Cystic FibrosisPhase 1NCT02598999
Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients
A Phase 1 study testing ALX-009, OSCN- and bLF in people with Cystic Fibrosis and Bronchiectasis, run by Alaxia SAS at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Bronchiectasis
- What is being tested?
- ALX-009, OSCN- and bLF
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 92 participants
- Where
- 1 siteFrance
- Study dates
- Nov 2015to Dec 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized, Double Blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics After Single Ascending Doses or Multiple Ascending Doses of OSCN-, bLF or ALX-009 in Healthy Male and CF and Non-CF Bronchiectasis Patients
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).
Read the detailed description
Part I: SAD of OSCN- and bLF in healthy male volunteers (cohorts 1 to 3) - Part II: SAD and MAD of ALX-009 in healthy male volunteers (cohorts 4 and 5) - Part III: MAD of OSCN- and bLF in patients suffering from cystic fibrosis (cohort III-1) and in healthy volunteers (cohorts III-2 and III-3) - Part IV: MAD of ALX-009 in healthy volunteers (Part IVa - Cohorts IV-1a to IV-3a) and in patients (Part IVb - Cohorts IV-1b to IV-3b)
- Conditions
- Cystic Fibrosis, Bronchiectasis
- Being tested
- ALX-009, OSCN-, bLF, Placebo
- Medical subject headings
- Cystic Fibrosis, Bronchiectasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALX-009Drug
Also called Association of OSCN- and bLF
Solution for inhalation administered through nebulization
OSCN-Drug
Also called Hypothiocyanite
Solution for inhalation administered through nebulization
bLFDrug
Also called bovine Lactoferrin
Solution for inhalation administered through nebulization
PlaceboDrug
Solution for inhalation administered through nebulization, Sodium Chloride 0.9%
| Study group | Role | What they receive |
|---|---|---|
| Part I, SAD | Experimental | Single administration of OSCN- or bLF or Placebo in healthy male volunteers Receives: Drug: OSCN-; Drug: bLF; Drug: Placebo |
| Part II, SAD and MAD | Experimental | Single and multiple administrations of ALX-009 or Placebo in healthy male volunteers Receives: Drug: ALX-009; Drug: Placebo |
| Part III, MAD | Experimental | Multiple administrations of OSCN- or bLF or Placebo in CF patients in healthy volunteers Receives: Drug: OSCN-; Drug: bLF; Drug: Placebo |
| Part IV, MAD | Experimental | Multiple administrations of ALX-009 or Placebo in healthy volunteers and in patients (CF and NCFBE) Receives: Drug: ALX-009; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability: number of subjects who experience serious adverse events, adverse events, potential clinically significant changes in ECG, 24-holter, vital signs, physical examinations, laboratory tests, spirometry, O2 saturation (Part III only)
Measured: Day (D) 8 post dosing for part I and D14 post dosing for parts II, III and IV
Secondary outcomes (14)
Maximal concentration (Cmax) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Area under the curve (AUC) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
First time to reach Cmax (Tmax) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration half life of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of anti-bLF antibodies in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-1β in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-6 in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-8 in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-10 in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of TNF-α in blood and sputum
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of SC5b-9 in blood
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of total IgE in blood
Measured: D8 post dosing for part I and D14 post dosing for parts II, III and IV
For patients only, quantitative assessment of different species in sputum
Staphylococcus aureus, Staphylococcus aureus MRSA, Pseudomonas aeruginosa, Pseudomonas aeruginosa MDR, Haemophilus influenzae, Stenotrophomonas maltophilia, Achromobacter xylosoxidans, Burkholderia cepacia complex, Aspergillus fumigatus and Aspergillus terreus
Measured: D7 post dosing
For patients only, volume of sputum over 24hours period
Measured: D8 post dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2015 (estimated)
- Study startNov 2015
- Primary completionDec 2021
- Study completionDec 2021
Why it stopped: Financial issues
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alaxia SAS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Isabelle Durieu, Prof., MD, Principal investigator, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02598999
- Sponsor's protocol ID
- ALX-009-CL-038
- Other IDs
- 2014-002401-38, 2014
- Status last verified
- Jan 2022
- First submitted
- 4 Nov 2015
- Last updated
- 3 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double Blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics After Single Ascending Doses or Multiple Ascending Doses of OSCN-, bLF or ALX-009 in Healthy Male and CF and Non-CF Bronchiectasis Patients
- ClinicalTrials.gov ID
- NCT02598999
- Acronym / sponsor ID
- ALX-009-CL-038
- Conditions
- Cystic Fibrosis, Bronchiectasis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Financial issues
- Enrolment
- 92 participants (actual)
- Lead sponsor
- Alaxia SASIndustry (pharma, biotech, devices)
- Study start
- Nov 2015 (actual)
- Primary completion
- Dec 2021 (actual)
- Study completion
- Dec 2021 (actual)
- First posted
- 6 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Part I: SAD of OSCN- and bLF in healthy male volunteers (cohorts 1 to 3) - Part II: SAD and MAD of ALX-009 in healthy male volunteers (cohorts 4 and 5) - Part III: MAD of OSCN- and bLF in patients suffering from cystic fibrosis (cohort III-1) and in healthy volunteers (cohorts III-2 and III-3) - Part IV: MAD of ALX-009 in healthy volunteers (Part IVa - Cohorts IV-1a to IV-3a) and in patients (Part IVb - Cohorts IV-1b to IV-3b)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part I, SAD | Experimental | Drug: OSCN-, Drug: bLF, Drug: PlaceboSingle administration of OSCN- or bLF or Placebo in healthy male volunteers |
| Part II, SAD and MAD | Experimental | Drug: ALX-009, Drug: PlaceboSingle and multiple administrations of ALX-009 or Placebo in healthy male volunteers |
| Part III, MAD | Experimental | Drug: OSCN-, Drug: bLF, Drug: PlaceboMultiple administrations of OSCN- or bLF or Placebo in CF patients in healthy volunteers |
| Part IV, MAD | Experimental | Drug: ALX-009, Drug: PlaceboMultiple administrations of ALX-009 or Placebo in healthy volunteers and in patients (CF and NCFBE) |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALX-009Also: Association of OSCN- and bLFSolution for inhalation administered through nebulization | Drug | Part II, SAD and MAD, Part IV, MAD |
| OSCN-Also: HypothiocyaniteSolution for inhalation administered through nebulization | Drug | Part I, SAD, Part III, MAD |
| bLFAlso: bovine LactoferrinSolution for inhalation administered through nebulization | Drug | Part I, SAD, Part III, MAD |
| PlaceboSolution for inhalation administered through nebulization, Sodium Chloride 0.9% | Drug | Part I, SAD, Part II, SAD and MAD, Part III, MAD, Part IV, MAD |
Primary outcome
Safety and tolerability: number of subjects who experience serious adverse events, adverse events, potential clinically significant changes in ECG, 24-holter, vital signs, physical examinations, laboratory tests, spirometry, O2 saturation (Part III only)
Time frame: Day (D) 8 post dosing for part I and D14 post dosing for parts II, III and IV
14 secondary outcomes
Maximal concentration (Cmax) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Area under the curve (AUC) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
First time to reach Cmax (Tmax) of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration half life of bLF and SCN- in plasma, sputum and urine (for SCN- only)
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of anti-bLF antibodies in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-1β in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-6 in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-8 in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of IL-10 in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of TNF-α in blood and sputum
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of SC5b-9 in blood
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
Concentration of total IgE in blood
Time frame: D8 post dosing for part I and D14 post dosing for parts II, III and IV
For patients only, quantitative assessment of different species in sputum
Time frame: D7 post dosing
For patients only, volume of sputum over 24hours period
Time frame: D8 post dosing
Results and safety
The study was stopped early (reason given: Financial issues). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Isabelle Durieu, Prof., MD principal investigator
Hospices Civils de Lyon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALX-009, OSCN- and bLF in people with Cystic Fibrosis and Bronchiectasis, run by Alaxia SAS at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Financial issues). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability: number of subjects who experience serious adverse events, adverse events, potential clinically significant changes in ECG, 24-holter, vital signs, physical examinations, laboratory tests, spirometry, O2 saturation (Part III only) (time frame: Day (D) 8 post dosing for part I and D14 post dosing for parts II, III and IV). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2022; AzTrials last refreshed its copy on 8 Nov 2026.