Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
32
Countries
1
Sites
11
Patients displaying a non-classified, severe and resistant inflammatory disease
To evaluate the efficacy of targeted treatments in patients displaying a non-classified, severe and resistant inflammatory disease. Targeted treatments for each patient will have been selected through an algorithm based on molecular analysis of specific altered inflammatory signaling pathway.
Key facts
- Sponsor
- Assistance Publique Hopitaux De Paris
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20], Diseases [C] - Skin and Connective Tissue Diseases [C17]
- Trial duration
- 9 Sep 2024 → 20 Nov 2025
- Decision date (initial)
- 2024-09-09
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- French Ministry of Health PHRCN 2016
External identifiers
- EU CT number
- 2024-513647-86-00
- EudraCT number
- 2017-000519-18
- ClinicalTrials.gov
- NCT03651518
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
To evaluate the efficacy of targeted treatments in patients displaying a non-classified, severe and resistant inflammatory disease. Targeted treatments for each patient will have been selected through an algorithm based on molecular analysis of specific altered inflammatory signaling pathway.
Secondary objectives 1
- (a) to analyze the safety at several time points of targeted treatment selected through molecular based algorithm, (b) to analyze the other clinical global and cutaneous efficacy parameters (c) to study whether the treatment selected has been able to inhibit the culprit pathway using mRNA analysis before and after treatment as well as serum markers before and after treatment (d) to assess whether full RNA sequencing can depict in lesional skin, overexpressed pathway(s) that the targeted RNA set has not detected
Conditions and MedDRA coding
Patients displaying a non-classified, severe and resistant inflammatory disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10062249 | Skin inflammation | 10040785 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- -Patients (men or women) aged 18 years old and over -Patients presenting inflammatory non classified disease targeting at least 2 organs involvement: skin, lymph nodes, hemopoietic system, joints, digestive tract, eye, nerves and brain tissues, respiratory tract, cardio-vascular disorders, genito-urinary tract including kidney, musculo-skeletal tissues. Skin involvement is mandatory in order to be able to compare involved and non involved tissue -Signed informed consent The disease should be considered as non-classified despite classical and adapted investigations and evaluation through expert assessment of the Scientific committee meeting (cf paragraph 10.2). The disease alters significantly quality of life. The impairment of quality of life will be assessed based on the investigator’s assessment. The disease has been resistant to at least two prior lines of treatment [for example : Hydroxychloroquine, Chloroquine, Colchicine, Methotrexate, Ciclosporine, Azathioprine, Mycophenolate mofetil, Disulone, Corticosteroids (prednisone, prednisolone, dexamethasone, methylprednisolone…)].
Exclusion criteria 1
- -Patients presenting disease which is not featured by lesional and healthy skin areas, easy to biopsy -Patients refusing biopsies -Pregnancy -Women of child-bearing potential unable to receive highly efficient contraception such as combined oral contraceptives, intra-uterine disposals, hormonal implants or the use of male condoms recommended in case of unstable or irregular partner or as a replacement method for transient unacessebility to hormonal method -Breastfeeding -Patients presenting disease needing urgent therapeutic measures -Patients without health insurance or social security -Participation in another interventional trial -Patients under legal protection -Patients unable to respect the wash out delay of previously taken medications before biopsy and before treatment initiation -Patients with contra-indications to treatments
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Response will be assessed at month 6 after treatment initiation with a composite endpoint defined as improvement of at least 2 of the 3 following parameters: - 50% improvement of the systemic activity assessed by the clinician following a visual analog scale (0-10), - and/or 50% improvement of cutaneous activity assessed by the involved skin surface area, - and/or 50% decrease or nomalisation of biological markers of inflammation (either CRP, ESR or fibrin)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 6
SCP15622163 · ATC
- Active substance
- Ustekinumab
- Substance synonyms
- Bmab 1200, CNTO 1275, BAT1406, ABP-654, CNTO-1275, BAT2206, CT-P43
