tracheostomy, or no initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days after 24, 48, 60 and 72 weeks of treatment;
13. Absolute value and change from baseline in slow vital capacity (SVC, liters) at 24, 48, 60 and 72 weeks of treatment;
14. Absolute value and change from baseline in the overall mega score for the hand-held dynamometer (HHD) at 24, 48, 60 and 72 weeks of treatment.

QoL
1. Absolute values and change from baseline in the total score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) Form at 24, 48 and 72 weeks of treatment;
2. Absolute values and change from baseline in EuroQoL – 5 Dimensions-5 Levels (EQ-5D-5L) questionnaire score and health related QoL at 24, 48, 60 and 72 weeks of treatment.
3. Visual Analogue Scale (VAS) score at 24, 48, 60 and 72 weeks of treatment.

Cognition
1. Proportion of subjects with a change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and behavioural ALS Screen) total score;
2. Time to a mean change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS total score.

Pharmacokinetics
(Population) PK parameters of FAB122 and riluzole

Criterios secundarios clave de valoración
1. Evaluación combinada de función y supervivencia (CAFS) a las 48 y 72 semanas de tratamiento.
2. Tiempo de supervivencia (es decir, tiempo hasta el fallecimiento, traqueostomía o comenzarse a usar ventilación no invasiva durante más de 20 horas al día en un periodo de más de 10 días consecutivos, durante 72 semanas de tratamiento).
Eficacia
1. Cambios con respecto al inicio en la puntuación de la ALSFRS-R tras 12, 24, 60 y 72 semanas de tratamiento.
2. La pendiente de la disminución de la puntuación de la ALSFRS-R en el tiempo a las 24, 48, 60 y 72 semanas de tratamiento.
3. Puntuación absoluta de la ALSFRS-R por momento de evaluación.
4. Cambios con respecto al inicio en la puntuación de la ALSFRS-R en la función bulbar (pregunta 1-3 de la ALSFRS-R) después de 12, 24, 60 y 72 semanas de tratamiento.
5. Cambios con respecto al inicio en la puntuación de la ALSFRS-R en la función motora fina (pregunta 4-6 de la ALSFRS-R) después de 12, 24, 60 y 72 semanas de tratamiento.
6. Cambios con respecto al inicio en la puntuación de la ALSFRS-R en la función motora gruesa (pregunta 7-9 de la ALSFRS-R) después de 12, 24, 60 y 72 semanas de tratamiento.
7. Cambios con respecto al inicio en la puntuación de la ALSFRS-R en la función respiratoria (pregunta 10-12 de la ALSFRS-R) después de 12, 24, 60 y 72 semanas de tratamiento.
8. Tiempo hasta un cambio de 3, 6, 9 y 12 puntos desde el inicio en la puntuación ALSFRS- R.
9. Proporción de sujetos con cambios en la puntuación de la ALSFRS-R, con respecto al inicio, a las 24, 48, 60 y 72 semanas de tratamiento en las categorías: las categorías incluirán cambio ≥0, cambio entre <0 y ≥-1, cambio entre <-1 y ≥-2, etc.
10. Estadificación de la evolución de la enfermedad (sistema de estadificación de King y MiToS).
11. Supervivencia global: Proporción de sujetos con vida (tasa de supervivencia) tras 24, 48, 60 y 72 semanas de tratamiento.
12. Proporción de sujetos con vida y sin traqueostomía, o sin haber comenzado a usar ventilación no invasiva durante más de 20 horas al día en un periodo de más de 10 días consecutivos, tras 24, 48, 60 y 72 semanas de tratamiento.
13. Valor absoluto y cambios con respecto al inicio en la capacidad vital lenta (CVL, litros) a las 24, 48, 60 y 72 semanas de tratamiento.
14. Valor absoluto y cambios con respecto al inicio en la puntuación global para el dinamómetro manual (HHD) a las 24, 48, 60 y 72 semanas de tratamiento.
Calidad de vida
1. Valores absolutos y cambios desde el inicio en la puntuación total del formulario de evaluación de 40 preguntas de la ELA (ALSAQ-40) a las 24, 48 y 72 semanas de tratamiento.
2. Valores absolutos y cambios desde el inicio en la puntuación del cuestionario de calidad de vida y salud EuroQoL - 5 Dimensiones-5 Niveles (EQ-5D-5L) a las 24, 48, 60 y 72 semanas de tratamiento.
3. Puntuación de la escala visual analógica (EVA) a las 24, 48, 60 y 72 semanas de tratamiento.
Cognición
1. Proporción de sujetos con un cambio de ≥8, ≥4 y ≥9 en la puntuación total de la ELA específica, la ELA no específica y el ECAS (examen cognitivo y conductual en ELA de Edimburgo).
2. Plazo hasta un cambio medio de ≥8, ≥4 y ≥9 en la puntuación total de la ELA específica, ELA no específica y ECAS.
Farmacocinética
(Población) Parámetros PK de FAB122 y riluzol.

E.5.2.1

Timepoint(s) of evaluation of this end point

As indicated in the end points, after 12, 24, 48, 60, and/or 72 weeks of treatment

Tal como se indica en los criterios de valoración, tras las 12, 24, 48, 60, y/o 72 semanas de tratamiento.

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

Yes

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

Yes

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

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

Russian Federation

Belgium

France

Germany

Ireland

Italy

Netherlands

Poland

Spain

United Kingdom

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

UVUP

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

E.8.9.2

In all countries concerned by the trial days

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:

250

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

250

F.4.2.2

In the whole clinical trial

300

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)

The Sponsor commits to offering all patients continuation of the treatment with the IMP, FAB122, after the end of the study until FAB122 becomes available, unless the Sponsor is forced to terminate the clinical development program of FAB122 for any reason, e.g. if analysis of the data fails to confirm significant treatment effects on one or more of the endpoints, or in case of any serious safety effects of the treatment with FAB122.

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

2021-10-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-09-22

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

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