Clinical Trials Register

Summary
EudraCT Number:	2020-000253-27
Sponsor's Protocol Code Number:	Pelle-926-301E
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-06-17
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2020-000253-27

A.3

Full title of the trial

A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects with Gorlin Syndrome (Basal Cell Nevus Syndrome)

Studio di estensione di fase III, in aperto, multicentrico su patidegib gel topico 2% in soggetti con sindrome di Gorlin (sindrome del carcinoma basocellulare nevoide)

A.3.1

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

A Phase 3, Multicenter, unblinded Extension Study of Patidegib Topical Gel, 2% in patient with Gorlin Syndrome

Studio di estensione di fase III, in aperto, multicentrico su patidegib gel topico 2% in soggetti con sindrome di Gorlin

A.3.2

Name or abbreviated title of the trial where available

PATIDEGIB Phase 3 OLE study in subjects with Gorlin syndrome

Pelle-926-301E

A.4.1

Sponsor's protocol code number

Pelle-926-301E

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	PellePharm, 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	PellePharm, 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	PellePharm, Inc.
B.5.2	Functional name of contact point	VP, Clinical Development
B.5.3	Address:
B.5.3.1	Street Address	101 Mission Street, Suite 1950
B.5.3.2	Town/ city	San Francisco
B.5.3.3	Post code	CA 94105
B.5.3.4	Country	United States
B.5.4	Telephone number	6505809025
B.5.6	E-mail	spendyala@pellepharm.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.2.1.2	Country which granted the Marketing Authorisation	Italy
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/18/1998
D.3 Description of the IMP
D.3.1	Product name	Patidegib Topical Gel, 2%
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Gel
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Topical use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	Patidegib
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/W) percent weight/weight
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
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

Basal cell carcinomas (BCCs) in patients with Gorlin syndrome

sindrome di Gorlin (sindrome del carcinoma basocellulare nevoide)

E.1.1.1

Medical condition in easily understood language

Skin cancer in patients with Gorlin syndrome

sindrome di Gorlin (sindrome del carcinoma basocellulare nevoide)

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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	10004151
E.1.2	Term	Basal cell nevus syndrome
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of the study is to assess the safety and tolerability of Patidegib Topical Gel, 2% in patients who have completed PellePharm Study Pelle-926-201 or Pelle-926-301

Valutare la sicurezza e la tollerabilità di patidegib gel topico 2% in pazienti che hanno completato lo studio Pelle-926-201 o lo studio Pelle-926-301 di PellePharm

E.2.2

Secondary objectives of the trial

The secondary objective of the study is to assess the efficacy of Patidegib Topical Gel, 2% in patients who complete PellePharm Study Pelle-926-201 or Pelle-926-301

Valutare l’efficacia di patidegib gel topico 2% in pazienti che hanno completato lo studio Pelle-926-201 o lo studio Pelle-926-301 di PellePharm

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. The subject must be at least 18 years old.
2. The subject must provide written informed consent prior to any study procedures.
3. The subject must have completed PellePharm Study 926-201 or 926-301 with adequate compliance
4. Study 926-301 subjects must have completed the End of Treatment Visit in Study 301, prior to the Screening Visit in this study. They must also complete all Study 301 related procedures prior to the Baseline Visit of this study.
5. The subject must meet diagnostic criteria for Gorlin (basal cell nevus) syndrome, including major criterion #a plus 1 additional major criterion or plus 2 additional minor criteria listed in Appendix 17.2
6. The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator. Moisturizers and emollients are allowed. Subjects will be encouraged to use their preferred sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all exposed skin sites.
7. Female subjects must have a negative pregnancy test (serum pregnancy test at Screening Visit, urine pregnancy test at Baseline Visit). For Study 301 subjects, a negative serum pregnancy test result from Study 301 is acceptable if the test was done within 7 days of the Screening Visit of this study.
8. If the subject is a woman of child bearing potential (WOCBP), she must be willing to use birth control methods which may be considered highly effective (Appendix 17.1). Hormonal contraception must be supplemented with a barrier method (preferably condom). Birth control must start prior to Baseline, continue through the duration of the study, and for 30 days after last application of IP
9. If the subject is a male with a female sex partner who is a WOCBP, the subject must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 3 months after the last application of IP.
10. The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator
11. The subject is willing to forego treatment of facial BCCs with anything other than the study IP except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. In such instances, the only other allowed form of treatment is surgical.

