Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
20
Countries
1
Sites
5
Epidermolysis bullosa simplex
To study the efficacy of apremilast over the 3 periods of a challenge-dechallenge-rechallenge design like study in patients ≥ 6 years presenting with EBS-sev.
Key facts
- Sponsor
- Centre Hospitalier Universitaire De Nice
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Skin and Connective Tissue Diseases [C17]
- Trial duration
- 10 Jan 2025 → ongoing
- Decision date (initial)
- 2024-06-18
- 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 study the efficacy of apremilast over the 3 periods of a challenge-dechallenge-rechallenge design like study in patients ≥ 6 years presenting with EBS-sev.
Secondary objectives 4
- To describe safety of apremilast treatment over the study period
- To describe the evolution of the following efficacy and health outcomes measures within each study period: - Severity (patient and clinician global assessment) - Itch and pain (visual analogic scales) - Duration of dressing - QoL (adult and children dermatological quality of life scales) -EB severity scores
- To study the patients’ compliance during treatment periods
- To validate a new score developed to evaluate EBS-sev severity
Conditions and MedDRA coding
Epidermolysis bullosa simplex
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 25.0 | LLT | 10087382 | Epidermolysis bullosa simplex | 100000004848 |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-000715-PIP03-11
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Male or female patients 6 years or older
- Laboratory confirmed diagnosis of EBS-sev due to KRT5 or 14 mutation (autosomal)
- Mean daily number of new blisters >4.
- Subject/caregiver agrees not to use any topical therapies other than the investigator approved
Exclusion criteria 5
- EBS lesions requiring oral therapy to treat an infection
- Use of any diacerein containing product within 6 months prior to Visit 1
- Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
- Use of systemic steroidal therapy within 30 days prior to Visit 1
- Use of 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
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The aim of this study is to assess the efficacy of apremilast in the treatment of patients with EBS-sev.
Secondary endpoints 4
- Safety and tolerability will be assessed through the description of the following specific events occurring during the treatment periods of the study plus 1 week to be conservative
- Secondary efficacy and health outcomes measures:Severity, itch, pain, Duration of dressing, Quality of life
- compliance
- Validation of a new severity scale for EBS-sev patients
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
Otezla 10mg, 20mg, 30 mg film-coated tablets
PRD7877792 · Product
- Active substance
- Apremilast
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA32 — -
- Marketing authorisation
- EU/1/14/981/001
- MA holder
- AMGEN EUROPE B.V.
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Otezla 10mg, 20mg, 30 mg film-coated tablets
PRD7877790 · Product
- Active substance
- Apremilast
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA32 — -
- Marketing authorisation
- EU/1/14/981/001
- MA holder
- AMGEN EUROPE B.V.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Otezla 30 mg film-coated tablets
PRD7877794 · Product
- Active substance
- Apremilast
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA32 — -
- Marketing authorisation
- EU/1/14/981/002
- MA holder
- AMGEN EUROPE B.V.
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Otezla 10mg, 20mg, 30 mg film-coated tablets
PRD7877791 · Product
- Active substance
- Apremilast
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA32 — -
- Marketing authorisation
- EU/1/14/981/001
- MA holder
- AMGEN EUROPE B.V.
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- 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
- Christine CHIAVERINI
Locations
1 EU/EEA country · 5 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 20 | 5 |
| Rest of world | — | 0 | — |
Investigational sites
Centre Hospitalier Universitaire De Toulouse
dermatologie, 24 Chemin De Pouvourville, 31400, Toulouse
Centre Hospitalier Regional Et Universitaire De Brest
Dermatology, 2 Avenue Marechal Foch, 29200, Brest
Hopital Necker Enfants Malades
Dermatology, 149 Rue De Sevres, 75015, Paris
Centre Hospitalier Universitaire De Nice
Dermatology, 151 Route De Saint Antoine, 06200, Nice
Assistance Publique Hopitaux De Paris
Dermatology, 1 Avenue Claude Vellefaux, 75010, Paris
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 | 2025-01-10 | 2025-06-17 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 22 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_protocol_2023-508794-83-00 | 3.0 |
| Protocol (for publication) | D4_diary_ inf 50kg_visite3 | 1.0 |
| Protocol (for publication) | D4_diary_ sup 50kg_visite2 | 1.0 |
| Protocol (for publication) | D4_diary_ sup 50kg_visite3 | 1.0 |
| Protocol (for publication) | D4_diary_inf 50kg_visite2 | 1.0 |
| Protocol (for publication) | D4_diary_inf 50kg_visite5 | 1.0 |
| Protocol (for publication) | D4_diary_sup 50kg_visite5 | 1.0 |
| Protocol (for publication) | D4_diary_visite1 | 0.0 |
| Protocol (for publication) | D4_diary_visite4 | 1.0 |
| Recruitment arrangements (for publication) | K1- Recruitment arrangement | 1.0 |
| Subject information and informed consent form (for publication) | L1 SIS partenaire femme | 0.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 16 17 ans_ | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 6 12 ans | 0.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_13-15 | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_13-15 track change | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_16 17 ans_track change | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adulte | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_parent | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_patientdevenumajeur | 3.2 |
| Subject information and informed consent form (for publication) | L1_SIS femme enceinte | 0.1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2 otezla | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2023-508794-83-00 | 2.0 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-03-12 | France | Acceptable 2024-06-04
|
2024-06-18 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-09-23 | France | Acceptable 2024-06-04
|
2024-09-23 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-09-24 | France | Acceptable 2024-10-30
|
2024-11-28 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-01-09 | France | Acceptable 2024-10-30
|
2025-01-09 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-07-25 | France | Acceptable 2024-10-30
|
2025-07-25 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-11-21 | France | Acceptable 2026-01-28
|
2026-02-27 |