Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - First administration to humans
Status
Ongoing, recruiting
Participants planned
38
Countries
1
Sites
10
Inclusion Body Myositis
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] - Nervous System Diseases [C10]
- Trial duration
- 24 Oct 2024 → ongoing
- Decision date (initial)
- 2024-10-24
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-511858-44-00
- EudraCT number
- 2020-005876-36
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Inclusion Body Myositis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10029323 | Neuromyopathy | 100000004852 |
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
- Investigateur coordonnateur
Public contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Investigateur coordonnateur
Locations
1 EU/EEA country · 10 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 38 | 10 |
| Rest of world | — | 0 | — |
Investigational sites
Assistance Publique Hopitaux De Paris
Centre d'Investigation Neuromusculaire, 43 Boulevard De L Hopital, 75013, Paris
Centre Hospitalier Universitaire Rouen
Service de Chirurgie plastique et de la main, 1 Rue De Germont, Bp 96031, Rouen Cedex
Centre Hospitalier Regional De Marseille
Centre de thérapie cellulaire, 147 Boulevard Baille, 13005, Marseille
Centre Hospitalier Universitaire Rouen
Immunologie et thérapie cellulaire, 1 Rue De Germont, 76000, Rouen
Centre Hospitalier Regional De Marseille
Chirurgie de la main - Chirurgie plastique réparatrice des membres, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Regional De Marseille
Centre de référence des maladies neuromusculaires, 264 Rue Saint Pierre, 13005, Marseille
Assistance Publique Hopitaux De Paris
Service de neuromyologie, 43 Boulevard De L Hopital, 75013, Paris
Assistance Publique Hopitaux De Paris
Médecine interne et immunologie clinique, 43 Boulevard De L Hopital, 75013, Paris
Aix Marseille University
Centre de Résonance Magnétique en Biologie et Médecine, 27 Boulevard Jean Moulin, 13005, Marseille
Assistance Publique Hopitaux De Paris
Centre d'investigation neuromusculaire, 43 Boulevard De L Hopital, 75013, 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 | 2024-10-24 | 2024-10-24 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Corrective measures 2 · Art. 77 CTR
Corrective measure CM-FR-0001
- Member state
- France
- Publication date
- 2025-12-22
- Type
- 4
- Reason
- 7
- Immediate action required
- No
- Justification
- As part of its activities, the APHM sent an email 26/11/2025 to the ANSM to inform it of an unexpected change in the design of a critical medical device (MD): the diameter of one of the tubes in the PUREGRAFT bag, used for the initial collection of adipose tissue and for the first stages of manufacture. This modification, which could impact the quality of the sterile connection, required an immediate adaptation of the process.
This change affects six clinical trials:2024-516342-19-00 2024-513306-65-00,2024-517384-24-
00,2023-505149-20-00 2024-517794-24-00 et 2024-511858-44-00
In order to guarantee the quality and sterility of the product, a new transfer set has been introduced. This set, custom-made by Sartorius, is subjected to gamma irradiation to meet sterility requirements. From a quality management perspective, this change has been formally managed, incorporating all prior validations: documentary, aseptic, MPT, surgical use, leak testing, as well as the associated validation batches.
The IMPD will be gradually updated during 2026 to ensure the traceability and regulatory compliance of this change.
The sponsor is requested to submit immediately an MSA part I + II inclunding
- The applicant must provide a photo/diagram or detailed description of the new device (transfer set) and the contact time with the product.
- Proof of CE marking or the device's certificate of conformity must be provided.
- The documents available as mentioned in the initial email regarding prior validations of the device (documentary, aseptic, MPT, surgical use, watertightness, validation batches) must be provided.
- The complete change control report must be provided.
so that the ANSM can evaluate the change in sterile connection and allow it to continue to be included in this clinical trial
Corrective measure CM-FR-0002
- Member state
- France
- Publication date
- 2026-02-25
- Type
- 4
- Reason
- 6, 7
- Immediate action required
- No
- Justification
- linked to CM-FR-0001 concerning the 2024-511858-44-00 in particularly site of APHM.
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-03 | France | Acceptable 2024-10-18
|
2024-10-24 |