Clinical Trials Register

Summary
EudraCT Number:	2021-001442-35
Sponsor's Protocol Code Number:	PTZ
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-06-07
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-001442-35

A.3

Full title of the trial

A PHASE 2 MONOCENTRIC PILOT STUDY TO EVALUATE THE EFFICACY AND SAFETY PROFILE OF PIOGLITAZONE IN SUBJECTS WITH ACTIVE NON-SEGMENTAL VITILIGO

STUDIO PILOTA DI FASE 2 PER LA VALUTAZIONE DELL’EFFICACIA E DEL PROFILO DI SICUREZZA DEL PIOGLITAZONE IN SOGGETTI CON VITILIGINE NON SEGMENTALE IN FASE ATTIVA

A.3.1

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

A PHASE 2 MONOCENTRIC PILOT STUDY TO EVALUATE THE EFFICACY AND SAFETY PROFILE OF PIOGLITAZONE IN SUBJECTS WITH VITILIGO

STUDIO PILOTA DI FASE 2 PER LA VALUTAZIONE DELL’EFFICACIA E DEL PROFILO DI SICUREZZA DEL PIOGLITAZONE IN SOGGETTI CON VITILIGINE

A.3.2

Name or abbreviated title of the trial where available

PIOGLITAZONE IN VITILIGO

PIOGLITAZONE NELLA VITILIGINE

A.4.1

Sponsor's protocol code number

PTZ

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	ISTITUTI FISIOTERAPICI OSPITALIERI
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support
B.4.2	Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	ISTITUTI FISIOTERAPICI OSPITALIERI
B.5.2	Functional name of contact point	UOC FISIOPATOLOGIA CUTANEA
B.5.3	Address:
B.5.3.1	Street Address	via Elio Chianesi 53
B.5.3.2	Town/ city	Roma
B.5.3.3	Post code	00144
B.5.3.4	Country	Italy
B.5.4	Telephone number	0652666257
B.5.5	Fax number	0652666247
B.5.6	E-mail	mauro.picardo@ifo.gov.it

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	Yes
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	Pioglitazone
D.3.2	Product code	[PTZ]
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	PIOGLITAZONE CLORIDRATO
D.3.9.2	Current sponsor code	PTZ
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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

NON SEGMENTAL VITILIGO WITH BSA BETWEEN 2% AND 50% AND A NON-SATISFACTORY RESPONSE (LESS THAN 10%) TO A PREVIOUS PHOTOTHERAPY CYCLE

VITILIGINE NON SEGMENTALE CON ESTENSIONE (BSA) COMPRESO TRA 2% E 50% E CON UNA SCARSA RISPOSTA (INFERIORE AL 10%) AD UN PRECEDENTE TRATTAMENTO FOTOTERAPICO

E.1.1.1

Medical condition in easily understood language

DIFFUSE VITILIGO WITH A NON-SATISFACTORY RESPONSE TO PHOTOTHERAPY

VITILIGINE DIFFUSA SCARSAMENTE RESPONSIVA ALLA FOTOTERAPIA

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	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10047643
E.1.2	Term	Vitiligo vulgaris
E.1.2	System Organ Class	100000004858

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the efficacy of Pioglitazone alone or in association with nbUVB in adult subjects with non-segmental vitiligo.

Valutare l'efficacia del Pioglitazone da solo o in associazione alla fototerapia UVB a banda stretta in soggetti adulti affetti da vitiligine non-segmentale

E.2.2

Secondary objectives of the trial

To evaluate the safety and tolerability of Pioglitazone over time in adult subjects with active non-segmental vitiligo.
To evaluate the efficacy of Pioglitazone alone or in association with nbUVB as measured by other clinical assessments over time in adult subjects with active non-segmental vitiligo

Valutare la sicurezza e la tollerabilità del Pioglitazone in soggetti adulti affetti da vitiligine non-segmentale
Valutare l'efficacia del Pioglitazone da solo o in associazione alla fototerapia UVB a banda stretta in soggetti adulti affetti da vitiligine non-segmentale misurata con altre valutazioni cliniche

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. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
2. Willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.

3. Male or female subjects between 18-65 years of age, inclusive, at time of informed consent.

4. Female subjects of childbearing potential and at risk for pregnancy must agree to use one method of contraception throughout the study and for at least 28 days after the last dose of assigned treatment.

Female subjects of non-childbearing potential must meet at least 1 of the following criteria:

a. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with a serum follicle-stimulating hormone (FSH) level confirming the postmenopausal state;

b. Have undergone a documented hysterectomy and/or bilateral oophorectomy;

c. Have medically confirmed ovarian failure.

All other female subjects (including female subjects with tubal ligations) are considered childbearing potential.
5. Must meet the following non-segmental vitiligo criteria at the Screening Visit and the Baseline Visit:
a. Have a clinical diagnosis of non-segmental vitiligo for at least 3 months; and

b. Body surface area (BSA) involvement 2% - 50%; and

c. BSA ¿0.2% involvement on the face (confirmed by photographs); and

d. Have obtained <10% response in previous phototherapy treatment.

6. If receiving concomitant medications for any reason other than vitiligo, must be on a stable regimen, which is defined as not starting a new drug or changing dosage within 7 days prior to Day 1. Subject must be willing to stay on a stable regimen during the duration of the study

7. Must agree to avoid prolonged exposure to the sun and not to use tanning booths, sun lamps or other ultraviolet light sources other than provided/requested by the study team during the study

1. Firma del consenso informato
2. Maschi o femmine di età compresa tra i 18-65 anni
3. Le donne in età fertile devono accettare l’utilizzo di un metodo contraccettivo per tutta la durata dello studio e almeno 28 giorni successivi all’ultima dose di farmaco assunta
4. Soggetti affetti da vitiligine non-segmentale da almeno 3 mesi, con superficie corporea (BSA) compresa tra 2% e 50%, BSA sul viso di almeno 0.2% e che abbiano ottenuto una risposta <10% ad un precedente trattamento fototerapico
5. Se il paziente riceve altri farmaci per altre indicazioni deve essere su un regime stabile da almeno 7 giorni
6. Il paziente deve consentire a limitare il più possibile la prolungata esposizione solare e a non esporsi a fonti artificiali di luce UV

E.4

Principal exclusion criteria

1. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator,
2. Subjects considered in imminent need for surgery (for example in the next 6 months) or with elective surgery scheduled to occur during the study.

3. Subjects that have other types of vitiligo (including but not limited to segmental vitiligo). Note: Mixed vitiligo is permitted.

4. Currently have active forms of other hypopigmentation . Note: Coexistence of halo nevus/nevi (also known as Sutton nevus/nevi) is permitted.

5. Currently have active forms of inflammatory skin disease(s) or evidence of skin conditions at the time of the Screening or Day 1 Visit that in the opinion of the investigator would interfere with evaluation of vitiligo or response to treatment.

6. Other acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator or sponsor, would make the subject inappropriate for entry into this study.
7. Pregnant female subjects; breastfeeding female subjects; and female subjects of childbearing potential who are unwilling or unable to use one method of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product.

8. Have current or recent history of clinically significant severe, progressive, or uncontrolled disease; or have any other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration, or interfere with the interpretation of study results; history of severe allergic or anaphylactoid reaction; or in the opinion of the investigator, the subject is inappropriate for entry into this study, or unwilling/unable to comply with Study Procedures and Lifestyle Requirements.

9. History of type I or II diabetes

10. Have a history of any lymphoproliferative disorder such as Epstein Barr Virus (EBV) related lymphoproliferative disorder, history of lymphoma, history of leukemia, or signs and symptoms suggestive of current lymphatic or lymphoid disease.

11. Have a history of systemic infection requiring hospitalization, parenteral antimicrobial therapy, or as otherwise judged clinically significant by the investigator within 6 months prior to Day 1.

12. Have active acute or chronic skin infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 4 weeks prior to Day 1, or superficial skin infections within 2 weeks prior to Day 1. NOTE: patients may be rescreened after the infection resolves.

13. Have a history of alcohol or substance abuse within 6 months prior to Day 1 that in the opinion of the investigator will preclude participation in the study or protocol adherence in the study.

14. Have a known immunodeficiency disorder or a first-degree relative with a hereditary immunodeficiency.

15. Have any malignancies or have a history of malignancies including adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.

16. Have undergone significant trauma or major surgery within 1 month of the first dose of IP

17. ANY specific abnormalities in clinical laboratory tests at screening, as assessed by the study-specific laboratory and confirmed by a single repeat,

•
•
• In the opinion of the investigator, have any uncontrolled clinically significant laboratory abnormality that would affect interpretation of study data or the subject’s participation in the study.

1. Membri dello staff e delle loro famiglie
2. Soggetti con interventi chirurgici programmati nei 6 mesi successivi all’arruolamento
3. Soggetti affetti da altri tipi di vitiligine (per es. vitiligine segmentale)
4. Soggetti affetti da altre forme di ipopigmentazione che possano alterare la valutazione della vitiligine (la coesistenza di nevi di Sutton è permessa)
5. Soggetti affetti da altre forme di patologie infiammatorie cutanee attive allo screening che possano interferire con la valutazione della vitiligine
6. Altre condizioni cliniche acute o croniche e/o anormalità di laboratorio che possano aumentare il rischio di partecipazione allo studio
7. Donne in gravidanza e/o allattamento; donne in età fertile che rifiutano l’uso di contraccettivi
8. Storia clinica attuale o recente di patologie progressive, severe o incontrollate
9. Diabete di tipo I o II
10. Storia di patologie linfoproliferative
11. Storia di patologie infettive sistemiche
12. Infezioni cutanee acute o croniche
13. Storia di abuso di alcool
14. Qualsiasi disturbo di immunodeficienza
15. Pregresse neoplasie
16. Specifiche anormalità allo screening di laboratorio (v. protocollo)

E.5 End points

E.5.1

Primary end point(s)

vitiligo area scoring index (VASI) at Week 12

vitiligo area scoring index (VASI) alla settimana 12

E.5.1.1

Timepoint(s) of evaluation of this end point

At week 12

a 12 settimane

E.5.2

Secondary end point(s)

• Incidence of treatment-emergent adverse events (AEs) and serious adverse events (SAEs) up to Week 16.; • Percent change from baseline in VASI, facial-VASI, vitiligo extent score (VES), and self-assessment VES (SA-VES) and absolute change from baseline in VASI at designated time points

Incidenza di eventi avversi correlati al farmaco ed eventi avversi severi fino alla 16 settimana; Variazione percentuale dal baseline del VASI, VASI facciale, vitiligo extent score (VES), e self-assessment VES (SA-VES) e cambiamenti assoluti nel VASI a specifici time-points

E.5.2.1

Timepoint(s) of evaluation of this end point

At weeks 16; at week 12

alla 16ma settimana; a settimana 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

E.6.5

Efficacy

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)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

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

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Farmaco versus farmaco+fototerapia

Drug versus drug+phototherapy

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

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

Information not present in EudraCT

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

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

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

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

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

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)

According to standard of care

Secondo normale pratica clinica

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

2021-06-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-03-23

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

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