Cystic FibrosisPhase 2NCT02722122
Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®
A Phase 2 study testing AIR DNase™ in people with Cystic Fibrosis, run by Protalix at 5 sites in Israel.
Trial summary
This study evaluates the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients with Cystic Fibrosis previously treated with Pulmozyme®. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- AIR DNase™
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesIsrael
- Study dates
- May 2016to Feb 2017
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Main Inclusion Criteria:
- Age ≥ 12 years of age (inclusive) at the time of screening
- Weight ≥ 36 kg
- Prior confirmed diagnosis of CF
- At least 4 months treatment with Pulmozyme® prior to screening.
- The subject is medically stable for at least one month prior to the screening visit.
- Stable inhaled regimen of either: antibiotics, steroids, hypertonic saline at least four months prior to screening visit.
- FEV1 of >40% and <90% and FVC ≥ 40% of predicted normal for age, gender, and height at screening .
- Female and male subjects whose co-partners are of child bearing potential must agree to use two medically acceptable methods of contraception, not including the rhythm method.
- Be willing and able to adhere to the study visit schedule and other protocol requirements
- Be willing and able to provide voluntary written informed consent
Main Exclusion Criteria:
- Has a history of lung transplantation.
- Female subjects who are pregnant or lactating.
- History of severe or unexplained adverse reactions during aerosol delivery of any medicinal product.
- History or presence of hypersensitivity or reaction to inhaled proteins.
- Participation in another clinical trial within 60 days prior to screening.
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's safety or compliance with the requirements of the study.
- Have positive serology for human immunodeficiency virus (HIV) or Hepatitis B or Hepatitis C infection.
Trial locations and contacts
5 sites in 1 country.
Einat Dekel, DVM contact
Yasmin Omer Azulay, MPH contact
5 sites in 1 country
Israel5 sites, 5 recruiting
- Recruiting
Carmel MC
Haifa
Michal Shteinberg, M.D., 04-8250564
Michal Shteinberg, M.D.
- Recruiting
Rambam MC
Haifa
Lea Bentur, M.D., 04-7774360
Lea Bentur, M.D.
- Recruiting
Hadassah MC
Jerusalem
Eitan Kerem, M.D., 02-6777749
Eitan Kerem, M.D.
- Recruiting
Schneider MC
Petah Tikva
Hanna Blau, M.D., 03-9253803
Hanna Blau, M.D.
- Recruiting
Sheba MC
Ramat Gan
Ori Efrati, M.D., 03-5302884
Ori Efrati, M.D.
About this study
Official title: A Proof-of-Concept, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®
This study evaluates the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients with Cystic Fibrosis previously treated with Pulmozyme®.
Read the detailed description
This is a proof-of-concept, open label study, to evaluate the safety, tolerability, pharmacokinetics and exploratory efficacy of 2.5 mg AIR DNase TM administered once daily for 28 days via inhalation to Cystic Fibrosis subjects who have previously been treated with Pulmozyme®.
This multicenter study will be conducted in approximately 10 sites and will enroll up to 15 subjects.
- Conditions
- Cystic Fibrosis
- Being tested
- AIR DNase™
- Keywords
- Cystic Fibrosis, CF, AIR DNase™, Digestive System Diseases, Genetic Diseases, Inborn, Infant, New born, Diseases, Lung diseases, Pancreatic Diseases, Respiratory Tract Diseases
- Medical subject headings
- Cystic Fibrosis, Digestive System Diseases, Genetic Diseases, Inborn, Disease, Lung Diseases, Pancreatic Diseases, Respiratory Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AIR DNase™Drug
| Study group | Role | What they receive |
|---|---|---|
| AIR DNase™ 2.5 mg | Experimental | 2.5 mg of AIR DNase™ administered once daily via inhalation for 28 days Receives: Drug: AIR DNase™ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events following daily administration of AIR DNase™
Adverse events from subject reporting or other assessments
Measured: 56 days
Secondary outcomes (3)
Area under the curve
AIR DNase concentrations measured at 0, 0.25, 0.5, 0.75, 1, 1.5, 2, and 4 hours
Measured: Up to 4 hours
Change from baseline to end of AIR DNase™ treatment in FEV1
Measured: Baseline and 28 days
Change from baseline to end of AIR DNase™ treatment in FVC
Measured: Baseline and 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2016 (estimated)
- Study startMay 2016
- Primary completionFeb 2017 (estimated)
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Einat Dekel, DVM, Study director, Sr. Director Clinical Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02722122
- Sponsor's protocol ID
- PB-110-CF02
- Status last verified
- Sep 2016
- First submitted
- 23 Mar 2016
- Last updated
- 28 Sep 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Proof-of-Concept, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®
- ClinicalTrials.gov ID
- NCT02722122
- Acronym / sponsor ID
- PB-110-CF02
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CF, AIR DNase™, Digestive System Diseases, Genetic Diseases, Inborn, Infant, New born, Diseases, Lung diseases, Pancreatic Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- Feb 2017 (estimated)
- First posted
- 29 Mar 2016 (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 evaluates the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients with Cystic Fibrosis previously treated with Pulmozyme®.
Detailed description
This is a proof-of-concept, open label study, to evaluate the safety, tolerability, pharmacokinetics and exploratory efficacy of 2.5 mg AIR DNase TM administered once daily for 28 days via inhalation to Cystic Fibrosis subjects who have previously been treated with Pulmozyme®.
This multicenter study will be conducted in approximately 10 sites and will enroll up to 15 subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AIR DNase™ 2.5 mg | Experimental | Drug: AIR DNase™2.5 mg of AIR DNase™ administered once daily via inhalation for 28 days |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 | AIR DNase™ 2.5 mg |
Primary outcome
Adverse events following daily administration of AIR DNase™
Time frame: 56 days
Adverse events from subject reporting or other assessments
3 secondary outcomes
Area under the curve
Time frame: Up to 4 hours
Change from baseline to end of AIR DNase™ treatment in FEV1
Time frame: Baseline and 28 days
Change from baseline to end of AIR DNase™ treatment in FVC
Time frame: Baseline and 28 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2016.
Related trials for Cystic Fibrosis
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 2
Phase 2 Study to Assess the Safety and Efficacy of ANG003
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Recruiting
Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
Cystic Fibrosis (CF), Bronchiectasis, Non-Cystic Fibrosis
Not yet recruitingObservational study
Vascular Health and Function in Cystic Fibrosis
Cystic Fibrosis, Endothelial Dysfunction
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
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 Dekel, DVM contact
Yasmin Omer Azulay, MPH contact
Einat Dekel, DVM study director
Sr. Director Clinical Development
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AIR DNase™ in people with Cystic Fibrosis, run by Protalix at 5 sites in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: status not confirmed, as of the sponsor's last update on 28 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Einat Dekel, DVM (Einat.Dekel@protalix.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events following daily administration of AIR DNase™ (time frame: 56 days). 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 28 Sep 2016.