ICoN-1 Phase 3 Study of the Efficacy and Safety of Treatment With MNKD-101, Clofazimine Inhalation Suspension

NCT ID: NCT06418711

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

132 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-11

Study Completion Date

2025-11-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This clinical trial is designed to compare the efficacy and safety of Clofazimine Inhalation Suspension versus placebo when added to guideline-based therapy (GBT)

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Randomized, double-blind, placebo-controlled study (Part A) designed to compare the efficacy and safety of Clofazimine Inhalation Suspension versus placebo when added to guideline-based therapy (GBT). The primary objective of this study will be to compare the efficacy of Clofazimine Inhalation Suspension versus placebo as assessed by the co-primary endpoints, sputum culture conversion and change in Quality of Life-Bronchiectasis Respiratory Symptoms Score (QoL-B RSS).

An open label extension study (Part B) will be offered to qualified participants for treatment with Clofazimine Inhalation Suspension.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

MAC Lung Disease Treatment Refractory MAC Lung Disease Mycobacterium Infections, Nontuberculous

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Bronchiectasis NTM (Nontuberculous mycobacteria) MAC (Mycobacterium avium complex) MABSC (Mycobacterium abscessus complex) Pulmonary Nontuberculous Mycobacteria Respiratory Infection antimycobacterial activity antimycobacterial therapy MAC infections MAC Lung disease MAC pulmonary infection Mycobacteria mycobacterium Mycobacterium Avium Complex Infections Mycobacterium avium complex lung disease mycobacterium Infections Nontuberculous Non-tuberculous mycobacterial (NTM) infections Nontuberculous mycobacterial lung disease Non-tuberculous mycobacterial pulmonary disease NTM infection NTM lung disease NTM Pulmonary Disease NTM lung infection Pulmonary MAC disease Pulmonary Mycobacterium Avium Complex disease Treatment refractory MAC lung disease Treatment refractory mycobacterial lung disease Treatment refractory NTM lung infection Treatment refractory NTM pulmonary disease Respiratory Tract diseases Actinomycetales Infections Gram-Positive Bacterial Infections Bacterial Infections Bacterial Infections and Mycoses Infections Mycobacterium Infections, Nontuberculous Mycobacterium avium-intracellulare Infection Lung diseases Anti-Bacterial Agents Anti-infective Agents Antitubercular Agents Azithromycin Amikacin Ethambutol Clofazimine Lamprene

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

At least 234 eligible participants will be randomized in a 2:1 ratio of 1 of 2 possible treatment assignments in order to ensure that a minimum of 180 participants are evaluable for efficacy: Clofazimine Inhalation Suspension (N=120) and placebo (n=60). Randomization will be stratified by type of nontuberculous mycobacteria (NTM) infection (Mycobacterium avium complex \[MAC\] infection only vs MAC co-infection with Mycobacterium abscessus complex \[MABSC\] or other NTM species) based on historical samples used to establish eligibility during screening. Enrollment of participants with MAC coinfection with MABSC or other NTM species will be capped at 20% of all participants to limit heterogeneity in the participant population.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
During Part A of the study, the identity of the treatments will be concealed by the use of a placebo, and treatment unblinding will only occur in the case of participant emergencies or if requested by the Data and Safety Monitoring Board (DSMB).

Sputum Test Results: Results of post-baseline testing for the presence of NTM in sputum will remain concealed until the participant has completed Part A of the study and the participant's result for the sample taken at the end of Month 6 becomes available.

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Clofazimine Inhalation Suspension

MNKD-101 (Clofazimine Inhalation Suspension) is a micronized suspension with a concentration of 20 mg/mL. Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days. Dose: 80 mg

Group Type EXPERIMENTAL

Clofazimine Inhalation Suspension

Intervention Type DRUG

Eligible participants will be randomized in a 2:1 ratio to 1 of 2 possible treatment assignments, Clofazimine Inhalation Suspension or Placebo.

Placebo

The placebo is comprised of isotonic saline (0.9% weight/volume sodium chloride). Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Eligible participants will be randomized in a 2:1 ratio to 1 of 2 possible treatment assignments, Clofazimine Inhalation Suspension or Placebo.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Clofazimine Inhalation Suspension

Eligible participants will be randomized in a 2:1 ratio to 1 of 2 possible treatment assignments, Clofazimine Inhalation Suspension or Placebo.

Intervention Type DRUG

Placebo

Eligible participants will be randomized in a 2:1 ratio to 1 of 2 possible treatment assignments, Clofazimine Inhalation Suspension or Placebo.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

MNKD-101

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Evidence of signed and dated informed consent document(s) indicating the participant has been informed of all pertinent aspects of the trial.
2. Age ≥18 years or legal age for the participating country (e.g., the legal age in South Korea is 19 years) and ≤85 years.
3. Evidence of underlying nodular bronchiectasis and/or fibrocavitary disease on a chest radiograph or chest computed tomography, as determined by the investigator, within the last 12 months.
4. MAC-positive culture results from at least two separates (at least 1 week apart) expectorated sputum samples, one taken within 12 months, and another taken within 3 months prior to the date of informed consent. Note: A sputum culture will be obtained at baseline, but the participant may be randomized prior to availability of the results.
5. Be able to produce at least 3 mL of sputum or be willing to undergo an induction that produces at least 3 mL of sputum for mycobacteriology.
6. FEV1 ≥40% of predicted during screening, as calculated by the local spirometry laboratory standards.
7. Currently receiving a multi-drug regimen of GBT for pulmonary NTM infection in line with the 2020 ATS/ERS/ESCMID/IDSA guideline for the treatment of NTM pulmonary disease for at least 6 months prior to consenting in this study, with no changes in this regimen within 2 months of screening.
8. For female participants of childbearing potential, a negative serum pregnancy test and agreement to use a protocol-recommended method of contraception during heterosexual intercourse from the start of the screening period until ≥12 months after the final dose of study therapy. Note: A female participant is considered to be of childbearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle-stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
9. For male participants who can father a child and are having intercourse with females of childbearing potential, agreement to use a protocol-recommended method of contraception from the start of the study therapy until ≥12 months after the final dose of study therapy and to refrain from sperm donation from the start of study therapy until ≥12 months after administration of the final dose of study therapy. Note: A male participant is considered fertile after puberty unless permanently sterile by bilateral orchidectomy.
10. Willingness and ability to comply with scheduled visits, drug inhalation plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria

