Clinical Trials Register

Summary
EudraCT Number:	2021-002365-18
Sponsor's Protocol Code Number:	CP543.5002
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:	2021-10-26
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2021-002365-18

A.3

Full title of the trial

A MULTICENTER, OPEN-LABEL, EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY AND EFFICACY OF CTP-543 IN ADULT PATIENTS WITH MODERATE TO SEVERE ALOPECIA AREATA

ESTUDIO DE EXTENSIÓN ABIERTO, MULTICÉNTRICO, PARA EVALUAR LA EFICACIA Y SEGURIDAD A LARGO PLAZO DE CTP-543 EN PACIENTES ADULTOS CON ALOPECIA AREATA DE MODERADA A GRAVE

A.3.1

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

Study to assess the long-term efficacy and safety following administration of CTP-543 in adult patients with moderate to severe alopecia areata

Estudio para evaluar la eficacia y seguridad a largo plazo tras la administración de CTP-543 en pacientes adultos con alopecia areata de moderada a grave

A.4.1

Sponsor's protocol code number

CP543.5002

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05041803

A.5.4

Other Identifiers

Name:

IND

Number:

131,423

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	Concert 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	Concert 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	Linical France SARL
B.5.2	Functional name of contact point	Medical Manager
B.5.3	Address:
B.5.3.1	Street Address	52 Take Ionescu Boulevard
B.5.3.2	Town/ city	Timisoara
B.5.3.3	Post code	300073
B.5.3.4	Country	Romania
B.5.4	Telephone number	+40256207271
B.5.5	Fax number	+40256207273
B.5.6	E-mail	diana.chera@linical.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.3.1	Product name	deuruxolitinib (Tablet 8 mg every 12 hours)
D.3.2	Product code	CTP-543
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	deuruxolitinib phosphate
D.3.9.1	CAS number	2147706-60-1
D.3.9.2	Current sponsor code	CTP-543 phosphate
D.3.9.3	Other descriptive name	C-21543; D8-RUXOLITINIB
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	8
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	deuruxolitinib (Tablet 12 mg every 12 hours)
D.3.2	Product code	CTP-543
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	deuruxolitinib phosphate
D.3.9.1	CAS number	2147706-60-1
D.3.9.2	Current sponsor code	CTP-543 phosphate
D.3.9.3	Other descriptive name	C-21543; D8-RUXOLITINIB
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	12
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

MODERATE TO SEVERE ALOPECIA AREATA IN ADULT PATIENTS

ALOPECIA AREATA EN PACIENTES ADULTOS DE MODERADA A GRAVE

E.1.1.1

Medical condition in easily understood language

EPISODE OF HAIR LOSS ASSOCIATED WITH ALOPECIA AREATA IN ADULT PATIENTS

EPISODIO DE PÉRDIDA DE CABELLO ASOCIADO A ALOPECIA AREATA EN PACIENTES ADULTOS

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10001761
E.1.2	Term	Alopecia areata
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue disorders

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 overall objectives of the study are to evaluate long-term safety of CTP-543 and to assess long-term effects of CTP-543 on treating hair loss in adult patients with moderate to severe alopecia areata

Los objetivos generales del estudio son evaluar la seguridad a largo plazo de CTP-543 y valorar los efectos a largo plazo de CTP-543 en el tratamiento de la pérdida del cabello en pacientes adultos con alopecia areata de moderada a grave.

