Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sol-Gel Technologies Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

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

Trial duration

23 May 2024 → ongoing

Decision date (initial)

2024-04-16

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Sol-Gel Technologies Ltd.

External identifiers

EU CT number

2023-507528-21-00

ClinicalTrials.gov

NCT06050122

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Therapy, Efficacy, Safety

To assess the efficacy of Patidegib Gel 2% in subjects with Gorlin syndrome

Secondary objectives 1

1. To assess the safety and tolerability of Patidegib Gel 2% in subjects with Gorlin syndrome treated for 12 months

Conditions and MedDRA coding

Gorlin Syndrome

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. The subject must be at least 18 years old at the Screening Visit
2. The subject must be confirmed to have a PTCH1 mutation
3. The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1)
4. The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator

Exclusion criteria 5

1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP)
2. The subject is known to have hypersensitivity to any of the ingredients in the IP formulation
3. The subject has uncontrolled systemic disease
4. The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast
5. Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) on the face of subjects with Gorlin syndrome as assessed by the CPRB

Secondary endpoints 17

1. Number of New Surgically Eligible BCCs (nSEBs) at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) on the face of subjects with Gorlin syndrome as assessed by the CPRB
2. Change from Baseline to Month 12 in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion symptoms scale score
3. Change from Baseline to Month 12 in confirmed BCCs lesion diameter
4. Proportion of Surgically Eligible BCCs (SEBs) and nSEBs assessed as resolved by Month 12
5. Proportion of SEBs and nSEBs per subject that have regressed by Month 12
6. Proportion of subjects developing ≥2 facial new BCCs at Month 12
7. Change in total number of confirmed BCCs from Baseline to Month 12
8. Change from Baseline to Month 12 in the Skin Cancer Index quality of life questionnaire score
9. Adverse events (AEs), including serious adverse events (SAEs) occurring at any time during the study
10. Investigator Dermal Safety and Local Tolerability Assessments score at any time during the study
11. Change from Baseline to Month 12 in Patient Global Impression in disease Severity (PGI-S)
12. Change in Patient Global Impression of Change in clinical status (PGI C) at Month 12
13. Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) on the face of subjects with Gorlin syndrome
14. Number of nSEBs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) on the face of subjects with Gorlin syndrome in the ITT analysis set for this study (SGT 610 01)
15. Time to first new BCC
16. Skin pain and peak pruritus scores as reported by the subject on a numeric rating scale
17. Number of nSEBs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) on the face of subjects with Gorlin syndrome in the pooled ITT analysis set from this study (SGT-610-01) and the pre-specified ITT subset of Study Pelle-926-301 restricted to subjects who met SGT 610-01 inclusion criteria

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sol-Gel Technologies Ltd.

Sponsor organisation

Sol-Gel Technologies Ltd.

Address

7 Golda Meir

City

Ness Ziona

Postcode

7403650

Country

Israel

Scientific contact point

Organisation

Sol-Gel Technologies Ltd.

Contact name

Clinical team member

Public contact point

Organisation

Sol-Gel Technologies Ltd.

Contact name

Clinical team member

Third parties 7

Locations

6 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 100 files

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

Application history

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