Clinical Trials Register

Summary
EudraCT Number:	2017-000572-28
Sponsor's Protocol Code Number:	SHP647-302
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:	2017-12-19
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

2017-000572-28

A.3

Full title of the trial

A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Efficacy and Safety Study of SHP647 as Induction Therapy in Subjects with Moderate to Severe Ulcerative Colitis (FIGARO UC 302)

Estudio en fase 3, aleatorizado, doble ciego, controlado con placebo y de grupos paralelos para evaluar la eficacia y la seguridad de SHP647 como tratamiento de inducción en sujetos con colitis ulcerosa moderada o grave (FIGARO UC 302)

A.3.1

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

Research study to determine whether an investigational drug, SHP647, is safe and effective in the treatment of moderate to severe Ulcerative Colitis, compared with placebo (dummy treatment) – using a randomised and blinded study design (investigator and patients are not aware whether they receive study drug or placebo)(FIGARO UC 302).

Estudio de investigación para determinar si un fármaco en investigación, SHP647, es seguro y eficaz en el tratamiento de la colitis ulcerosa de moderada a severa, en comparación con el placebo (tratamiento ficticio) - utilizando un diseño de estudio aleatorizado y ciego (el investigador y los pacientes no son conscientes de si reciben fármaco de estudio o placebo) (FIGARO UC 302).

A.3.2

Name or abbreviated title of the trial where available

FIGARO UC 302

A.4.1

Sponsor's protocol code number

SHP647-302

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	Shire Human Genetic Therapies, 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	Shire Human Genetic Therapies, 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	Shire Human Genetic Therapies, Inc.
B.5.2	Functional name of contact point	Melanie Ivarsson
B.5.3	Address:
B.5.3.1	Street Address	300 Shire Way
B.5.3.2	Town/ city	Lexington
B.5.3.3	Post code	MA 02421
B.5.3.4	Country	United States
B.5.4	Telephone number	+34900834223
B.5.6	E-mail	RegistroEspanolDeEstudiosClinicos@druginfo.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	SHP647
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Anti-MAdCAM antibody
D.3.9.2	Current sponsor code	SHP647
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	75
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	SHP647
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Anti-MAdCAM antibody
D.3.9.2	Current sponsor code	SHP647
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Ulcerative Colitis

Colitis Ulcerosa

E.1.1.1

Medical condition in easily understood language

Long-term condition that results in inflammation and ulcers of the colon and rectum

Afeccion prolongada que provoca inflamacion y ulceras en el colon y recto

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10045365
E.1.2	Term	Ulcerative colitis
E.1.2	System Organ Class	100000004856

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of the study is to evaluate the efficacy of SHP647 in inducing remission,based on composite score of patient-reported symptoms and centrally read endoscopy, in subjects with moderate to severe ulcerative colitis (UC).

El objetivo principal del estudio es evaluar la eficacia de SHP647 para inducir la remisión, determinada mediante la puntuación combinada de los síntomas percibidos por el paciente y la evaluación centralizada de la endoscopia, en sujetos con colitis ulcerosa (CU) moderada o grave.

E.2.2

Secondary objectives of the trial

To evaluate the efficacy of SHP647 in achieving endoscopic remission, based on centrally read endoscopy.
• To evaluate the efficacy of SHP647 in achieving clinical remission, based on composite score of patient-reported symptoms.
• To evaluate the efficacy of SHP647 in inducing clinical response, based on composite score of patient-reported symptoms and centrally read endoscopy.
• To evaluate the efficacy of SHP647 in achieving mucosal healing, based on endoscopic and histological assessment.

• Evaluar la eficacia de SHP647 para conseguir la remisión endoscópica, determinada mediante la evaluación centralizada de la endoscopia.
• Evaluar la eficacia de SHP647 para conseguir la remisión clínica, determinada mediante la puntuación combinada de los síntomas percibidos por el paciente.
• Evaluar la eficacia de SHP647 para inducir la respuesta clínica, determinada mediante la puntuación combinada de los síntomas percibidos por el paciente y la evaluación centralizada de la endoscopia.
• Evaluar la eficacia de SHP647 para conseguir la cicatrización de la mucosa, determinada mediante la evaluación endoscópica e histológica.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the study:
1. Subjects must be between ≥16 and ≤80 years of age at the time of the signing of the informed consent/assent form.
NOTE: Subjects <18 years of age must weigh ≥40 kg and must have body mass index (BMI) ≥16.5.
2. Subjects must have a documented diagnosis (radiologic or endoscopic with histology) of UC for ≥3 months before screening. The following must be available in each subject’s source documentation:
• A biopsy report to confirm the histological diagnosis.
• A report documenting disease duration based upon prior colonoscopy.
NOTE: If this documentation is not available at the time of screening, a colonoscopy with biopsy to confirm the diagnosis is required during the screening period.
3. Subjects must be willing to undergo a flexible sigmoidoscopy or colonoscopy (if preferred), including biopsy sample collection, during screening after all other inclusion criteria have been met.
4. Subjects must have moderate to severe active UC, defined as a total Mayo score of ≥6, including a centrally read endoscopic subscore ≥2, rectal bleeding subscore ≥1, and stool frequency subscore ≥1 at baseline (Visit 2).
5. Subjects must have evidence of UC extending proximal to the rectum (ie, not limited to proctitis).
6. Subjects must have had an inadequate response to, or lost response to, at least 1 conventional treatment such as mesalamine (5 aminosalicylic acid [5-ASA]), glucocorticoids, immunosuppressants (azathioprine [AZA], 6 mercaptopurine [6 MP], or methotrexate [MTX]), or anti-TNF.

Los sujetos deberán cumplir todos los criterios de inclusión siguientes pata poder participar en el estudio:
1. Tener (el sujeto, o su progenitor o representante legal) entendimiento, capacidad y voluntad para cumplir plenamente con los procedimientos y las restricciones del estudio.
2. Ser capaz de otorgar voluntariamente el consentimiento o asentimiento informado (según proceda) por escrito, firmado y fechado personalmente o a través de un representante legal, para participar en el estudio.
3. Tener entre 16 y 80 años de edad en el momento de firmar el documento de consentimiento o asentimiento informado.
NOTA: Los sujetos menores de 18 años deben tener un peso ≥ 40 kg y un índice de masa corporal (IMC) ≥ 16,5.
4. Tener un diagnóstico (radiológico o endoscópico con examen histológico) documentado de CU desde al menos 3 meses antes de la selección. En la documentación original de todos los pacientes deberá constar lo siguiente:
• Un informe de biopsia que confirme el diagnóstico histológico.
• Un informe que confirme la duración de la enfermedad a partir de una colonoscopia anterior.
NOTA: Si en el momento de la selección no se dispone de estos documentos, habrá que realizar una colonoscopia con biopsia para confirmar el diagnóstico durante el período de selección.
5. Estar dispuesto a someterse a una sigmoidoscopia o colonoscopia (lo que sea preferible) con endoscopio flexible y obtención de muestra de biopsia durante la selección, una vez confirmado que se cumplen todos los demás criterios de inclusión.
6. Tener una CU moderada o grave, definida por una puntuación ≥ 6 en el índice de Mayo con una subpuntuación de la evaluación centralizada de la endoscopia ≥ 2, de rectorragia ≥ 1 y de frecuencia de las deposiciones ≥ 1 en el momento basal (visita 2)
7. Tener signos de extensión de la CU proximal al recto (es decir, no se limita a proctitis).
8. Haber presentado una respuesta insuficiente, o pérdida de la respuesta, a al menos un tratamiento convencional como mesalamina (ácido 5 aminosalicílico [5-ASA]), glucocorticoides, inmunodepresores (azatioprina [AZA], 6 mercaptopurina [6 MP] o metotrexato [MTX]) o anti-TNF.
9. Los sujetos que estén recibiendo alguno de los tratamientos para la UC que se describen en la sección 5.2.1 del protocolo podrán participar siempre que la dosis sea estable y esté previsto mantenerla durante el período de tiempo que se indica.
10. Ser varón o mujer no embarazada ni en período de lactancia y, en caso de mantener relaciones sexuales, comprometerse a cumplir los requisitos de anticoncepción que exige el protocolo (excepto las mujeres que no estén en edad fértil). Los varones y mujeres en edad fértil que mantengan relaciones sexuales deben comprometerse a utilizar métodos anticonceptivos aceptables durante todo el estudio.

