A phase II study of Sonidegib in Nevoid Basal Cell Carcinoma Syndrome (Gorlin) syndrome and sporadic BCC patients in EADO stage IIb. SIBLINGS TRIAL

2024-515989-14-00 Protocol SIBLINGS Therapeutic exploratory (Phase II) Ongoing, recruiting

Start 17 Apr 2025 · Status Ongoing, recruiting · 1 EU/EEA countries · 8 sites · Protocol SIBLINGS

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Ongoing, recruiting
Participants planned 15
Countries 1
Sites 8

Nevoid Basal Cell Carcinoma Syndrome

Treatment compliance in an NBCCS - population / sporadic BCC patients in EADO stage IIb treated for 16 weeks with daily sonidegib followed by a pulsed dosing schedule maintained for the next 24 weeks.

Key facts

Sponsor
Gruppo Oncologico Del Nord Ovest
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Neoplasms [C04]
Trial duration
17 Apr 2025 → ongoing
Decision date (initial)
2025-02-21
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes
Funding sources
Sun Pharma ltd

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

Treatment compliance in an NBCCS - population / sporadic BCC patients in EADO stage IIb treated for 16 weeks with daily sonidegib followed by a pulsed dosing schedule maintained for the next 24 weeks.

Secondary objectives 4

  1. The activity of a pulsed sonidegib regimen after 16 weeks with daily sonidegib.
  2. The efficacy of a pulsed sonidegib regimen after 16 weeks with daily sonidegib.
  3. The safety of a pulsed sonidegib regimen after 16 weeks with daily sonidegib.
  4. The impact on quality of life of a pulsed sonidegib regimen after 16 weeks with daily sonidegib.

Conditions and MedDRA coding

Nevoid Basal Cell Carcinoma Syndrome

VersionLevelCodeTermSystem organ class
20.0 LLT 10078878 Nevoid basal-cell carcinoma syndrome 10010331

Study design 1 period

#TitleAllocationBlindingRoles blindedArms
1 SIBLINGS TRIAL
This is a phase II, single arm open label clinical trial aiming to assess treatment compliance in stage II B NBCCS population treated for 16 weeks with daily sonidegib followed by a pulsed dosing schedule maintained for the next 24 weeks.
 2 None Single arm: The study is divided in 2 phases, each treatment cycle lasts 4 weeks:
- induction phase: 16 weeks full dose;
- maintenance phase: 24 weeks of pulsed dose.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

  1. Be willing and able to provide written informed consent for the trial.
  2. Be ≥ 18 years of age on day of signing informed consent.
  3. One of the following conditions: - subject with established diagnosis of NBCCS; - subject with common\sporadic BCC.
  4. Clinical stage IIB according to EADO Classification [Very high number of common BCC (>10) or multiple complex BCC (> 5) in the setting of apparently sporadic cases or in Gorlin syndrome. When at least 1 of the multiple BCC can be classified III or IV, the patient will be classified accordingly, and not IIB].
  5. Being deemed difficult to treat: not amenable for surgery or RT after the evaluation of a multidisciplinary team involving at least a dermatologist and a medical oncologist and therefore eligible for HHI therapy.
  6. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

Exclusion criteria 5

  1. Distant metastatic disease (M1), visceral and/or distant nodal.
  2. Prior HHI therapy in the last 2 years.
  3. Received previous systemic treatment for BCC; local therapy (e.g. imiquimod) are allowed.
  4. Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
  5. Hypersensitivity to sonidegib or any of its excipients.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. A discontinuation rate <54% due to AEs at 40 weeks from study entry in a NBCCS - population/sporadic BCC patients in EADO stage IIb treated for 16 weeks with daily sonidegib followed by a pulsed dosing for the subsequent 24 weeks.

Secondary endpoints 5

  1. The ORR according to RECIST criteria.
  2. The PFS at 40 weeks from study entry.
  3. The DOR of a pulsed sonidegib regimen.
  4. The rate of any grade drug related (DR) AEs and G3/4 DR AEs of a pulsed sonidegib regimen in a NBCCS - population / sporadic BCC patients in EADO stage IIb after 16 weeks with daily sonidegib.
  5. The EORTC QLQ C30 score at baseline, after 16 weeks of continuous daily sonidegib therapy, and after 24 weeks of pulsed sonidegib regimen.