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 45 mg milligram(s)
- Max total dose
- 135 mg milligram(s)
- Max treatment duration
- 20 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AC05 — USTEKINUMAB
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP183367 · ATC
- Active substance
- Anakinra
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 100 mg milligram(s)
- Max total dose
- 20300 mg milligram(s)
- Max treatment duration
- 29 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AC03 — ANAKINRA
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP107114078 · ATC
- Active substance
- Adalimumab
- Substance synonyms
- ABP 501, BI 695501, MSB11022
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 80 mg milligram(s)
- Max total dose
- 480 mg milligram(s)
- Max treatment duration
- 29 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB04 — ADALIMUMAB
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP1642996 · ATC
- Active substance
- Secukinumab
- Substance synonyms
- Recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class, AIN457
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 3000 mg milligram(s)
- Max treatment duration
- 28 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AC10 — SECUKINUMAB
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP176238 · ATC
- Active substance
- Tocilizumab
- Substance synonyms
- RO4877533, BIIB800, ATLIZUMAB, TOCILIZUMABUM
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 800 mg milligram(s)
- Max total dose
- 5600 mg milligram(s)
- Max treatment duration
- 28 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AC07 — TOCILIZUMAB
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP24437829 · ATC
- Active substance
- Rituximab
- Substance synonyms
- CT-P10, PF-05280586, ABP 798, BI 695500, JHL1101, HLX01
- Route of administration
- INTRAVENOUS PERFUSION USE
- Max daily dose
- 1000 mg milligram(s)
- Max total dose
- 3000 mg milligram(s)
- Max treatment duration
- 28 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XC02 — RITUXIMAB
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Assistance Publique Hopitaux De Paris
- Sponsor organisation
- Assistance Publique Hopitaux De Paris
- Address
- Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
- City
- Paris Cedex 10
- Postcode
- 75475
- Country
- France
Scientific contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Pr Selim ARACTINGI
Public contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Pr Selim ARACTINGI
Locations
1 EU/EEA country · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 32 | 11 |
| Rest of world | — | 0 | — |
Investigational sites
Hospices Civils De Lyon
Dermatologie, Pavillon R, 5 Place D Arsonval, 69437, Lyon Cedex 03
Assistance Publique Hopitaux De Paris
Dermatologie et Allergologie et médecine vasculaire, 4 Rue De La Chine, 75020, Paris
Assistance Publique Hopitaux De Paris
Dermatologie, 51 Av Du Mal De Lattre De Tassigny, 94000, Creteil
Centre Hospitalier Universitaire De Lille
Médecine interne, Rue Michel Polonovski, 59037, Lille Cedex
Assistance Publique Hopitaux De Paris
Hématologie Clinique Adulte, 149 Rue De Sevres, 75015, Paris
Assistance Publique Hopitaux De Paris
Médecine interne, 184 Rue Du Faubourg Saint Antoine, 75012, Paris
Les Hopitaux Universitaires De Strasbourg
Dermatologie, 1 Place De L Hopital, 67000, Strasbourg
Assistance Publique Hopitaux De Paris
Dermatologie, 1 Avenue Claude Vellefaux, 75010, Paris
Assistance Publique Hopitaux De Paris
Médecine interne, 27 Rue Du Faubourg Saint Jacques, 75014, Paris
Les Hopitaux Universitaires De Strasbourg
Rhumatologie, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Assistance Publique Hopitaux De Paris
Dermatologie, 27 Rue Du Faubourg Saint Jacques, 75014, 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 | 2024-09-09 | 2025-11-20 | 2024-09-09 | 2024-09-25 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 10 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-513647-86-00 | 8-0 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements | 1-0 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF adulte | 6-0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Cosentyx | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Imraldi | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Kineret | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Rixathon | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Roactemra | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Stelara | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_FR 2024-513647-86-00 | 8-0 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-09 | France | Acceptable 2024-09-03
|
2024-09-09 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-03-07 | France | Acceptable 2025-04-08
|
2025-04-17 |