E.2.2

Secondary objectives of the trial

Not applicable

No aplica

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Written informed consent, and authorization for release and use of protected health information.
2.Have completed a 24-week Treatment Period in a previous qualifying CTP-543 clinical trial.
3.Female subjects are eligible to participate if at least one of the following conditions applies:
a) Is a woman of childbearing potential (WOCBP) and using a medically highly effective form of birth control with a failure rate less than 1% per year from at least 4 weeks prior to Baseline until at least 30 days following last dose of study drug. Examples of medically highly effective birth control methods include:
i.Combined (estrogen and progestogen containing) hormonal contraception (oral, patch, vaginal ring)
ii.Progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
iii.Intrauterine device or intrauterine hormone-releasing system
iv.Bilateral tubal occlusion
v.Vasectomized partner (partner is the sole sexual partner of the WOCBP trial participant and the vasectomized partner has received medical assessment of the surgical success)
vi.Sexual abstinence (reliable as refraining from heterosexual intercourse during the above-mentioned period)
b) Is not a WOCBP:
i. Premenopausal with one of the following:
a. Documented hysterectomy;
b. Documented bilateral salpingectomy;
c. Documented bilateral oophorectomy.
ii. Postmenopausal (cessation of menses for at least 12 months prior to screening)
Postmenopausal is defined as no menses for 12 months without an alternative medical cause. In addition, a high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm postmenopausal in women under 60 years old and not using hormonal contraception or hormone replacement therapy (HRT). However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. Females on HRT and whose menopausal status is in doubt will be required to use one of the nonestrogen hormonal highly effective contraception methods if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of postmenopausal status before study enrollment
4.Male participants must:
a.Agree to use, with their partners, male contraception (condom) and one of the highly effective contraceptive methods listed in Inclusion Criterion 3, from Baseline until at least 90 days following last dose of study drug.
b.Refrain from donating sperm during the study and for at least 90 days after the end of the study.
5.Willing to comply with the study visits and requirements of the study protocol.

1. Consentimiento informado por escrito y autorización para la divulgación y el uso de información médica protegida.
2. Haber completado un Periodo de Tratamiento de 24 semanas en un ensayo clínico de CTP-543 que haya cumplido los requisitos.
3. Las mujeres son aptas para participar si se cumple al menos una de las siguientes condiciones:
a) Ser una mujer en edad fértil (MEF) y utilizar un método anticonceptivo altamente eficaz desde el punto de vista médico con una tasa de fallo inferior al 1 % al año desde al menos 4 semanas antes del Inicio hasta al menos 30 días después de la última dosis del fármaco del estudio. Algunos ejemplos de métodos anticonceptivos de gran eficacia médica son los siguientes:
i. Anticoncepción hormonal combinada (con estrógeno y progestágeno) (oral, parche, anillo vaginal)
ii. Anticonceptivo hormonal solo con progestágeno asociado a la inhibición de la ovulación (oral, inyectable, implantable)
iii. Dispositivo intrauterino o sistema intrauterino liberador de hormonas
iv. Oclusión tubárica bilateral
v. Tener pareja vasectomizada (la pareja es la única pareja sexual de la participante MEF que participa en el ensayo y la pareja vasectomizada ha recibido una evaluación médica satisfactoria de la intervención)
vi. Abstinencia sexual (fiable se define como la abstinencia de relaciones heterosexuales durante el periodo mencionado).
b) No es una MEF:
i. Premenopáusica con una de las siguientes características:
a. Histerectomía documentada;
b. Salpingectomía bilateral documentada;
c. Ovariectomía bilateral documentada.
ii. Posmenopáusica (cese de la menstruación durante al menos 12 meses antes de la selección)
Posmenopáusica se define como ausencia de menstruación durante 12 meses sin una causa médica alternativa. Además, se puede utilizar un nivel alto de hormona foliculoestimulante (FSH) en el intervalo posmenopáusico para confirmar la posmenopausia en mujeres menores de 60 años y que no utilizan anticonceptivos hormonales o terapia de reemplazo hormonal (TRH). Sin embargo, en ausencia de 12 meses de amenorrea, una sola medición de FSH es insuficiente. A las mujeres en TRH y cuyo estado de menopausia sea dudoso deberán utilizar alguno de los métodos anticonceptivos hormonales de gran eficacia sin estrógenos si desean continuar con el TRH durante el estudio. En caso contrario, deben interrumpir el TRH para permitir la confirmación del estado posmenopáusico antes de la inclusión en el estudio
4. Los participantes de sexo masculino deben:
a. Aceptar el uso, con sus parejas, de la anticoncepción masculina (preservativo) y alguno de los métodos anticonceptivos de gran eficacia mencionados en el criterio de inclusión 3, desde el Inicio hasta al menos 90 días después de la última dosis del fármaco del estudio.
b. Abstenerse de donar esperma durante el estudio y durante al menos 90 días después del final del estudio.
5. Estar dispuesto a cumplir con las visitas y los requisitos del protocolo del estudio.