Investigational products

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

Test 1

Odomzo 200 mg hard capsules

PRD5130543 · Product

Active substance
Sonidegib
Pharmaceutical form
CAPSULE, HARD
Route of administration
ORAL
Max daily dose
200 mg milligram(s)
Max total dose
39200 mg milligram(s)
Max treatment duration
40 Week(s)
Authorisation status
Authorised
ATC code
L01XJ02 — -
Marketing authorisation
EU/1/15/1030/002
MA holder
SUN PHARMACEUTICAL INDUSTRIES EUROPE B.V.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Gruppo Oncologico Del Nord Ovest

3 Total trials 1 Recruiting 1 Ended
Academic / Non-commercial
Sponsor organisation
Gruppo Oncologico Del Nord Ovest
Address
Via Goffredo Mameli 3/1
City
Genoa
Postcode
16122
Country
Italy

Scientific contact point

Organisation
Gruppo Oncologico Del Nord Ovest
Contact name
Paolo Bossi

Public contact point

Organisation
Gruppo Oncologico Del Nord Ovest
Contact name
Paolo Bossi

Third parties 1

OrganisationCity, countryDuties
Clinical Research Technology S.r.l.
ORG-100027504
 Salerno, Italy On site monitoring, Code 12, Code 5, Data management, E-data capture, Code 8

Locations

1 EU/EEA country · 8 investigational sites

By country

CountryMS statusPlanned subjectsSites
Italy Ongoing, recruiting 15 8
Rest of world — 0 —

Investigational sites

Italy

8 sites · Ongoing, recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
UOC Dermatologia, Largo Francesco Vito 1, 00168, Rome
Azienda Sanitaria Universitaria Giuliano Isontina
UOC Clinica Dermatologica, Via Costantino Costantinides 2, 34128, Trieste
Azienda Ospedaliera Universitaria Federico II Di Napoli
U.O.C. Dermatologia clinica, Via Sergio Pansini 5, 80131, Naples
Humanitas Mirasole S.p.A.
Oncologia Medica, Via Alessandro Manzoni 56, 20089, Rozzano
Istituto Europeo Di Oncologia S.r.l.
Oncologia Medica del Melanoma e Sarcomi, Via Giuseppe Ripamonti 435, 20141, Milan
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
SSD Dermatologia Oncologica, Via Pietro Albertoni 15, 40138, Bologna
Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
UOSD Dermatologia, Via Sergio Pansini 5, 80131, Naples
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Dipartimento Medicina Generale e Specialistica-Dermatologia, Corso Bramante 88, 10126, Turin

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Italy 2025-04-17 2025-10-22

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.

TypeTitleVersion
Protocol (for publication) 1_SIBLINGS - Study Protocol_clean_FP 3
Recruitment arrangements (for publication) K1_SIBLINGS_Recruitment arrangements - Italy_v1_0_29Aug2024 1
Subject information and informed consent form (for publication) SIBLINGS- EORTC QLQ-C30 v 3_FP 3
Subject information and informed consent form (for publication) SIBLINGS-Diario del Paziente_v1_0_29Aug2024 1
Subject information and informed consent form (for publication) SIBLINGS-Foglio informativo e ICF 2.0
Subject information and informed consent form (for publication) SIBLINGS-ICF_of_personal_data_v 1_0_29Aug2024_Clean 1
Summary of Product Characteristics (SmPC) (for publication) SIBLINGS_odomzo-product-information_en NA
Synopsis of the protocol (for publication) SIBLINGS - English synopsis_clean 3
Synopsis of the protocol (for publication) SIBLINGS - Sinossi in italiano_clean_FP 3

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-10-22 Italy Acceptable
2025-01-23
 2025-02-21
2 SUBSTANTIAL MODIFICATION SM-1 2025-02-25 Italy Acceptable 2025-04-04
3 NON SUBSTANTIAL MODIFICATION NSM-2 2025-04-09 Italy Acceptable 2025-04-09
4 SUBSTANTIAL MODIFICATION SM-2 2025-11-04 Italy Acceptable
2025-12-15
 2025-12-18