1 il soggetto deve aver compiuto almeno 18 anni
2. il soggetto deve presentare il suo consenso scritto prima di iniziare le procedure dello studio
3. Il soggetto deve aver completato lo studio 926-201 o lo studio 926-301 di PellePharm.
4. I soggetti dello studio Pelle-926-301 devono aver completato la visita di fine trattamento dello studio 301 prima della visita di screening di questo studio.
5. Il soggetto deve soddisfare i criteri diagnostici per la sindrome di Gorlin (nevo cellulare basale), incluso il criterio principale #a più 1 criterio maggiore aggiuntivo o più 2 criteri minori aggiuntivi elencati nell'appendice 17.2
6. Il soggetto deve essere disposto ad astenersi dall’applicare farmaci topici non previsti dallo studio (su prescrizione o da banco) sulla pelle del viso per tutta la durata dello studio, salvo se prescritti dallo sperimentatore. Sono ammessi prodotti idratanti ed emollienti. I soggetti saranno incoraggiati ad utilizzare ogni giorno, su tutte le aree cutanee esposte, la loro protezione solare preferita con un fattore di protezione solare (SPF) di almeno 30.
7.I soggetti di sesso femminile devono ottenere un risultato negativo al test di gravidanza (sul siero alla visita di screening e sulle urine alla visita basale). Per i soggetti provenienti dallo studio 301 è accettabile un risultato negativo ottenuto ad un test di gravidanza sul siero eseguito in tale studio qualora esso sia stato effettuato entro 7 giorni dalla visita di screening di questo studio.
8. Se il soggetto è una donna potenzialmente fertile, deve essere disposta ad utilizzare metodi contraccettivi che possono essere considerati altamente efficaci (Appendice 17.1). La contraccezione ormonale deve essere integrata con un metodo di barriera (preferibilmente il preservativo). La contraccezione deve essere usata da prima del basale per tutta la durata dello studio e per 30 giorni dopo l’ultima applicazione del prodotto sperimentale (Investigational Product, IP).
9. Se il soggetto è un uomo con una partner sessuale potenzialmente fertile, deve essere disposto ad usare il preservativo, anche a seguito di una vasectomia, da prima del basale per tutta la durata dello studio e per almeno 3 mesi dopo l’ultima applicazione dell’IP.
10. Il soggetto è disposto a far valutare tutti i carcinomi basocellulari (Basal Cell Carcinoma, BCC) al viso e a seguire esclusivamente le raccomandazioni di trattamento dello sperimentatore.
11 Il soggetto è disposto a rinunciare al trattamento dei BCC al viso con qualsiasi altro prodotto diverso dall’IP dello studio, eccetto nel caso in cui lo sperimentatore ritenga che il ritardo nel trattamento di un BCC possa potenzialmente compromettere la salute del soggetto. In tali casi, l’unica forma di trattamento alternativa consentita è quella chirurgica.

E.4

Principal exclusion criteria

1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP. Among these are use of the following:
a. 5-fluorouracil, imiquimod, or Ingenol mebutate (except as topical treatment to anatomical areas other than the face) systemically or topically to the skin within the 2 months prior to the Screening and Baseline Visit.
b. Systemic chemotherapy within 1 year prior to the Screening and Baseline Visit.
c. Known inhibitors of the Hedgehog signaling pathway (e.g., vismodegib, sonidegib, itraconazole) topically (except as topical treatment to anatomical
areas other than the face) or systemically within 2 months prior to the Screening and Baseline Visit.
d. Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 2 months prior to the Screening and Baseline Visit.
2. The subject has a known hypersensitivity to any of the ingredients in the study IP formulation.
3. The subject is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests.
4. The subject has current, recent (within five half lives of the experimental drug or if half life not known, within the past 6 months prior to the Screening Visit), or planned participation in an experimental drug study (excluding Study 926-301) while enrolled in this study.
5. The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
6. The subject is pregnant or breastfeeding.
7. The subject has any condition or situation which, in the Investigator’s opinion, may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject’s participation in the study. This may include a history of other skin conditions (e.g., severe facial eczema) or diseases, metabolic dysfunction, physical examination (PE) findings, or clinical laboratory findings giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the subject at high risk from treatment complications.