E.4

Principal exclusion criteria

Subjects are excluded from the study if any of the following exclusion criteria are met:
1. Subjects with indeterminate colitis, microscopic colitis, ischemic colitis, infectious colitis, or clinical/histologic findings suggestive of Crohn’s disease.
2. Subjects with colonic dysplasia or neoplasia.
3. Subjects with past medical history or presence of toxic megacolon.
4. Subjects with colonic stricture, past medical history of colonic resection, a history of bowel surgery within 6 months before screening, or who are likely to require surgery for UC during the treatment period.
5. Subjects at risk for colorectal cancer must have a colonoscopy performed during the screening period with results available within 10 days before the baseline visit (Visit 2), unless the subject has had a colonoscopy within 1 year prior to screening, and any adenomatous polyps found at that examination have been excised.
6. Subjects have participated in other investigational studies within either 30 days or 5 half lives of investigational product used in the study (whichever is longer) before baseline.
7. Subjects with active enteric infections (positive stool culture and sensitivity), Clostridium difficile infection or pseudomembranous colitis [subjects with C. difficile infection at screening may be allowed re-test after treatment], evidence of active cytomegalovirus infection or Listeria monocytogenes, known active invasive fungal infections such as histoplasmosis or parasitic infections, clinically significant underlying disease that could predispose the subjects to infections, or a history of serious infection (requiring parenteral antibiotic and/or hospitalization) within 4 weeks before the baseline visit (Visit 2).
8. Subjects with abnormal chest x-ray findings at screening (Visit 1), such as presence of active tuberculosis (TB), general infections, heart failure, or malignancy.
9. Subjects with evidence of active or latent infection with Mycobacterium tuberculosis (TB) who have not completed a generally accepted full course of treatment before randomization are excluded.
10. Subjects with a pre-existing demyelinating disorder such as multiple sclerosis or new onset seizures, unexplained sensory motor, or cognitive behavioral, neurological deficits, or significant abnormalities noted during screening.
11. Subjects with any unexplained symptoms suggestive of progressive multifocal leukoencephalopathy (PML) based on the targeted neurological assessment during the screening period.

Se excluirá del estudio a los sujetos que cumplan alguno de los criterios siguientes:
1. Sujetos con colitis indeterminada, colitis microscópica, colitis isquémica, colitis infecciosa o hallazgos clínicos / histológicos indicativos de enfermedad de Crohn.
2. Sujetos con displasia o neoplasia colónica.
3. Sujetos con antecedentes médicos o presencia de megacolon tóxico.
4. Sujetos con estenosis del colon, antecedentes médicos de resección colónica, antecedentes de cirugía intestinal dentro de los 6 meses previos a la seleccion o que probablemente requieran cirugía para la CU durante el período de tratamiento.
5. En los sujetos con riesgo de cáncer colorrectal, haberse sometido a una colonoscopia durante el período de selección y disponer de los resultados en los 10 días anteriores a la visita basal (visita 2), a menos que se haya efectuado una colonoscopia en el año anterior a la selección y se hayan extirpado todos los pólipos que se observaron en aquella exploración.
6. Sujetos que hayan participado en otros estudios de investigación dentro de los 30 días o 5 semividas del producto en investigación utilizado en el estudio (el que sea mayor) antes del momento basal del estudio.ç
7. Sujetos con infecciones entéricas activas (cultivo y sensibilidad positivos de heces), infección por Clostridium difficile o colitis pseudomembranosa [sujetos con infección por C. difficile en la seleccion pueden volver a someterse a la prueba después del tratamiento], evidencia de infección activa por citomegalovirus o Listeria monocytogenes, infecciones fúngicas invasivas activas conocidas como histoplasmosis o infecciones parasitarias, enfermedad subyacente clínicamente significativa que pudiese predisponer a los sujetos a infecciones, o antecedentes de infección grave (que requieran antibióticos parenterales y/u hospitalización ) dentro de las 4 semanas previas a la visita basal (visita 2).
8. Sujetos con hallazgos anormales en la radiografía de tórax durante la seleccion (Visita 1), como presencia de tuberculosis activa (TB), infecciones generales, insuficiencia cardíaca o malignidad.
9. Se excluyen los sujetos con evidencia de infección activa o latente con Mycobacterium tuberculosis (TB) que no hayan completado un curso completo de tratamiento generalmente aceptado antes de la aleatorización.
10. Sujetos con una enfermedad desmielinizante preexistente como esclerosis múltiple, convulsiones o déficits neurológicos sensitivomotores o cognitivo-conductuales de nueva aparición o anomalías significativas observadas durante la selección.
11. Sujetos con cualquier síntoma no justificado indicativo de leucoencefalopatía multifocal progresiva (LMP) en la exploración neurológica dirigida que se realiza en el período de selección.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is the proportion of subjects in remission at the Week 12 visit. Remission is defined as a composite score of patient-reported symptoms using daily e-diary and centrally read endoscopy as follows:
• stool frequency subscore of 0 or 1 with at least a 1-point change from baseline
AND
• rectal bleeding subscore of 0
AND
• endoscopic subscore of 0 or 1 (modified, excludes friability).

El criterio de valoración principal de la eficacia es el porcentaje de pacientes en remisión en la visita de la semana 12. La remisión se define, según la puntuación compuesta de los síntomas percibidos por el paciente mediante el diario electrónico y la evaluación centralizada de la endoscopia, del modo siguiente:
• Subpuntuación de la frecuencia de las deposiciones de 0 o 1 con una variación de al menos 1 punto con respecto al valor basal.
Y
• Subpuntuación de rectorragia de 0.
Y
• Subpuntuación endoscópica de 0 o 1 (modificada, se excluye la friabilidad).

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 12 visit

Visita de la semana 12

E.5.2

Secondary end point(s)

• Proportion of subjects with endoscopic remission, as defined by centrally read endoscopic subscore 0 or 1 (modified, excludes friability), at the Week 12 visit.
• Proportion of subjects with clinical remission, as defined by stool frequency subscore of 0 or 1 with at least a 1-point change from baseline in stool frequency subscore, and rectal bleeding subscore of 0, at the Week 12 visit.
• Proportion of subjects with clinical response based on composite score at the Week 12 visit. Clinical response (composite) is defined as a decrease from baseline in the composite score of patient-reported symptoms using daily e-diary and centrally read endoscopy of at least 2 points and at least 30%, with an accompanying decrease in the subscore for rectal bleeding ≥1 point or a subscore for rectal bleeding ≤1.
• Proportion of subjects with mucosal healing based on endoscopic and histological assessment at the Week 12 visit. Mucosal healing is defined by centrally read endoscopic subscore 0 or 1 (modified, excludes friability) and centrally read Geboes score of ≤2.

• Porcentaje de sujetos con remisión endoscópica, definida por una subpuntuación de 0 o 1 en la evaluación centralizada de la endoscopia (modificada, se excluye la friabilidad), en la visita de la semana 12.
• Porcentaje de sujetos con remisión clínica, definida por una subpuntuación de frecuencia de las deposiciones de 0 a 1 con una variación de al menos 1 punto con respecto al valor basal y una subpuntuación de rectorragia de 0, en la visita de la semana 12.
• Porcentaje de sujetos con respuesta clínica según la puntuación compuesta en la visita de la semana 12. La respuesta clínica (compuesta) se define como una disminución de la puntuación compuesta de los síntomas percibidos por el paciente mediante el diario electrónico y la evaluación centralizada de la endoscopia de al menos 2 puntos y al menos el 30% con respecto al momento basal, acompañada de una disminución de la subpuntuación de la rectorragia de al menos 1 punto o una subpuntuación de la rectorragia ≤ 1.
• Porcentaje de sujetos con cicatrización de la mucosa según la evaluación endoscópica e histológica en la visita de la semana 12. La cicatrización de la mucosa se define por una puntuación de 0 o 1 en la evaluación centralizada de la endoscopia (modificada, se excluye la friabilidad) y una puntuación ≤ 2 en la escala de Geboes determinada centralmente.

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 12 visit

Visita de la semana 12

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

Yes

E.6.13.1

Other scope of the trial description

Quality of Life

Calidad de vida

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

Yes

E.8.2.3.1

Comparator description

25 mg or 75 mg SHP647

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

Argentina

Belgium

Bosnia and Herzegovina

Bulgaria

Canada

Colombia

France

Greece

Hungary

Ireland

Japan

Korea, Republic of

Mexico

Portugal

Slovakia

Spain

Switzerland

Ukraine

United States

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 Study Completion Date is defined as the date the final subject, across all sites, completes their final protocol-defined assessment. Please note that this includes the follow-up visit or contact, whichever is later.

La fecha de finalización del estudio se define como la fecha en que el ultimo sujeto, en todos los centros, completa su evaluación final definida por el protocolo. Teniendo en cuenta que esto incluye la visita de seguimiento o contacto, lo que sea posterior.

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)

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)

Yes

F.1.2.1

Number of subjects for this age range:

726

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

Yes

F.3.3.7.1

Details of other specific vulnerable populations

Adolescents (16-17 years)

Adolescentes (16-17 años)

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

253

F.4.2.2

In the whole clinical trial

825

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)

At the end of the 12 week treatment period, eligible subjects will be offered the opportunity to participate in a double-blind maintenance study (SHP647-303; for subjects who achieve clinical response) or a long-term safety extension (LTS) study (SHP647-304; for subjects who do not achieve a clinical response). At the end of SHP647 study participation, patients will return to standard of care.

Al final del período de tratamiento de 12 semanas, se les ofrecerá a los sujetos elegibles la oportunidad de participar en un estudio de mantenimiento doble ciego (SHP647-303, para sujetos que logran respuesta clínica) o un estudio de extensión de seguridad a largo plazo (LTS) ( SHP647-304; para sujetos que no logran una respuesta clínica). Al final de la participación en el estudio SHP647, los pacientes volverán a la atención 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

2018-02-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-02-08

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2020-10-06

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