Overview
Sponsor-declared trial summary
Inflammatory EPidermal GENodermatoses
To describe efficacy of deucravacitinib over the 3 periods of a challenge-dechallenge-rechallenge design like study in adults with inflammatory congenital ichthyosis (ICI) erythrodermic congenital ichtyosis (CEI) /severe keratinopathic ichtyosis (KI) or severe simplex EB (EBS-sev).
Key facts
- Sponsor
- Centre Hospitalier Universitaire De Nice
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Skin and Connective Tissue Diseases [C17]
- Trial duration
- 30 Jan 2024 → ongoing
- Decision date (initial)
- 2023-09-25
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
To describe efficacy of deucravacitinib over the 3 periods of a challenge-dechallenge-rechallenge design like study in adults with inflammatory congenital ichthyosis (ICI) erythrodermic congenital ichtyosis (CEI) /severe keratinopathic ichtyosis (KI) or severe simplex EB (EBS-sev).
Secondary objectives 4
- To describe safety of deucravacitinib treatment over the study period
- To describe the evolution of the following efficacy and health outcomes measures within each study period: -Severity-Itch-Pain -QoL
- To study the patients’ compliance during treatment periods
- To describe cytokines evolution over the study follow-up
Conditions and MedDRA coding
Inflammatory EPidermal GENodermatoses
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10014989 | Epidermolysis bullosa | 100000004850 |
| 20.0 | LLT | 10010511 | Congenital ichthyosis | 10010331 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Patient > 18 years of both sexes
- Patient with a laboratory confirmed diagnosis of EBS due to KRT5 or 14 mutation or CIE or with KI due to KR1 orKRT10 mutation
Exclusion criteria 9
- Subjects with another form of ICI ie Netherton syndrome, Kid syndrome etc
- Infectious/Immune-related Exclusions
- Medical History and Concurrent Diseases
- Subject has another member of her/his immediate family (i.e., living in the same household) enrolled in this study.
- Subject has used any diacerein containing product within 6 months prior to Visit 1.
- Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
- Subject has used systemic steroidal therapy within 30 days prior to Visit 1.
- Subject has used any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study assessments within 30 days prior to Visit 1.
- Subject has a history of sensitivity to any of the ingredients of deucravacitinib.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The aim of this study is to assess the efficacy of deucravacitinib in the treatment of patients with EBS-sev and inflammatory congenital ichthyosis
Secondary endpoints 4
- Safety of deucravacitinib treatment
- Secondary efficacy and health outcomes measures : severity, itch, pain, quality of life
- Compliance
- Baseline and functional cytokine profile
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD9836762 · Product
- Active substance
- Deucravacitinib
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 6 mg milligram(s)
- Max total dose
- 6 mg milligram(s)
- Max treatment duration
- 32 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Universitaire De Nice
- Sponsor organisation
- Centre Hospitalier Universitaire De Nice
- Address
- 4 Avenue Reine Victoria
- City
- Nice
- Postcode
- 06000
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Universitaire De Nice
- Contact name
- Christine CHIAVERINI
Public contact point
- Organisation
- Centre Hospitalier Universitaire De Nice
- Contact name
- Eric MONCH
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 10 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2024-01-30 | 2024-03-18 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 16 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D4_Patient Facing documents_EB V1_2022-502879-32-00 | 1.1 |
| Protocol (for publication) | D4_Patient Facing documents_EB V3_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing documents_EB V4 6 7_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing documents_EB V5_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing Documents_EB_V2_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing documents_IC V4-6-7_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing documents_IC V5_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing Documents_IC_V2_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | D4_Patient Facing documents_IC_V3_2022-502879-32-00 | 2.0 |
| Protocol (for publication) | Protocol 2022-502879-32-00 | 4.0 |
| Recruitment arrangements (for publication) | Template Recruitment arrangement | 0.0 |
| Subject information and informed consent form (for publication) | *sis and ICF adultes ichtyose | 1.0 |
| Subject information and informed consent form (for publication) | SIS and ICF adultes EB1 | 3.0 |
| Synopsis of the protocol (for publication) | 2022-502879-32-00_PROTOCOL SYNOPSIS | 0-1 |
| Synopsis of the protocol (for publication) | protocol synopsis 2022-502879-32-00 | 4.0 |
| Synopsis of the protocol (for publication) | protocol synopsis lay people 2022-502879-32-00 | 0.1 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-06-15 | France | Acceptable 2023-09-21
|
2023-09-25 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-10-12 | France | Acceptable 2024-01-10
|
2024-01-10 |
| 3 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-05-31 | France | Acceptable 2024-07-02
|
2024-07-22 |
| 4 | SUBSTANTIAL MODIFICATION | SM-4 | 2024-10-11 | France | Acceptable 2024-10-31
|
2024-11-27 |
| 5 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-02-10 | France | Acceptable 2025-03-24
|
2025-04-11 |
| 6 | SUBSTANTIAL MODIFICATION | SM-6 | 2025-09-05 | France | Acceptable 2025-10-01
|
2025-10-17 |