Amikacin Liposome Inhalation Suspension for Treatment of Mycobacterium Xenopi Pulmonary Infection

NCT06585020 | Recruiting Phase 2

• 1 other identifier: PI2021_843_0148
• Study Type: interventional
• Target: 190
• Locations: 1 country
• Sites: 1

Brief Summary

Treatment of Mycobacterium xenopi (MX) lung disease is not-well- tolerated and concerned a growing number of patients, especially with chronic pulmonary diseases or immunosuppression. The outcome of these patients is poor, and treatment is very long. Indeed, this duration is based on the date of sputum conversion. Treatment should be continued until 12 months after sputum conversion. In the vast majority patients have converted after 6 months of treatment, so a 18 months duration in total. Unfortunately, few data are available for MX, as it is rare in USA, but it is the second NTM isolated in France and concerns an increasing number of patients. As it is uncommon in USA, no clinical studies conducted by the pharmaceutical laboratory will be planned. In a murine model of MX infection, the only drug which decreased the number colony formant units in mice lungs, was amikacin. Until now, amikacin was only available intravenously and used only for patients with very severe disease, because of renal and auditory toxicity. Amikacin liposome inhalation suspension (ARIKAYCE®) is amikacin sulfate encapsulated in liposomes for inhalational delivery. ARIKAYCE® increases amikacin uptake into alveolar macrophages, a refuge for NTM organisms; allows biofilm penetration; and limits systemic amikacin exposure ARIKAYCE® has already be tested in a randomized study on M. avium complex (MAC) refractory pulmonary infections. In this study, the culture conversion rate in the ARIKAYCE® group was higher than standard regimen group.

Conditions

Mycobacterium; Xenopi; Lung Diseases

Keywords

Mycobacterium; Xenopi; Lung Diseases

Outcome Measures

Primary Outcomes (1)

• variation of sputum conversion rate in ARIKAYCE® addition group compared to standard treatment

  3 months

Secondary Outcomes (3)

• variation of time to culture conversion between both groups

  at 3 month

• variation of mortality between both groups

  at 12 months

• variation of mortality between both groups

  at 24 months

Study Arms (2)

ARIKAYCE

EXPERIMENTAL

Experimental arm = ARIKAYCE® during 6 months in addition to standard treatment (duration determined with date of sputum conversion)

Drug: Arikayce

Standard treatment

ACTIVE COMPARATOR

Control arm = standard treatment Standard treatment = Rifampicin, ethambutol and clarithromycin

Drug: standard treatment

Interventions

Arikayce DRUG

Treatment regimens containing three molecules, rifampicin, ethambutol, and clarithromycin with ARIKAYCE® during the 6 first months of treatment. After having confirmed the presence of all inclusion criteria and the absence of all exclusion criteria, and after having obtained the patient's free and informed consent, the patient will be included and randomized to one of the treatment regimens.

ARIKAYCE

standard treatment DRUG

treatment regimens containing three molecules, rifampicin, ethambutol, and clarithromycin during the 6 first months of treatment. After having confirmed the presence of all inclusion criteria and the absence of all exclusion criteria, and after having obtained the patient's free and informed consent, the patient will be included and randomized to one of the treatment regimens.

Standard treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years old or older
• with an highly effective or acceptable contraception
• must present ATS/IDSA 2020 criteria for nontuberculous mycobacterial pulmonary infection
• the NTM should be M. xenopi

You may not qualify if:

• Patients presenting any of the following criteria cannot be included:
• 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 :
• Contraindication to the use of ARIKAYCE®:
• Hypersensitivity to the active substance, to aminoglycosides or to any of the excipients listed in section 6.1 of the CPR.
• Hypersensitivity to soy.
• Co-administration with any other aminoglycoside, regardless of the route of administration
• Severe Renal Failure
• Contraindication to the use of Clarithromycine:

Sponsors & Collaborators

• Centre Hospitalier Universitaire, Amiens: lead
• University Hospital, Angers: collaborator
• Hospital Avicenne: collaborator
• University Hospital, Bordeaux: collaborator
• University Hospital, Brest: collaborator
• CH Compiègne: collaborator
• Créteil Hospital: collaborator
• Centre Hospitalier Universitaire Dijon: collaborator
• University Hospital, Grenoble: collaborator
• Centre Hospitalier le Mans: collaborator
• Hôpital de la Croix-Rousse: collaborator
• APHM - Nord: collaborator
• Hôpital Saint Joseph: collaborator
• CH Mulhouse: collaborator
• CH Orléans: collaborator
• Tenon Hospital, Paris: collaborator
• Hôpital Necker-Enfants Malades: collaborator
• Centre hospitalier de Perpignan: collaborator
• Poitiers University Hospital: collaborator
• CH Pontoise: collaborator
• CHU de Reims: collaborator
• Rennes University Hospital: collaborator
• CHU de Rouen - Accueil: collaborator
• Central Hospital Saint Quentin: collaborator
• IHU Strasbourg: collaborator
• University Hospital, Tours: collaborator
• CH Abbeville: collaborator
• CH Cannes: collaborator

Study Sites (1)

CHU Amiens-Picardie

Amiens, 80054, France

RECRUITING

MeSH Terms

Conditions

Mycobacterium Infections; Lung Diseases

Condition Hierarchy (Ancestors)

Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections; Respiratory Tract Diseases

Central Study Contacts

Claire ANDREJAK, Pr

CONTACT

03 22 08 79 98; Andrejak.Claire@chu-amiens.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 13, 2024
• First Posted: September 5, 2024
• Study Start: October 20, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2027
• Last Updated: November 19, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)