Clinical Trials Register

Summary
EudraCT Number:	2021-001015-82
Sponsor's Protocol Code Number:	ALXN1840-WD-302
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:	2021-08-23
Trial results	View 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

2021-001015-82

A.3

Full title of the trial

A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease.

Estudio multicéntrico, aleatorizado, controlado, abierto y ciego para el evaluador para evaluar la eficacia, la seguridad, la farmacocinética y la farmacodinámica de ALXN1840 en comparación con el tratamiento habitual en participantes pediátricos con enfermedad de Wilson

A.3.1

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

Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease

Estudio de fase III abierto de ALXN1840 en comparación con el tratamiento habitual en participantes pediátricos con enfermedad de Wilson

A.4.1

Sponsor's protocol code number

ALXN1840-WD-302

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/261/2021

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Alexion Pharmaceuticals, Inc.
B.1.3.4	Country	United States
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	Alexion Pharmaceuticals, Inc
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Alexion Pharma Spain S.L.
B.5.2	Functional name of contact point	Anna Anguera
B.5.3	Address:
B.5.3.1	Street Address	Av. Diagonal 601 1º
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	+34630 918 794
B.5.6	E-mail	anna.anguera@alexion.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	EU/3/12/1089
D.3 Description of the IMP
D.3.1	Product name	ALXN1840
D.3.2	Product code	ALXN1840
D.3.4	Pharmaceutical form	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	tiomolibdic acid
D.3.9.1	CAS number	649749-10-0
D.3.9.2	Current sponsor code	ALXN1840
D.3.9.3	Other descriptive name	BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate
D.3.9.4	EV Substance Code	SUB168578
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	15
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.3.1	Product name	ALXN1840
D.3.2	Product code	ALXN1840
D.3.4	Pharmaceutical form	Tablet
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	tiomolibdic acid
D.3.9.1	CAS number	649749-10-0
D.3.9.2	Current sponsor code	ALXN1840
D.3.9.3	Other descriptive name	BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate
D.3.9.4	EV Substance Code	SUB168578
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	1.25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Wilson Disease

Enfermedad de Wilson

E.1.1.1

Medical condition in easily understood language

Wilson Disease

Enfermedad de Wilson

E.1.1.2

Therapeutic area

Body processes [G] - Metabolic Phenomena [G03]

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	10047988
E.1.2	Term	Wilson's disease
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 evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to SoC, on copper control in participants with WD aged 3 to < 18 years of age at the time of enrollment

Evaluar la eficacia de ALXN1840 administrado durante 48 semanas, en comparación con el TH, sobre el control del cobre en participantes con EW de 3 a <18 años en el momento de la inclusión

E.2.2

Secondary objectives of the trial

- Evaluate the safety and tolerability of ALXN1840 administered for up to 48 weeks.
- Evaluate PD and biomarkers of ALXN1840 vs SoC administered for 48 weeks.
- Evaluate the effects of ALXN1840 and SoC on the NCC responder rate.
- Evaluate the effects of ALXN1840 and SoC on participant reported disability status.
- Evaluate the effects of ALXN1840 and SoC on rater-blinded neurological status.
- Evaluate PK of ALXN1840 administered for 48 weeks.
- Evaluate the effects of ALXN1840 and SoC on global clinical symptoms as assessed by the Investigator.
- Evaluate the effects of ALXN1840 and SoC on hepatic status.

- Evaluar la seguridad y la tolerabilidad de ALXN1840 administrado durante un máximo de 48 semanas.
- Evaluar la FD y los biomarcadores de ALXN1840 en comparación con el TH administrado durante 48 semanas.
- Evaluar los efectos de ALXN1840 y del TH sobre la tasa de respuesta del CNUC.
- Evaluar los efectos de ALXN1840 y del TH sobre el estado de discapacidad comunicado por los participantes.
- Evaluar los efectos de ALXN1840 y del TH sobre el estado neurológico ciego para el evaluador.
- Evaluar la FC de ALXN1840 administrado durante 48 semanas.
- Evaluar los efectos de ALXN1840 y del TH sobre los síntomas clínicos globales evaluados por el investigador.
- Evaluar los efectos de ALXN1840 y del TH sobre el estado hepático.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
Age
1. Participants must be aged 3 to <18 years at time of signing the informed consent/assent.
Type of Participants and Disease Characteristics
2. Established diagnosis of WD by Leipzig-Score ≥ 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines (Ferenci, 2003; EASL, 2012). Note: Historical test results for WD, including some or all of the following: presence of KF rings, neurologic symptoms, serum ceruloplasmin below the reference range, Coombs-negative hemolytic anemia, elevated liver or urinary copper, presence of mutations in the ATP7B gene, or other, as considered appropriate, may be used instead to confirm the diagnosis of Wilson disease.
3. Participant's parent/proxy must be willing and able to give written informed consent and the participant must be willing to give written informed assent (if applicable as determined by the central or local Institutional Review Board [IRB]/Institutional [or Independent] Ethics Committee [IEC]). If allowable per local regulations, a participant’s Legally Acceptable Representative (LAR) may provide informed consent if a participant is unable to do so.
4. Adequate venous access to allow collection of required blood samples.
5. Able to swallow intact ALXN1840 tablets or mini-tablets. Participants who require gastrostomy devices for feeding or medications may be enrolled upon agreement by the Medical Monitor.
6. Willing to avoid intake of foods and drinks with high contents of copper throughout the study duration
Sex
7. Female participants of childbearing potential and male participants must follow protocol-specified contraception guidance as described in Section 10.4 of the protocol.
Informed Consent
8. Capable of giving signed informed consent or assent as described in Section 10.1.3 of the protocol, which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol.

Únicamente podrán ser incluidos en el estudio los participantes que cumplan todos los criterios siguientes:
Edad
1. Los participantes deberán tener entre 3 y <18 años en el momento de firmar el consentimiento/asentimiento informado.
Tipo de participantes y características de la enfermedad
2. Diagnóstico establecido de EW según una puntuación de Leipzig  4 documentada mediante análisis según se describe en las directrices de práctica clínica de la Asociación Europea para el Estudio de la Cirrosis Hepática de 2012 (Ferenci, 2003; EASL, 2012). Nota: Para confirmar el diagnóstico de EW pueden utilizarse los resultados de los análisis históricos de la EW, incluidos algunos o todos los siguientes: presencia de anillos de KF, síntomas neurológicos, ceruloplasmina sérica por debajo del intervalo de referencia, anemia hemolítica negativa para Coombs, elevación del cobre hepático o urinario, presencia de mutaciones en el gen ATP7B u otros, lo que se considere apropiado.
3. Los padres o representantes del participante deberán estar dispuestos y ser capaces de otorgar su consentimiento informado por escrito, y el participante deberá estar dispuesto a otorgar su asentimiento informado por escrito (si procede según lo determinado por el Comité de Ética de la Investigación con medicamentos [CEIm] central o local). Si lo permite la normativa local, el representante legal del participante podrá otorgar su consentimiento informado si el participante no puede hacerlo.
4. Acceso venoso adecuado para permitir la obtención de las muestras de sangre necesarias.
5. Capacidad para tragar comprimidos o minicomprimidos de ALXN1840 inalterados. Los participantes que necesiten dispositivos de gastrostomía para alimentarse o recibir medicamentos podrán ser incluidos con el acuerdo previo del monitor médico.
6. Estar dispuesto a evitar el consumo de alimentos y bebidas con alto contenido de cobre durante todo el estudio.
Sexo
7. Las mujeres participantescon capacidad reproductiva y los varones participantes deberán seguir las normas sobre métodos anticonceptivos especificadas en el protocolo que se describen en la sección 10.4 del protocolo
Consentimiento informado
8. Capacidad para otorgar el consentimiento o asentimiento informado firmado, como se describe en el apartado 10.1.3 dl protocolo, lo que incluye el cumplimiento de los requisitos y restricciones recogidos en el documento de consentimiento o asentimiento informado y en este protocolo.

E.4

Principal exclusion criteria

Participants are excluded from the study if any of the following criteria apply:
Medical Conditions
1. Decompensated hepatic cirrhosis.
2. MELD score > 13 (ages 12 to <18) or PELD score > 13 (ages 3 to < 12).
3. Modified Nazer score > 7.
4. Clinically significant gastrointestinal (GI) bleed within past 3 months
5. Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) for participants treated for > 28 days with WD therapy (Cohort 1).
6. ALT > 5 × ULN for treatment naïve participants or participants who have been treated for ≤ 28 days (Cohort 2)
7. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
8. Hemoglobin less than lower limit of the reference range for age and sex.
9. History of seizure activity within 6 months prior to informed consent/assent.
Prior/Concomitant Therapy
10. Previous use of ALXN1840 or ammonium tetrathiomolybdate; concomitant use of penicillamine, trientine, or zinc (for participants randomized to ALXN1840).
Prior/Concurrent Clinical Study Experience
11. The use of an investigational drug within 30 days before initiation of the first dose of study intervention.
Diagnostic assessments
12. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5]) or estimated glomerular filtration rate < 30 mL/min/1.73m2.
13. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus (participants with positive hepatitis C antibody result would require confirmation of active disease with a positive hepatitis C polymerase chain reaction test), or seropositivity for HIV.
14. Any disability acquired from trauma or another illness that, in the opinion of the Investigator, could interfere with evaluation of disability due to WD.
15. Systemic disease or other illness, or any deviation in laboratory values that are confirmed on re-examination to be clinically significant by the Investigator that would, in the opinion of the Investigator, compromise participant safety or interfere with the collection or interpretation of study results.
Other Exclusions
16. Pregnant (or females who are planning to become pregnant) or breastfeeding females.
17. Known sensitivity to ALXN1840, ALXN1840 excipients (anhydrous di-calcium phosphate, anhydrous sodium carbonate), or any of the ingredients contained in ALXN1840 or related compounds.
18. Regular alcohol consumption within 6 months prior to the study defined as > 14 units for males or > 7 units for females per week. One unit is equivalent to 14 g of alcohol: a half pint (approx. 240 mL) of beer, 1 glass (125 mL) of wine or 1 (25 mL) measure of spirits.
19. Abuse of illicit or prescribed drugs.
20. In the opinion of the Investigator, the participant and/or their parent/proxy is likely to be non-compliant or uncooperative during the study.

