Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients

• Status: Recruiting
• Trial end date: 2021-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of a single ascending doses (SAD) and multiple ascending doses (MAD) of Hypothiocyanite (OSCN-), bovine lactoferrin (bLF) and their combination (ALX-009) in healthy male volunteers and patients suffering from cystic fibrosis (CF) and non-CF bronchiectasis (NCFBE).

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Alaxia SAS
• Treatments: Hypothiocyanite ion; Lactoferrin

Criteria

Inclusion Criteria:

• Healthy male subject or

• Patient suffering from cystic fibrosis defined as a positive sweat chloride test or CF-causing mutations, documented in the patient's medical record or patient suffering from non-CF and non COPD bronchiectasis with a diagnosis confirmed by a chest CT scan demonstrating bronchiectasis in 1 or more lobes documented in the patient's medical record

• Aged between 18 and 50 years inclusive

• Subject's Body Mass Index between 18 and 30 kg/m²

• Subject with normal blood pressure, heart rate, ECG recording and laboratory parameters at the screening visit

• Subject having given a written informed consent prior to selection

• Subject covered by Health Insurance System and/ or in compliance with the recommendations of National Law in force relating to biomedical research

Specific Inclusion Criteria for patients:

• FEV1 more than or equal to 60% of predicted normal value

• Subject in a stable state (no exacerbation for 1 month or prescription of antibiotic by intravenous route)

• Females of childbearing potential: commitment to consistently and correctly use an acceptable method of birth control for the duration of the trial and for 4 months after the last study drug administration / Female of non-childbearing potential: either surgically sterilized or at least 1 year postmenopausal

Exclusion Criteria:

• Presence of cardiovascular, pulmonary, gastro-intestinal, hepatic, renal, metabolic, haematological, neurologic, psychiatric, systemic or infectious disease

• Frequent headaches and/or migraines, recurrent nausea and/or vomiting

• Symptomatic hypotension

• Blood donation (including in the frame of a clinical trial) within 2 months before administration

• General anaesthesia within 3 months before administration

• Presence or history of drug hypersensitivity, or any allergic disease

• Medical history of reactions to cow's milk proteins

• Subject who can not be contacted in case of emergency

• History or presence of drug or alcohol abuse

• Positive Hepatitis B surface (HBs) antigen or anti Hepatitis C virus (HCV) antibody, or positive results for Human Immunodeficiency Virus (HIV) 1 or 2 tests

• Subject who, in the judgement of the Investigator, is likely to be non-compliant or uncooperative during the study, or unable to cooperate because of a language problem, poor mental development.

Specific exclusion criteria for study Parts III and IV:

• Known bronchial hyper-reactivity to drug inhalation

• Known contra-indication to inhaled salbutamol

• Subject with bronchial hyper-reactivity, defined by a positive response to bronchodilator with FEV1 increase ≥ 200 mL

Specific exclusion crtieria for patients:

• Active allergic bronchopulmonary aspergillosis currently treated

• Medical history of allergic bronchopulmonary aspergillosis in the past 2 years.