1. Cystic fibrosis.
2. Active tuberculosis. Note: Participants with a history of treated latent or active tuberculosis may be eligible as long as their sputum cultures in the last year are negative for tuberculosis and they are deemed by the investigator as not having current active tuberculosis.
3. Disseminated MAC or MABSC infection or participants with isolated MABSC infection.
4. Recent (i.e., within the last 3 months from date of screening) ICU admission with or without mechanical ventilation.
5. Inability to inhale with a nebulizer, in the opinion of the investigator.
6. Participants with known hypersensitivity to any of the ingredients or excipients of clofazimine.
7. Prior therapy with clofazimine in the previous 4 months from date of screening.
8. Participants with known resistance to clofazimine as treatment for MAC (i.e., MIC \>8 ug/mL for MAC).
9. Prior therapy with amikacin by any route of administration (e.g., inhaled or IV) in the previous 2 months from date of screening.
10. Ongoing participation in any other interventional drug or device clinical trial, or exposure to another investigational drug within 28 days prior to start of study treatment. Note: For investigational therapies that have a prolonged half-life, a case-by-case assessment will be made regarding the required washout period prior to being eligible for this study.
11. Current (or planned during the study) pregnancy or breastfeeding.
12. QT prolongation during screening (450 ms or longer), and/or uncontrolled sinus rhythm (\>110/minute).
13. Increased risk of proarrhythmia (e.g., recent \[within 6 months\] myocardial infarction, stroke, heart failure decompensation or left ventricular ejection of \<45%, ventricular arrhythmias, torsade de pointes, unstable angina, or high-degree atrioventricular block).
14. A family history of sudden cardiac death, unexplained death, long-QT syndrome, or death from a primary dysrhythmia potentially associated with QT prolongation.
15. Recent (within 6 months) initiation of or change in the dosing regimen of any concomitant medication that is known to prolong the QT interval. Note: Participants who are on a stable regimen, in the opinion of the investigator, of the concomitant medication during screening are eligible.
16. Chronic and clinically meaningful, in the opinion of the investigator, abnormalities in potassium, magnesium, or calcium levels.
17. Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within 3 years before screening or anticipated during the study period.
18. Current alcohol, medication, or illicit drug abuse.
19. Prior or ongoing social or medical condition (e.g., concomitant illness, psychiatric condition, behavioral disorder), medical history, physical findings, ECG findings or laboratory abnormality that, in the opinion of the investigator, could adversely affect the safety of the participant, makes it unlikely that the course treatment or follow-up would be completed, or could impair the assessment of study results.
20. Any prior use of bedaquiline within 1 year of screening.
21. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>1.5 times upper limit of normal (ULN) or total bilirubin \>1.5 times ULN during screening.
22. Absolute neutrophil count \<500/µL during screening.
23. Use of prednisone ≥10mg/day within 3 months prior to screening, or other significant immunosuppression as deemed by the investigator.
24. Estimated glomerular filtration rate \<30mL/minute/1.73 m2 (according to the CKD-EPI 2021 creatine equation) during screening.
25. Advanced liver disease (Child-Pugh Class A, B, or C).
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Mannkind Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Wassim Fares, MD

Role: STUDY_DIRECTOR

Mannkind Corporation

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of Alabama at Birmingham School of Medicine

Birmingham, Alabama, United States

Site Status

University of California San Francisco Fresno

Fresno, California, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

Hoag Hospital

Newport Beach, California, United States

Site Status

University of California Davis Medical Center

Sacramento, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Santa Barbara Cottage Hospital

Santa Barbara, California, United States

Site Status

Stanford University

Stanford, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

UCONN Health

Farmington, Connecticut, United States

Site Status

Yale University

New Haven, Connecticut, United States

Site Status

Georgetown University Hospital

Washington D.C., District of Columbia, United States

Site Status

The George Washington University Medical Faculty Associates

Washington D.C., District of Columbia, United States

Site Status

VA Bay Pines

Bay Pines, Florida, United States

Site Status

St. Francis Sleep, Allergy & Lung Institute

Clearwater, Florida, United States

Site Status

Malcolm Randall Veterans Affairs Medical Center

Gainesville, Florida, United States

Site Status

University of Florida College of Medicine

Jacksonville, Florida, United States

Site Status

Theia Clinical Research

St. Petersburg, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Midway Specialty Care Center

West Palm Beach, Florida, United States

Site Status

Cleveland Clinic

Weston, Florida, United States

Site Status

Flourish Research

Winter Park, Florida, United States

Site Status

Emory University School Of Medicine

Atlanta, Georgia, United States

Site Status

Medster Research

Valdosta, Georgia, United States

Site Status

University of Hawaii

Honolulu, Hawaii, United States

Site Status

University of Iowa Hospital and Clinics

Iowa City, Iowa, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Infectious Disease Consultants Clinical Research

Wichita, Kansas, United States

Site Status

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Missouri

Columbia, Missouri, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Rutgers New Jersey Medical School

Newark, New Jersey, United States

Site Status

Northwell Health

New Hyde Park, New York, United States

Site Status

NYU Langone Health

New York, New York, United States

Site Status

Mount Sinai-National Jewish Respiratory Institute

New York, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

NYC Health and Hospitals-Elmhurst

Queens, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Temple Lung Center

Philadelphia, Pennsylvania, United States

Site Status

Reading Hospital

West Reading, Pennsylvania, United States

Site Status

AnMed Health

Anderson, South Carolina, United States

Site Status

Low Country Infectious Diseases

Charleston, South Carolina, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

North Texas Infectious Diseases Consultants

Dallas, Texas, United States

Site Status

The University of Texas Health Science Center

Tyler, Texas, United States

Site Status

UVA Health Infectious Diseases Clinic

Charlottesville, Virginia, United States

Site Status

Macquarie University Clinical Trials Unit

Sydney, New South Wales, Australia

Site Status

Sunshine Coast University Hospital

Birtinya, Queensland, Australia

Site Status

The Prince Charles Hospital

Brisbane, Queensland, Australia

Site Status

Gallipoli Medical Research Foundation

Greenslopes, Queensland, Australia

Site Status

Monash University Centre for Inflammatory Diseases

Cranbourne, Victoria, Australia

Site Status

Alfred Health

Melbourne, Victoria, Australia

Site Status

Royal Perth Hospital Respiratory Clinic

Perth, Western Australia, Australia

Site Status

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

Concord Repatriation General Hospital

Concord, , Australia

Site Status

Aso Iizuka Hospital

Fukuoka, , Japan

Site Status

National Hospital Organization Fukuokahigashi Medical Center

Fukuoka, , Japan

Site Status

Fukuoka University Hospital

Fukuoka, , Japan

Site Status

Fukuoka University Chikushi Hospital

Fukuoka, , Japan

Site Status

National Hospital Organization Omuta National Hospital

Fukuoka, , Japan

Site Status

National Hospital Organization Shibukawa Medical Center

Gunma, , Japan

Site Status

Himeji Medical Center

Hyōgo, , Japan

Site Status

Ibarakihigashi National Hospital

Ibaraki, , Japan

Site Status

Kameda Clinic

Kamogawa-shi, , Japan

Site Status

SHOWA University Fujigaoka Hospital

Kanagawa, , Japan

Site Status

Minami Kyoto Hospital

Kyoto, , Japan

Site Status

Matsusaka Municipal Hospital

Matsusaka, , Japan

Site Status

Sendai Kousei Hospital

Miyagi, , Japan

Site Status

Nishiniigata Chuo Hospital

Niigata, , Japan

Site Status

National Hospital Organization - Osaka Toneyama Medical Center

Osaka, , Japan

Site Status

Saitama Prefectural Cardiovascular and Respiratory Center

Saitama, , Japan

Site Status

National Hospital Organization Kinki-Chuo Chest Medical Center

Sakai, , Japan

Site Status

Keio University Hospital

Shinjuku-ku, , Japan

Site Status

National Hospital Organization Tenryu Hospital

Shizuoka, , Japan

Site Status

Mutual Aid Associations Toranomon Hospital

Tokyo, , Japan

Site Status

Toho University Omori Medical Center

Tokyo, , Japan

Site Status

Center Hospital of the National Center for Global Health and Medicine

Tokyo, , Japan

Site Status

Japanese Red Cross Musashino Hospital

Tokyo, , Japan

Site Status

Japan Anti-Tuberculosis Association Fukujuji Hospital

Tokyo, , Japan

Site Status

SHOWA University Northern Yokohama Hospital

Yokohama, , Japan

Site Status

Chonnam National University Hospital

Gwangju, , South Korea

Site Status

Seoul National University Hospital

Seoul, , South Korea

Site Status

Severance Hospital

Seoul, , South Korea

Site Status

Asan Medical Center

Seoul, , South Korea

Site Status

Samsung Medical Center

Seoul, , South Korea

Site Status

The Catholic University of Korea Seoul St.Mary's Hospital

Seoul, , South Korea

Site Status

National Taiwan University Hospital

Hsinchu, , Taiwan

Site Status

Kaohsiung Medical University Hospital

Kaohsiung City, , Taiwan

Site Status

Taichung Veterans General Hospital

Taichung, , Taiwan

Site Status

Taipei Veterans General Hospital

Taipei, , Taiwan

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Australia Japan South Korea Taiwan

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

MKC-CI-002

Identifier Type: -

Identifier Source: org_study_id