Quedará excluido del estudio todo candidato que cumpla alguno de los criterios siguientes:
Enfermedades
1. Cirrosis hepática descompensada.
2. Puntuación MELD >13 (de 12 a <18 años) o PELD >13 (de 3 a <12 años).
3. Puntuación Nazer modificada >7.
4. Hemorragia digestiva de trascendencia clínica en los 3 últimos meses.
5. Alanina aminotransferasa (ALT) >2 veces el límite superior de la normalidad (LSN) en los participantes tratados durante >28 días con EW (cohorte 1).
6. ALT >5 veces el LSN en los participantes no tratados previamente o que hayan sido tratados durante ≤28 días (cohorte 2).
7. Enfermedad neurológica importante con necesidad de sonda de alimentación nasogástrica o atención médica hospitalaria intensiva.
8. Hemoglobina por debajo del límite inferior del intervalo de referencia para la edad y el sexo.
9. Antecedentes de actividad convulsiva en los 6 meses previos al consentimiento/asentimiento informado.
Tratamiento previo y concomitante
10. Uso previo de ALXN1840 o tetratiomolibdato de amonio; uso concomitante de penicilamina, trientina o zinc (en los participantes aleatorizados a ALXN1840).
Experiencia en estudios clínicos previos o simultáneos
11. Uso de un fármaco en investigación en los 30 días previos al inicio de la primera dosis de la intervención del estudio.
Evaluaciones diagnósticas
12. Participantes con insuficiencia renal, definida como nefropatía terminal en diálisis (nefropatía crónica en estadio 5 [NC 5]) o filtración glomerular estimada <30 ml/min/1,73 m2.
13. Infección activa por el virus de la hepatitis B (antígeno de superficie del virus de la hepatitis B positivo) o C (en los participantes con un resultado positivo de anticuerpos contra el virus de la hepatitis C sería necesaria la confirmación de enfermedad activa con un resultado positivo en la prueba de reacción en cadena de la polimerasa del virus de la hepatitis C) o seropositividad para el VIH.
14. Cualquier discapacidad derivada de un traumatismo u otra enfermedad que, en opinión del investigador, pudiera interferir en la evaluación de la discapacidad por EW.
15. Enfermedad sistémica u otra enfermedad, o cualquier desviación de los valores analíticos que el investigador confirme en la revisión que tenga importancia clínica y que, en su opinión, pudiera comprometer la seguridad del participante o interferir en la recopilación o interpretación de los resultados del estudio.
Otras exclusiones
16. Mujeres embarazadas (o que tengan previsto quedarse embarazadas) o en período de lactancia.
17. Sensibilidad conocida a ALXN1840, los excipientes de ALXN1840 (fosfato dicálcico anhidro, carbonato sódico anhidro) o cualquiera de los componentes contenidos en ALXN1840 o compuestos relacionados.
18. Consumo habitual de alcohol en los 6 meses previos al estudio, definido como >14 unidades en los varones o >7 unidades en las mujeres por semana. Una unidad equivale a 14 g de alcohol: media pinta (unos 240 ml) de cerveza, 1 vaso (125 ml) de vino o 1 (25 ml) de licor.
19. Abuso de drogas ilegales o recetadas.
20. En opinión del investigador, es probable que el participante o sus padres o representantes incumplan el tratamiento o no colaboren durante el estudio.

E.5 End points

E.5.1

Primary end point(s)

Percentage change from baseline (Day 1) to 48 weeks in NCC in plasma. For ALXN1840-treated participants, the NCC in plasma will be corrected for the amount of copper bound to the ALXN1840 TPC (NCCcorrected)

Variación porcentual del CNUC (cobre no unido a ceruloplasmina) en plasma entre el momento basal (día 1) y la semana 48. En los participantes tratados con ALXN1840, el CNUC en plasma se corregirá en función de la cantidad de cobre unido al complejo TPC de ALXN1840 (CNUCcorregido)

E.5.1.1

Timepoint(s) of evaluation of this end point

48 weeks

48 semanas

E.5.2

Secondary end point(s)

• Incidence of adverse AEs/SAEs, AESIs, tolerability, clinical laboratory test data (including liver function tests), neurological and physical examination findings, 12-lead ECG data, and vital signs.
• AUEC for NCC
• AUEC for plasma total copper
• Biomarkers: observed, absolute and percentage changes of ceruloplasmin-bound copper and ceruloplasmin
• NCC responder rate
• Change from baseline to Week 48 in the UWDRS Part II total score
• Change from baseline in UWDRS Part III total score or individual items/subscales, as appropriate
• PK: Cmax, tmax, AUCtau and t1/2 on Day 1, Day 43 (Week 6), and Day 337 (Week 48) for plasma total molybdenum and plasma ultrafiltrate
molybdenum concentrations, accumulation ratio of Day 43 to Day 1 and Day 337 to Day 1 based on Cmax and AUCtau
• Derived population PK parameters such as apparent total body clearance (CL/F) and apparent volume of distribution (Vd/F)
• CGI-I
• Change from Baseline to Week 48 in CGI-S
• Change from Baseline to Week 48 in MELD score (ages 12 years and older) or PELD score (ages 3 to < 12 years)
• Change from Baseline to Week 48 in Modified Nazer score

• Incidencia de AA/AAG, AAIE, tolerabilidad, datos analíticos (incluidas pruebas de función hepática), hallazgos de la exploración neurológica y física, datos del ECG de 12 derivaciones y constantes vitales.
• AUEC para el CNUC.
• AUEC para el cobre total plasmático.
• Biomarcadores: variaciones observadas, absolutas y porcentuales de la ceruloplasmina y del cobre unido a la ceruloplasmina.
• Tasa de respuesta del CNUC.
• Variación de la puntuación total en la parte II de la UWDRS entre el momento basal y la semana 48.
• Variación con respecto al momento basal de la puntuación total en la parte III de la UWDRS o de apartados/subescalas determinadas, según proceda.
• FC: Cmáx, tmáx, AUCtau y t1/2 los días 1, 43 (semana 6) y 337 (semana 48) para las concentraciones plasmáticas de molibdeno total y de molibdeno ultrafiltrado, cociente de acumulación entre los días 43 y 1 y entre los días 337 y 1 basado en la Cmáx y el AUCtau.
• Parámetros FC poblacionales derivados, como CL/F y Vd/F.
• CGI-I.
• Variación de la puntuación CGI-S entre el momento basal y la semana 48.
• Variación entre el momento basal y la semana 48 de las puntuaciones MELD (a partir de 12 años) o PELD (de 3 a <12 años).
• Variación de la puntuación de Nazer modificada entre el momento basal y la semana 48.

E.5.2.1

Timepoint(s) of evaluation of this end point

48 weeks

48 semanas

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

Yes

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)

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Ciego para el evaluador

Rater Blinded

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Tratamiento habitual (Penicilamina, Trientina, y Zinc)

Standard of Care (Penicillamine, Trientine, and Zinc)

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

Canada

Japan

Korea, Republic of

Russian Federation

Serbia

Turkey

United States

Austria

Denmark

France

Germany

Hungary

Netherlands

Poland

Spain

United Kingdom

Czechia

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

The end of the study is defined as the date the last participant completes the last visit shown in the Schedule of Activities (SoA) of Period 2.

El final del estudio se define como la fecha en que el último participante completa la última visita que se muestra en el calendario de actividades (CdA) del Periodo 2.

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

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Participant's parent/proxy and the participant must be willing and able to give written informed consent and assent, respectively, or a participant’s LAR may provide informed consent if a participant is unable to do so.

Padres/representantes del participante y el participante deben estar dispuestos y ser capaces de dar consentimiento informado y asentimiento por escrito, respectivamente, o el RL puede dar consentimiento informado si un participante no puede hacerlo.

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)

After completion of Period 2, participants will either:
• Transition to therapy that was discontinued before enrollment, or
• Participants who have successfully completed all study assessments and were not withdrawn prematurely may be eligible for post-study access. Participants will receive study drug for up to 2 years or until 1) the study drug is approved and available by prescription or 2) via Alexion post-study/early access programs.

Después de completar el Período 2, los participantes:
• Transición a la terapia que se suspendió antes del reclutamiento, o
• Los participantes que hayan completado con éxito las evaluaciones del estudio y no hayan sido retirados prematuramente pueden ser elegibles para el acceso post-estudio. Recibirán el fármaco del estudio durante un máximo de 2 años o hasta que 1) el fármaco esté aprobado y disponible con receta médica o 2) a través de los programas de post-estudio de Alexion.

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

2021-11-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-10-06

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2023-04-25

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