E.4

Principal exclusion criteria

1.Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or other scalp condition that may interfere with the SALT assessment, or untreated actinic keratosis anywhere on the body.
2.Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study medication.
3.Donation of blood at any point throughout the study and for 30 days after last dose of study medication.
4.Most recent hematologic parameters do not permit continued dosing; i.e., criteria for withholding IP have been met and have not recovered to values required to resume dosing.
5.Any medical, psychiatric, or social condition that is likely to unfavorably affect the risk-benefit of continued study participation, interfere with study compliance, or confound safety or efficacy assessments.

1. Inflamación activa del cuero cabelludo, psoriasis o dermatitis seborreica que requiera tratamiento tópico en el cuero cabelludo, traumatismo significativo en el cuero cabelludo u otra afección en el cuero cabelludo que pueda interferir en la evaluación con SALT o queratosis actínica no tratada en cualquier parte del cuerpo.
2. Mujeres en período de lactancia, embarazadas o que prevean quedarse embarazadas mientras participan en el estudio y durante 30 días después de la última dosis del medicamento del estudio.
3. Donación de sangre en cualquier momento a lo largo del estudio y durante 30 días después de la última dosis del medicamento del estudio.
4. La mayoría de los parámetros hematológicos recientes no permiten continuar con la administración; es decir, se han cumplido los criterios para suspender la administración del PEI y no se han recuperado los valores necesarios para reanudarla.
5. Cualquier problema médico, psiquiátrico o social que pueda afectar desfavorablemente a la relación riesgo-beneficio de la participación continuada en el estudio, interferir con el cumplimiento del estudio o confundir las evaluaciones de seguridad o eficacia.

E.5 End points

E.5.1

Primary end point(s)

Safety of CTP-543 will be assessed by evaluating adverse event, clinical laboratories, physical examinations, vital signs, concomitant medications, and electrocardiogram results.
Efficacy of CTP-543 will include all patients who receive study drug and have at least 1 post-treatment SALT assessment in this study. Relative change in SALT score over time will be summarized descriptively by visit, as appropriate.

La seguridad de CTP-543 se evaluará mediante la valoración de los acontecimientos adversos, las mediciones de laboratorio, las exploraciones físicas, las constantes vitales, los medicamentos concomitantes y los resultados del electrocardiograma.
La eficacia de CTP-543 incluirá a todos los pacientes que reciben el fármaco del estudio y tienen al menos 1 evaluación SALT posterior al tratamiento en este estudio. El cambio relativo en la puntuación SALT a lo largo del tiempo se resumirá de forma descriptiva por visita, según corresponda.

E.5.1.1

Timepoint(s) of evaluation of this end point

After at least 1 post-treatment SALT assessment (week 4) in this study

Después de al menos 1 evaluación SALT posterior al tratamiento (semana 4) en este estudio

E.5.2

Secondary end point(s)

Not applicable

No aplica

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

No aplica

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

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)

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

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

Information not present in EudraCT

E.8.1.4

Double blind

Information not present in EudraCT

E.8.1.5

Parallel group

Information not present in EudraCT

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

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

E.8.6.2

Trial being conducted completely outside of the EEA

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

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:

280

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

290

F.4.2.2

In the whole clinical trial

290

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)

None

Ninguno

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

2022-01-31

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-12-02

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

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