Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
190
Countries
1
Sites
34
Mycobacterium xenopi pulmonary infection
to demonstrate the superiority of ARIKAYCE addition to a standard treatment in term of 3-month sputum conversion rate compared to standard regimen alone in M. xenopi pulmonary infection (MX-PD).
Key facts
- Sponsor
- Centre Hospitalier Universitaire Amiens Picardie
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Bacterial Infections and Mycoses [C01]
- Trial duration
- 29 Oct 2025 → ongoing
- Decision date (initial)
- 2024-07-15
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- DIRECTION GENERALE DE L'OFFRE DE SOINS
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
to demonstrate the superiority of ARIKAYCE addition to a standard treatment in term of 3-month sputum conversion rate compared to standard regimen alone in M. xenopi pulmonary infection (MX-PD).
Secondary objectives 3
- time to culture conversion
- 6 months culture conversion
- 12 and 24 months mortality
Conditions and MedDRA coding
Mycobacterium xenopi pulmonary infection
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Two treatment regimens containing three molecules with or without ARIKAYCE. Two treatment regimens containing three molecules, rifampicin, ethambutol, and clarithromycin with or without ARIKAYCE® during the 6 first months of treatment.
|
Randomised Controlled | None | BRAS 1: rifampicin, ethambutol, and clarithromycin with ARIKAYCE® BRAS 2: rifampicin, ethambutol, and clarithromycin without ARIKAYCE® |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- 18 years old or older, must present 2020 ATS/ERS/ESCMID/IDSA criteria for nontuberculous mycobacterial pulmonary infection
- Clinical criteria Respiratory symptoms and the presence of nodular or cavitary lesion on chest x-ray, confirmed on chest high-resolution computed tomography. Lesions may also present in the form of diffuse micronodular syndrome.
- Microbiological criteria: o At least two positive cultures for MX on two sputum specimens obtained on two different days, at least separate of 7 days AND/OR o Positive culture for MX on bronchoalveolar lavage or bronchoscopic aspiration AND/OR o Transbronchial biopsy or surgical lung biopsy presenting histology in favour of mycobacterial infection (granuloma or positive Ziehl-Neelsen stain) and positive culture for MX OR biopsy showing histology compatible with mycobacterial infection and one or more sputum cultures positive for MX
- Exclusion of other diagnoses on CT scan, bronchoscopy and bacteriological specimens.
Exclusion criteria 14
- Known hypersensitivity to one of the molecules of the study
- Relapse of MX lung infection
- Treatment with molecules able to interfere with cytochrome P450 that cannot be replaced by another therapeutic class
- HIV 1 and 2 human immunodeficiency virus infection
- Renal failure with creatinine clearance less than 30 mL/min
- Pregnancy and breastfeeding
- Cystic fibrosis
- Contraindications to one of the antibiotic
- Inability to comply with the requirements of the protocol, especially substance abuse, according to the investigator.
- Limited life expectancy (e.g 36 months)
- Patients with hematologic malignancies and allogeneic haematopoietic stem cells
- Women of childbearing age and not using an effective method of contraception (Pearl Index <1%)
- The patient is treated with molecules prolonging the QT interval that cannot be replaced by another therapeutic class.
- The patient presents a heart failure with left ventricular ejection fraction less than 30%.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- 3 months sputum conversion rate
Secondary endpoints 1
- 6 months sputum conversion rate, recurrence, failure, relapse, cure, death and safety
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
ARIKAYCE liposomal 590 mg nebuliser dispersion
PRD8459878 · Product
- Active substance
- Amikacin
- Pharmaceutical form
- NEBULISER DISPERSION
- Route of administration
- INHALATION USE
- Max daily dose
- 590 mg milligram(s)
- Max total dose
- 590 mg milligram(s)
- Max treatment duration
- 6 Month(s)
- Authorisation status
- Authorised
- ATC code
- J01GB06 — AMIKACIN
- Marketing authorisation
- EU/1/20/1469/001
- MA holder
- INSMED NETHERLANDS B.V.
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Comparator 3
SCP4298332 · ATC
- Active substance
- Amoxicillin
- Substance synonyms
- AMOXICILLINE, AMOXICILLINUM
- Route of administration
- ORAL USE
- Max daily dose
- 1000 mg milligram(s)
- Max total dose
- 1000 mg milligram(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- J01FA09 — CLARITHROMYCIN
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP4890812 · ATC
- Active substance
- Rifampicin
- Substance synonyms
- RIFAMPIN
- Route of administration
- ORAL USE
- Max daily dose
- 10 mg/kg milligram(s)/kilogram
- Max total dose
- 10 mg/kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- J04AB02 — RIFAMPICIN
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
DEXAMBUTOL 500 mg, comprimé pelliculé
PRD2938117 · Product
- Active substance
- Ethambutol Hydrochloride
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 15 mg/kg milligram(s)/kilogram
- Max total dose
- 20 mg/kg milligram(s)/kilogram
- Max treatment duration
- 6 Month(s)
- Authorisation status
- Authorised
- ATC code
- J — ANTIINFECTIVES FOR SYSTEMIC USE
- Marketing authorisation
- 34009 311 621-7 9
- MA holder
- SOCIETE D'ETUDES ET DE RECHERCHES PHARMACEUTIQUES (SERP)
