Cystic Fibrosis (CF)Phase 1NCT02605590
Safety, Tolerability and Pharmacokinetics Study of AIR DNAse Administered by Inhalation to Healthy Adult Volunteers
A Phase 1 study testing AIR DNase in people with Cystic Fibrosis (CF), run by Protalix at 1 site in Israel.
Trial summary
This study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 55 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis (CF)
- What is being tested?
- AIR DNase
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 1 siteIsrael
- Study dates
- Dec 2015to Feb 2016
- 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 55 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subject who understands the study procedures and provides written informed consent to participate in the study.
- Healthy, male, 18-55 years of age, inclusive
- Non smoker, by declaration, for at least 6 months prior to screening
- Body mass index (BMI) ≥ 18.5 and ≤ 29.0 kg/m2 at screening.
- Healthy individual with no clinically significant findings in: medical history, physical examination, laboratory profiles , vital signs, 12-lead ECG
- Spirometry results within normal ranges.
- A subject, whose female co-partner is of child-bearing potential, must agree to use two medically acceptable methods of contraception throughout the study.
Who cannot join
- History of asthma, any chronic pulmonary disease, recurrent pneumonia, allergic rhinitis.
- History of any illness or medical condition (including psychiatric) that might confound the results of the study or poses an additional risk to the subject by his participation in the study.
- Any acute illness (e.g. acute infection) within 48 hours prior to the study drug administration, which is considered of significance by the Investigator.
- Known contraindication, hypersensitivity and/or allergy to any drug.
- History of adverse reactions during aerosol delivery of any medicinal product.
- History or presence of alcoholism or drug abuse within the past 2 years prior to screening.
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- Positive urine drug of abuse test result prior to first dosing.
- Positive breath alcohol test on admission to the CRC prior to first dosing.
- Positive results at screening for HIV, HBsAg, or HCV Ab.
- Sitting blood pressure is less than 90/40 mmHg or greater than 140/90 mmHg at screening.
- Sitting heart rate is lower than 45 beats per minute (bpm) or higher than 99 bpm at screening.
- Use of any prescription or over-the-counter medications, including vitamins, herbal, or dietary supplements within 14 days prior to dosing. Paracetamol, up to 2g /day is allowed up to 24 hours prior to dosing.
- Donation of blood within 90 days prior to dosing.
- Donation of bone marrow within the last 6 months prior to dosing.
- Participation in another clinical trial within 90 days prior to dosing in which a study drug has been administered.
- Subject is mentally or legally incapacitated or has significant emotional problems or is unable to communicate well with the investigators and CRC staff (i.e., language problem, poor mental development or impaired cerebral function).
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Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose and Multiple Ascending Dose Study of the Safety, Tolerability, and Pharmacokinetics of AIR-DNase When Administered by Inhalation to Healthy Adult Subjects
This study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects.
A thorough review of safety data will be conducted after completion of each dose level per subject and prior to both, moving to the next dose level in Part 1 and commencement of the multiple dose Part 2.
Read the detailed description
This is a Phase I study to evaluate the safety, tolerability and pharmacokinetics of inhaled AIR DNase in healthy volunteers. Up to 18 healthy males subjects (age 18 -55 inclusive) will be randomized to one of three dose cohorts (up to 6 subjects per cohort), receiving AIR DNase doses equivalent to 1.25 mg, 2.5mg or 5 mg or placebo in accordance. Subjects randomized to one of the cohorts will receive either active of placebo as single dose, in continuation, following safety assessments, will receive multiple doses, once daily for 5 consecutive days. Subjects will remain at the clinical center for 8 hours after the single administration of AIR DNase/placebo including PK sampling. During the multiple dosing subjects will arrive at the clinical center daily for AIR DNase/placebo administrations and study procedures and remain at the site for 4 hours post dosing.
Follow up visits will be performed 7±2 days following single administration and 7±2 days following the last administration of the multiple phase.
- Conditions
- Cystic Fibrosis (CF)
- Being tested
- AIR DNase, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AIR DNaseDrug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| 1.25 mg | Experimental | Part 1: single inhaled dose of 1.25 mg AIR-DNase followed by Part 2: once daily inhaled dose of 1.25 mg AIR DNase for 5 consecutive days. Receives: Drug: AIR DNase |
| 2.5 mg | Experimental | Part 1: single inhaled dose of 2.5 mg AIR-DNase followed by Part 2: once daily inhaled dose of 2.5 mg AIR DNase for 5 consecutive days. Receives: Drug: AIR DNase |
| 5 mg | Experimental | Part 1: single inhaled dose of 5 mg AIR-DNase followed by Part 2: once daily inhaled dose of 5 mg AIR DNase for 5 consecutive days. Receives: Drug: AIR DNase |
| Placebo | Placebo comparator | Placebo comparator for each of the dose levels, administered accordingly as single inhaled dose in Part 1 followed by once daily inhaled dose for 5 consecutive days in Part 2. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events following inhaled single dose (SD) of AIR-DNase
Adverse events from subject reporting or other assessments
Measured: 10 days
Adverse events following inhaled multiple doses (MD) of AIR-DNase
Adverse events from subject reporting or other assessments
Measured: 14 days
Secondary outcomes (1)
Area under the curve
AIR DNase concentrations measured at 0, 0.25, 0.5, 0.75, 1, 1.5, 2, and 4 hours
Measured: 4 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2015 (estimated)
- Study startDec 2015
- Primary completionFeb 2016
- Study completionFeb 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis (CF)Studies 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
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Einat Almon, PhD, Study director, Protalix Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02605590
- Sponsor's protocol ID
- PB-110-CF01
- Status last verified
- Apr 2016
- First submitted
- 10 Nov 2015
- Last updated
- 17 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose and Multiple Ascending Dose Study of the Safety, Tolerability, and Pharmacokinetics of AIR-DNase When Administered by Inhalation to Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT02605590
- Acronym / sponsor ID
- PB-110-CF01
- Conditions
- Cystic Fibrosis (CF)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- Dec 2015
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 16 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 study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects.
A thorough review of safety data will be conducted after completion of each dose level per subject and prior to both, moving to the next dose level in Part 1 and commencement of the multiple dose Part 2.
Detailed description
This is a Phase I study to evaluate the safety, tolerability and pharmacokinetics of inhaled AIR DNase in healthy volunteers. Up to 18 healthy males subjects (age 18 -55 inclusive) will be randomized to one of three dose cohorts (up to 6 subjects per cohort), receiving AIR DNase doses equivalent to 1.25 mg, 2.5mg or 5 mg or placebo in accordance. Subjects randomized to one of the cohorts will receive either active of placebo as single dose, in continuation, following safety assessments, will receive multiple doses, once daily for 5 consecutive days. Subjects will remain at the clinical center for 8 hours after the single administration of AIR DNase/placebo including PK sampling. During the multiple dosing subjects will arrive at the clinical center daily for AIR DNase/placebo administrations and study procedures and remain at the site for 4 hours post dosing.
Follow up visits will be performed 7±2 days following single administration and 7±2 days following the last administration of the multiple phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1.25 mg | Experimental | Drug: AIR DNasePart 1: single inhaled dose of 1.25 mg AIR-DNase followed by Part 2: once daily inhaled dose of 1.25 mg AIR DNase for 5 consecutive days. |
| 2.5 mg | Experimental | Drug: AIR DNasePart 1: single inhaled dose of 2.5 mg AIR-DNase followed by Part 2: once daily inhaled dose of 2.5 mg AIR DNase for 5 consecutive days. |
| 5 mg | Experimental | Drug: AIR DNasePart 1: single inhaled dose of 5 mg AIR-DNase followed by Part 2: once daily inhaled dose of 5 mg AIR DNase for 5 consecutive days. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo comparator for each of the dose levels, administered accordingly as single inhaled dose in Part 1 followed by once daily inhaled dose for 5 consecutive days in Part 2. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Subject who understands the study procedures and provides written informed consent to participate in the study.
- Healthy, male, 18-55 years of age, inclusive
- Non smoker, by declaration, for at least 6 months prior to screening
- Body mass index (BMI) ≥ 18.5 and ≤ 29.0 kg/m2 at screening.
- Healthy individual with no clinically significant findings in: medical history, physical examination, laboratory profiles , vital signs, 12-lead ECG
Key exclusion criteria (17 listed)
- History of asthma, any chronic pulmonary disease, recurrent pneumonia, allergic rhinitis.
- History of any illness or medical condition (including psychiatric) that might confound the results of the study or poses an additional risk to the subject by his participation in the study.
- Any acute illness (e.g. acute infection) within 48 hours prior to the study drug administration, which is considered of significance by the Investigator.
- Known contraindication, hypersensitivity and/or allergy to any drug.
- History of adverse reactions during aerosol delivery of any medicinal product.
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 |
|---|---|---|
| AIR DNase | Drug | 1.25 mg, 2.5 mg, 5 mg |
| Placebo | Drug | Placebo |
Primary outcome
Adverse events following inhaled single dose (SD) of AIR-DNase
Time frame: 10 days
Adverse events from subject reporting or other assessments
Adverse events following inhaled multiple doses (MD) of AIR-DNase
Time frame: 14 days
Adverse events from subject reporting or other assessments
1 secondary outcome
Area under the curve
Time frame: 4 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2016.
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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.
Einat Almon, PhD study director
Protalix Ltd.
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 AIR DNase in people with Cystic Fibrosis (CF), run by Protalix at 1 site in Israel.
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 7 inclusion and 17 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: finished, as of the sponsor's last update on 17 May 2016. 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 Israel. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse events following inhaled single dose (SD) of AIR-DNase (time frame: 10 days). The study lists 2 primary outcomes in all. 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 17 May 2016.