1 Il soggetto ha utilizzato un trattamento topico sul viso o terapie sistemiche che potrebbero interferire con la valutazione dell’IP dello studio Tra questi ci sono le seguenti:
a. un. 5-fluorouracile, imiquimod o Ingenol mebutate (tranne come trattamento topico in aree anatomiche diverse dal viso) sistemicamente o topicamente alla pelle entro i 2 mesi precedenti la visita di screening e basale.
b. Chemioterapia sistemica entro 1 anno dalla visita di screening e basale.
c. Inibitori noti della via di segnalazione "Hedgehog" (ad es. Vismodegib, sonidegib, itraconazolo) per via topica (tranne come trattamento topico in aree anatomiche diverse dalla faccia) o sistematicamente entro 2 mesi prima della visita di screening e basale.
2. Il soggetto ha una nota ipersensibilità a uno qualsiasi degli ingredienti nella formulazione IP dello studio.
3. Il soggetto non è in grado o non è disposto al recarsi al centro per le visite e i test di studio.
4. Il soggetto sta partecipando attualmente, ha partecipato di recente (entro cinque emivita del farmaco sperimentale o, se l’emivita non è nota, negli
ultimi 6 mesi prima della visita di screening) o ha in programma di partecipare (durante l’arruolamento a questo studio) a uno studio su un farmaco
sperimentale (escluso lo studio 926-301).
5. Il soggetto è una donna potenzialmente fertile che non è disposta o non è in grado di rispettare le misure di prevenzione della gravidanza.
6. Il soggetto è in stato di gravidanza o sta allattando al seno.
7. Secondo l'opinione dell'Investigatore, il soggetto non è nelle condizioni/situazione di partecipare allo studio e può mettere il soggetto a rischio significativo,compromettendone i risultati o interferendo in modo significativo sullo studio. Questo può includere altre condizioni della pelle (ad esempio, eczema facciale grave) o malattie, disfunzione metabolica, risultati di esami fisici (PE) o risultati di laboratorio clinici che danno il ragionevole sospetto di una malattia o condizione che controindica l'uso di un farmaco sperimentale o che potrebbe influire sull'interpretazione dei risultati dello studio o rendere il soggetto ad alto rischio a causa di complicanze del trattamento.

E.5 End points

E.5.1

Primary end point(s)

The number of facial BCCs removed by surgery is considered an appropriate study endpoint as surgery has significant morbidity in Gorlin Syndrome (e.g., scarring, functional loss of eyelid, nose, ear).

Il numero di BCC facciali rimossi dalla chirurgia è considerato un endpoint di studio appropriato in quanto la chirurgia ha una morbilità significativa nella sindrome di Gorlin (ad esempio, cicatrici, perdita funzionale delle palpebre, del naso, dell'orecchio).

E.5.1.1

Timepoint(s) of evaluation of this end point

At all quarterly visits

ad ogni visita, trimestrale

E.5.2

Secondary end point(s)

The efficacy endpoints are as follows:
1. The number of facial BCCs removed by surgery (per Investigator determination) and histologically verified as BCC; from Baseline to Month 12
2. The number of facial BCCs removed by surgery (per Investigator determination) and histologically verified as BCC; from Baseline to Month 6.
3. The number of facial BCCs removed by surgery (per Investigator determination) from Baseline to Month 12.
4. The number of facial BCCs removed by surgery (per Investigator determination) from Baseline to Month 6.
5. Change from Baseline to Month 12 in the total number of facial lesions suspicious for BCC
6. Count of facial lesions suspicious for BCC over time
7. aBCCdex change in lesion score from Baseline to Month 12

Gli endpoint di efficacia sono i seguenti:
1. Il numero di BCC facciali rimossi dall'intervento chirurgico (secondo la determinazione dello sperimentatore) e istologicamente verificati come BCC; dal basale al mese 12
2. Il numero di BCC facciali rimossi dall'intervento chirurgico (secondo la determinazione dello sperimentatore) e istologicamente verificati come BCC; dal basale al 6 ° mese.
3. Il numero di BCC facciali rimossi dall'intervento chirurgico (per determinazione dello sperimentatore) dal basale al mese 12.
4. Il numero di BCC facciali rimossi dall'intervento chirurgico (per determinazione dello sperimentatore) dal basale al 6 ° mese.
5. Variazione dal basale al mese 12 del numero totale di lesioni facciali sospette di BCC
6. Conteggio delle lesioni facciali sospette per BCC nel tempo
7. aBCCdex modifica del punteggio della lesione dal basale al mese 12

E.5.2.1

Timepoint(s) of evaluation of this end point

Please see section E.5.2

vedere sezione E5.2

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

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

E.8.1.2

Open

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

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

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

Canada

United States

Belgium

Denmark

France

Germany

Italy

Netherlands

Spain

United Kingdom

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)

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

Yes

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

200

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)

All subjects should return to their personal physicians to resume standard of care for their condition.

Tutti i soggetti dovrebbero tornare dai loro medici personali per riprendere le cure standard

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

2020-07-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-06-16

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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