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Universitaire Amiens Picardie
- Sponsor organisation
- Centre Hospitalier Universitaire Amiens Picardie
- Address
- 1 Rond Point Du Pr Christian Cabrol
- City
- Amiens Cedex 1
- Postcode
- 80054
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Universitaire Amiens Picardie
- Contact name
- ROUSSEAUX Justine
Public contact point
- Organisation
- Centre Hospitalier Universitaire Amiens Picardie
- Contact name
- ROUSSEAUX Justine
Locations
1 EU/EEA country · 34 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 190 | 34 |
| Rest of world | — | 0 | — |
Investigational sites
Centre Hospitalier Universitaire Amiens Picardie
Pneumology, 1 Rond Point Du Pr Christian Cabrol, 80054, Amiens Cedex 1
Centre Hospitalier D'Abbeville
Pneumology, 43 Rue De L Isle, 80100, Abbeville
Centre Hospitalier Intercommunal Creteil
Pneumology, 40 Avenue De Verdun, 94000, Creteil
Centre Hospitalier Regional Et Universitaire De Brest
Pneumology, Boulevard Tanguy Prigent, 29200, Brest
Centre Hospitalier Regional Universitaire De Tours
Respiratory disease, 2 Boulevard Tonnelle, 37000, Tours
Centre Hospitalier De Saint-Quentin
Pneumology, 1 Rue Michel De L Hospital, 02100, Saint Quentin
CHU De Rouen
Pneumology, 1 Rue De Germont, Bp 96031, Rouen Cedex
Centre Hospitalier De Cannes Simone Veil
Pneumology, 15 Avenue Des Broussailles, Cs 50008, Cannes Cedex
Assistance Publique Hopitaux De Paris
Infectious and tropical diseases, 149 Rue De Sevres, 75015, Paris
Hopital Saint Joseph
Pneumology, 26 Boulevard De Louvain, 13008, Marseille
Centre Hospitalier Regional D'orleans
Pneumology, 14 Avenue De L Hopital, Cs 86709, Orleans Cedex 2
Hopital Tenon
Pneumology, 4 Rue De La Chine, 75970, Paris Cedex 20
Les Hopitaux Universitaires De Strasbourg
Pneumology, 1 Place De L Hopital, Cs 80426, Strasbourg Cedex
Centre Hospitalier Universitaire D Angers
Pneumology, 4 Rue Larrey, 49100, Angers
Centre Hospitalier Universitaire Reims
Respiratory disease, 45 Rue Cognacq Jay, 51100, Reims
Hopital NOVO
Pneumology, 6 Avenue De L Ile De France, 95300, Pontoise
Centre Hospitalier Universitaire Grenoble Alpes
Pneumology, Boulevard De La Chantourne, Cs 10217, Grenoble Cedex 9
Centre Hospitalier Universitaire De Poitiers
Pneumology, 2 Rue De La Miletrie, 86000, Poitiers
Centre Hospitalier Universitaire De Rennes
Pneumology, 2 Rue Henri Le Guilloux, 35000, Rennes
Centre Hospitalier Universitaire De Dijon
Pneumology, 2 Boulevard Mal De Lattre De Tassigny, 21000, Dijon
Hopital Avicenne
Pneumology, 125 Rue De Stalingrad, 93009, Bobigny Cedex
Hospital La Croix Rousse Hcl
Pneumology, 103 Grande Rue De La Croix Rousse, 69317, Lyon Cedex 04
Centre Hospitalier Intercommunal Compiegne Noyon
Pneumology, 8 Avenue Henri Adnot, 60200, Compiegne
Assistance Publique Hopitaux De Marseille
Pneumology, 265 Chemin Des Bourrely, 13015, Marseille
Centre Hospitalier Le Mans
Pneumology and respiratory disease, 194 Avenue Rubillard, 72037, Le Mans Cedex 9
Centre Hospitalier Universitaire De Bordeaux
Respiratory disease, Avenue Du Haut Leveque, 33600, Pessac
Centre Hospitalier De Perpignan
Pneumology, 20 Avenue Du Languedoc, Cs 49954, Perpignan Cedex
Groupe Hospitalier De La Region De Mulhouse Et Sud Alsace
Pneumology, 20 Avenue Du Docteur Rene Laennec, 68100, Mulhouse
Centre Hospitalier Universitaire De Toulouse
Pneumology, 24 Chemin De Pouvourville, 31400, Toulouse
Groupe Hospitalier Du Havre
Pneumology, 55 B Rue Gustave Flaubert, 76600, Le Havre
Centre Hospitalier Universitaire De Nantes
Pneumology, 5 Allee De L Ile Gloriette, Cs 69301, Nantes Cedex 1
Assistance Publique Hopitaux De Paris
Respiratory medicine, 27 Rue Du Faubourg Saint Jacques, 75014, Paris
Centre Hospitalier Universitaire De Caen Normandie
Pneumology, Avenue De La Cote De Nacre, 14000, Caen
Centre Hospitalier Annecy Genevois
Pneumology, 1 Avenue De L Hopital, Bp 90074, Epagny Metz Tessy
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 | 2025-10-29 | 2025-10-29 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 14 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol - Extract (for publication) | D1_Protocol_AKAPI_2023-506843-40-00_V3_TC | 3 |
| Protocol (for publication) | D1_Protocol 2023-506843-40-00 | 2.2 |
| Protocol (for publication) | D1_Protocol_AKAPI_2023-506843-40-00_ V3_Clean | 3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Subject information and informed consent form - Extract (for publication) | L1_SIS and ICF Adults_AKAPI_2023-506843-40-00_V2_TC | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adults | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adults_2023-506843-40-00_AKAPI_V2_Clean | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Arikayce | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Clarithromycine | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Ethanbutol | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Rifampicine | 1 |
| Synopsis of the Protocol - Extract (for publication) | D1_Protocol synopsis_AKAPI_2023-506843-40-00 FRANCAIS_V3_TC | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2023-506843-40-00 FRANCAIS | 2 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_AKAPI_2023-506843-40-00 FRANCAIS_V3_Clean | 3 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-04-19 | France | Acceptable 2024-07-15
|
2024-07-15 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-09-15 | France | Acceptable 2025-10-24
|
2025-10-24 |