Clinical Trials Register

Summary
EudraCT Number:	2021-002365-18
Sponsor's Protocol Code Number:	CP543.5002
National Competent Authority:	Hungary - National Institute of Pharmacy
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-12-06
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Hungary - National Institute of Pharmacy

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

MULTICENTRIKUS, NYÍLT, KITERJESZTETT VIZSGÁLAT A CTP-543 HOSSZÚ TÁVÚ BIZTONSÁGOSSÁGÁNAK ÉS HATÁSOSSÁGÁNAK ÉRTÉKELÉSÉRE FELNŐTT, MÉRSÉKELTEN SÚLYOS / SÚLYOS ALOPECIA AREATAS BETEGEKNÉL

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

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	Sun Pharmaceutical Industries, 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	Sun Pharmaceutical Industries, 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
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.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

E.1.1.1

Medical condition in easily understood language

EPISODE OF HAIR LOSS ASSOCIATED WITH ALOPECIA AREATA IN ADULT PATIENTS

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

E.2.2

Secondary objectives of the trial

Not applicable

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.Írásos tájékoztatás utáni beleegyezés, valamint engedély a védett egészségügyi adatok kiadására és felhasználására.
2.A beteg befejezte a 24-hetes kezelési időszakot egy korábbi minősítő CTP-543 klinikai vizsgálatban.
3.A női alanyok akkor alkalmasak a részvételre, ha az alábbi feltételek közül legalább egy teljesül:
a)Fogamzóképes nő (woman of childbearing potential – WOCBP) és orvosilag nagyon hatékony fogamzásgátló módszert használ – amelynél a hibaarány kevesebb mint 1% évente, a kiindulás előtti legalább 4. héttel a vizsgálati készítmény utolsó adagja utáni legalább 30 napig. Példák az orvosilag nagyon hatékonynak számító fogamzásgátló módszerekre:
i.Kombinált (ösztrogént és progesztogént tartalmazó) hormonális fogamzásgátlás (orális, tapasz, hüvelygyűrű)
ii.Csak progesztogént tartalmazó hormonális fogamzásgátlás az ovuláció gátlásával (orális, injekció, implantátum)
iii.Méhen belüli eszköz vagy méhen belüli hormonkibocsátó rendszer
iv.Kétoldali petevezeték-elzárás
v.Vazektomizált partner (a partner a WOCBP vizsgálati résztvevő egyedüli szexuális partnere, és a műtét sikerét orvosilag értékelték a vazektomizált partnernél)
vi.Szexuális absztinencia (megbízható abban, hogy tartózkodik a heteroszexuális közösüléstől a fent említett időszak alatt)
b)Nem WOCBP:
i.Premenopauza az alábbiak egyikével:
a.Dokumentált hiszterektómia;
b.Dokumentált kétoldali szalpingektómia;
c.Dokumentált kétoldali petefészek-eltávolítás.
ii.Posztmenopauzában van (a szűrés előtt legalább 12 hónapja nem menstruál)
A posztmenopauza úgy határozható meg, hogy 12 hónapja nem jelentkezett menstruáció, és ennek nincs más orvosi oka. Ezenkívül a posztmenopauzális tartományban lévő magas follikulus stimuláló hormon (FSH) szint használható a posztmenopauza megerősítésére 60 év alatti nőknél, akik nem használnak hormonális fogamzásgátlást vagy hormonpótló terápiát (hormone replacement therapy – HRT). Azonban 12 hónapos amenorrhoea hiányában egyetlen FSH-mérés nem elegendő. A HRT-t alkalmazó nőknek, akiknek a menopauzális státusza kétséges, az ösztrogént nem tartalmazó hormonális, nagy hatékonyságú fogamzásgátló módszerek egyikét kell alkalmazniuk, ha a HRT-t a vizsgálat során folytatni kívánják. Egyébként abba kell hagyniuk a HRT-t, hogy lehetővé tegyék a posztmenopauzális státusz megerősítését a vizsgálatba való bevonás előtt.
4.A férfi résztvevőknek kötelezően:
a.Bele kell egyezniük, hogy partnereikkel férfi fogamzásgátlást (óvszert) és a 3. beválasztási kritériumban felsorolt, nagy hatékonyságú fogamzásgátló módszerek egyikét alkalmazzák a kiindulástól a vizsgálati készítmény utolsó adagja után legalább 90 napig.
b.Tartózkodniuk kell a spermaadományozástól a vizsgálat ideje alatt és a vizsgálat vége után még legalább 90 napig.
5.Hajlandó betartani a vizsgálati viziteket és a vizsgálati protokoll követelményeit.

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.Aktív fejbőrgyulladás, pikkelysömör vagy seborrheás dermatitisz, amely helyi kezelést igényel a fejbőrön, jelentős fejbőrsérülés vagy egyéb fejbőrbetegség, amely zavarhatja a SALT-értékelést, vagy kezeletlen aktinikus keratózis a test bármely részén.
2.Olyan nők, akik szoptatnak, terhesek vagy teherbe kívánnak esni a vizsgálat ideje alatt és a vizsgálati készítmény utolsó adagja után még 30 napig.
3.Véradás a vizsgálat során bármikor és a vizsgálati készítmény utolsó adagja után még 30 napig.
4.A legutóbbi hematológiai paraméterek nem teszik lehetővé a folyamatos adagolást, azaz az IP-visszatartás kritériumai teljesültek, és az adagolás folytatásához szükséges értékekre nem tértek vissza.
5.Bármilyen orvosi, pszichiátriai vagy szociális állapot, amely valószínűleg kedvezőtlenül befolyásolja a vizsgálatban való további részvétel kockázat-előny arányát, nem egyeztethető össze a vizsgálati előírások betartásával, vagy zavarja a biztonságossági vagy hatásossági értékeléseket.

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.

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

E.5.2

Secondary end point(s)

Not applicable

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

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

E.8.1.4

Double blind

E.8.1.5

Parallel group

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:

403

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

407

F.4.2.2

In the whole clinical trial

407

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

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

2022-01-18

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-01-04

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

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials