Amikacin Liposome Inhalation Suspension for Treatment of Mycobacterium Xenopi Pulmonary Infection (AKAPI)

November 17, 2025 updated by: Centre Hospitalier Universitaire, Amiens
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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
190

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • France
      • Amiens, France, 80054
        • Recruiting
        • CHU Amiens-Picardie
        • Principal Investigator:
          • Léa COLOMBAIN, Dr
        • Principal Investigator:
          • Gilles DEVOUASSOUX, Pr
        • Principal Investigator:
          • Hugues MOREL, Dr
        • Contact:
          • Claire Andréjak, MD
        • Principal Investigator:
          • Frédéric GAGNADOUX, Pr
        • Principal Investigator:
          • Diane BOUVRY, Dr
        • Principal Investigator:
          • Elodie BLANCHARD, DR
        • Principal Investigator:
          • Cécile L'HEVEDER, Pr
        • Principal Investigator:
          • Antoine BELLE, Dr
        • Principal Investigator:
          • Bernard MAITRE, Pr
        • Principal Investigator:
          • Philippe BONNIAUD, Dr
        • Principal Investigator:
          • Rebecca HAMIDFAR, Dr
        • Principal Investigator:
          • Marie JOUVENOT, Dr
        • Principal Investigator:
          • Martine REYNAUD GAUBERT, Pr
        • Principal Investigator:
          • Cristina SOCOLOVSCHI AUDOLY, Dr
        • Principal Investigator:
          • Didier DEBIEUVRE, Dr
        • Principal Investigator:
          • Thomas MAITRE, Dr
        • Principal Investigator:
          • Alexandra SERRIS, Pr
        • Principal Investigator:
          • Lucien GUERIN, Dr
        • Principal Investigator:
          • Jean Francois BOITIAUX, Dr
        • Principal Investigator:
          • Gaëtan DESLEE, Pr
        • Principal Investigator:
          • Mallorie KERJOUAN, Dr
        • Principal Investigator:
          • Hélène MORISSE PRADIER, Dr
        • Principal Investigator:
          • Youcef DOUADI, Dr
        • Principal Investigator:
          • Loic KASSEGNE, Dr
        • Principal Investigator:
          • Marlène MURRIS-ESPIN, Dr
        • Principal Investigator:
          • Sylvain MARCHAND ADAM, Pr
        • Principal Investigator:
          • Estelle HOGUET, Dr
        • Principal Investigator:
          • Florence LE MEUNIER, Dr

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 18 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

Exclusion Criteria:

  • 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:

Allergy to macrolides or to any of the excipients listed in section 6.1;

  • Association with :

    • colchicine,
    • ergot alkaloids, including for example dihydroergotamine, ergotamine, methylergometrine, methysergide: risk of ergotism,
    • pimozide, mizolastine: risk of QT interval prolongation and cardiac rhythm disorders, in particular ventricular tachycardia, ventricular fibrillation and torsades de pointes,
    • simvastatin, due to the increased risk of myopathy, including rhabdomyolysis.
    • lomitapide,
    • alfuzosin
    • dapoxetine
    • avanafil
    • ivabradine,
    • eplerenone,
    • dronedarone,
    • Quetiapine,
    • ticagrelor,
    • cisapride,
    • astemizole,
    • terfenadine,
    • ranolazine,
    • domperidone,
  • Congenital or acquired prolongation of the QT interval (see sections 4.4 and 4.5 of the CPR)
  • History of QT interval prolongation or ventricular rhythm disorders, in particular torsades de pointe (see sections 4.4 and 4.5 of the CPR);
  • Electrolyte imbalances (hypokalaemia or hypomagnesaemia, due to the risk of QT interval prolongation) (see sections 4.4 and 4.5 of the CPR).Clarithromycin should not be used in patients with severe hepatic insufficiency in association with renal insufficiency.

Contraindication to the use of Rifampicine:

  • Hypersensitivity to rifamycins or to any of the excipients listed in section 6.1 of the CPR.
  • Porphyrias.
  • Association with bictegravir, cobicistat, daclatasvir, dasabuvir, delamanid, grazoprevir/elbasvir, ritonavir-boosted protease inhibitors, isavuconazole, lédipasvir, lurasidone, midostaurine, ombitasvir/paritaprévir, praziquantel, rilpivirine, sofosbuvir, velpatasvir, voriconazole, voxilaprévir, (see section 4. 5 of the CPR). In children under 6 years of age, due to the risk of malaria

Contradiction the the use of Ethambutol:

  • Known hypersensitivity to ethambutol
  • Optic neuritis
  • This medicine is contraindicated in patients with a wheat allergy (other than coeliac disease).
  • Inability to comply with the requirements of the protocol, especially substance abuse, according to the investigator.
  • Limited life expectancy (e.g 3 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%.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ARIKAYCE
Experimental arm = ARIKAYCE® during 6 months in addition to standard treatment (duration determined with date of sputum conversion)
Drug: Arikayce
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.
Active Comparator: Standard treatment
Control arm = standard treatment Standard treatment = Rifampicin, ethambutol and clarithromycin
Drug: standard treatment
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
variation of sputum conversion rate in ARIKAYCE® addition group compared to standard treatment
Time Frame: 3 months
3 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
variation of time to culture conversion between both groups
Time Frame: at 3 month
at 3 month
variation of mortality between both groups
Time Frame: at 12 months
at 12 months
variation of mortality between both groups
Time Frame: at 24 months
at 24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Centre Hospitalier Universitaire, Amiens

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
University Hospital, Angers
Centre Hospitalier Universitaire Dijon
CHU de Reims
Central Hospital Saint Quentin
University Hospital, Bordeaux
Poitiers University Hospital
University Hospital, Brest
University Hospital, Grenoble
Rennes University Hospital
University Hospital, Tours
IHU Strasbourg
Hôpital de la Croix-Rousse
Tenon Hospital, Paris
Hôpital Necker-Enfants Malades
Centre Hospitalier le Mans
Hospital Avicenne
Créteil Hospital
Centre hospitalier de Perpignan
CHU de Rouen - Accueil
CH COMPIEGNE
CH Abbeville
APHM - Nord
Hôpital Saint Joseph
CH Mulhouse
CH Orléans
CH Pontoise
CH Cannes

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 20, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 13, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 4, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 5, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 19, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 17, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • PI2021_843_0148

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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