EmphysemaPhase 2NCT03114020
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
A Phase 2 study testing Hyaluronic Acid Inhalation Solution in people with Emphysema and Alpha 1-Antitrypsin Deficiency, run by Gerard Turino at 6 sites in 2 countries.
Trial summary
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Emphysema, Alpha 1-Antitrypsin Deficiency
- What is being tested?
- Hyaluronic Acid Inhalation Solution
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 22 Mar 2017to 12 Nov 2019
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able and willing to provide written informed consent and comply with study requirements
- Men or women aged 18 through 80 years at the time of consent
- Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III
- Evidence of emphysema on radiographic imaging.
- A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening
- FEV1 ≥ 30% and ≤ 79% (post-bronchodilator) of predicted normal at screening
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- Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI and sponsor at screening
- Women of childbearing potential and men who are sexually active must agree to use an adequate method of contraception (oral contraceptives, depot progesterone, condom plus spermicidal, or IUD) during the study and for 1 month after the final dose of study drug.
- Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ deficiency. Individuals with PiMZ deficiency are not allowed in the study.
- Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT) augmentation therapy at least 3 months before entering study.
Who cannot join
- Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO
- Subjects unable to perform 3 reproducible spirometry tests after 8 attempts
- Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1)
- Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months
- Use of supplemental oxygen therapy
- Requirement for ventilator support within the last year
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- Exacerbation requiring treatment with systemic corticosteroids within the last 3 months
- History of lung transplant or liver transplant.
- Presence of clinically relevant abnormality on electrocardiogram (ECG)
- Any medical condition that, in the investigator's judgment, would compromise study participation or the evaluation of the study drug
- Women who are pregnant or breastfeeding
- Receipt of an investigational drug within 30 days prior to screening
- Patients who are current smokers or have smoked within the last 3 months -
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Pulmonary Health Physicians, PC
Fayetteville, New York, 13066
University of Miami Hospital
Miami, Florida, 33136
Medical College of Wisconsin / Froedtert Hospital
Milwaukee, Wisconsin, 53226
University of Texas Health Science Center
Tyler, Texas, 75708
About this study
Official title: Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency
Read the detailed description
The study primarily aims to establish desmosine and isodesmosine concentrations in plasma, sputum and urine measured as markers of elastin degradation systemically in the lung and also markers of inflammation and fibrinogen. Assessment of vital signs, lab tests, carbon monoxide diffusing capacity, oxygen saturation, pulmonary function tests, ECGs, physical exams and adverse events.
- Conditions
- Emphysema, Alpha 1-Antitrypsin Deficiency
- Being tested
- Hyaluronic Acid Inhalation Solution, Placebo Inhalation Solution
- Keywords
- Emphysema, COPD, Alpha-1 Antitrypsin Deficiency
- Medical subject headings
- Emphysema, alpha 1-Antitrypsin Deficiency, Pulmonary Disease, Chronic Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hyaluronic Acid Inhalation SolutionDrug
Also called active inhalation solution
twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution
Placebo Inhalation SolutionDrug
Twice a day 3 ml of placebo inhalation solution
| Study group | Role | What they receive |
|---|---|---|
| