“A phase 2 multi-center study investigating the efficacy, safety and tolerability of pioglitazone in adult participants with non-segmental vitiligo”

Overview

Sponsor-declared trial summary

Key facts

Sponsor

I.F.O. Istituti Fisioterapici Ospitalieri

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

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

Decision date (initial)

2025-11-26

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

The specific object of the present clinical trial is the use of a PPARγ selective activator for the treatment of vitiligo, such as non-segmental vitiligo. The selective PPARγ activator chosen is a thiazolidinediones (TZDs), the pioglitazone (PGZ) will be used simultaneously to the phototherapy treatment.

Conditions and MedDRA coding

Vitiligo

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Subjects must meet all the following inclusion criteria at both screening and baseline to be eligible for enrollment into the study: i) Adults between 18 years and 65 years of all genders; ii) Has a clinical diagnosis of non-segmental vitiligo with depigmented areas; iii) Has a face involvement BSA ≥0.3% at screening and baseline. The face will not include the scalp, ears, neck, or surface area of the lips but will include the nose and the eyelids; iv) Has a Total Vitiligo Scoring Index (T-VASI) ≥3 at screening and baseline; v) Has a Facial Vitiligo Area Scoring Index (F-VASI) ≥0.3 at screening and baseline. vi) Previous failure of at least one first-line treatment (e.g. topical corticosteroids or calcineurin inhibitors). vii) Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study; viii) Agree to discontinue all other vitiligo treatments from screening until the final follow-up visit in accordance with the washout times reported below in Table 2. ix) Female subjects of childbearing potential (WOCBP) and at risk for pregnancy must agree to use a highly effective method of contraception (confirmed by the investigator) throughout the study and for at least 30 days after the last PGZ dose of assigned treatment and have a negative pregnancy test result on visit 1 and each 4 weeks during the entire study; x) Female subjects of non-childbearing potential must meet at least 1 of the following criteria: • 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; • Have undergone a documented hysterectomy and/or bilateral oophorectomy; • Have medically confirmed ovarian failure. • All other female subjects (including female subjects with tubal ligations) are considered childbearing potential.

Exclusion criteria 1

1. i. Unable to consent (adults lacking capacity); ii. Participants that have a diagnosis of other types of vitiligo including segmental and mixed vitiligo; iii. Has ≥50% leukotrichia on face or body; iv. Unable to return for follow-up visits; v. Pregnant women and/or breastfeeding, or those who have recently delivered a baby within the past 6 months; vi. Has any other dermatological diseases that would interfere with vitiligo assessments; vii. History of type I or II diabetes viii. Has a transplanted organ, which requires continued immunosuppression; ix. Has a history of any oncological malignancy; x. Familiar atypical multiple mole and melanoma (FAMMM) syndrome; xi. Systemic lupus erythematosus; xii. Dermatomyositis; xiii. Genetic conditions that predispose an individual to skin cancer (Gorlin syndrome, xeroderma pigmentosum); xiv. Bloom syndrome or Cockayne syndrome; xv. Has had major surgery within 3 months before Screening OR has a major surgery planned during the study; xvi. Has hypersensitivity to the active substance or to any of the excipients; xvii. Has cardiac failure or history of cardiac failure (NYHA stages I to IV); xviii. Has current bladder cancer or a history of bladder cancer xix. Has uninvestigated macroscopic haematuria. xx. Any evidence of fluid overload xxi. Prior/Concurrent investigational clinical study experience; xxii. Enrolled in a clinical study of any other investigational drug or device; xxiii. Liver disease, or kidney disease; xxiv. Hypoglycemia as defined by fasting blood glucose <70 mg/dL assessed at a fasting study visit; xxv. ANY of the following abnormalities in clinical laboratory tests at screening, as assessed by the study-specific laboratory and confirmed by a single repeat, if deemed necessary: xxvi. Absolute neutrophil count of <2.5 x 109/L (<2500/mm3); xxvii. Hemoglobin <10.0 g/dL or hematocrit <30%; xxviii. Platelet count below the lower limit of normal (LLN) at Screening; xxix. Absolute lymphocyte count of <0.8 x 109 /L (<800/mm3); xxx. Serum creatinine > upper limit of normal (ULN) or eGFR <60 ml/min/1.73m2 based on the age-appropriate calculation. xxxi. Enzymes aspartate aminotransferase (AST) or alanine aminotransferase (ALT) values >2 times the ULN; xxxii. Total bilirubin 1.5 times the ULN; subjects with a history of Gilbert's syndrome may have a direct bilirubin measured and would be eligible for this study provided the direct bilirubin is ULN; xxxiii. Creatine kinase (CK) >3 times the ULN and positive urine myoglobin; xxxiv. 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. xxxv. Patients who, in the opinion of the investigator, are unable or unlikely to comply with the administration schedule and study evaluation.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. - Proportion of subjects achieving F-VASI50 (defined as at least 50% improvement in F-VASI from baseline) at Week 16

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

I.F.O. Istituti Fisioterapici Ospitalieri

Sponsor organisation

I.F.O. Istituti Fisioterapici Ospitalieri

Address

Via Elio Chianesi N 53

City

Rome

Postcode

00144

Country

Italy

Scientific contact point

Organisation

I.F.O. Istituti Fisioterapici Ospitalieri

Contact name

PRINCIPAL INVESTIGATOR

Public contact point

Organisation

I.F.O. Istituti Fisioterapici Ospitalieri

Contact name

PRINCIPAL INVESTIGATOR

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 9 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications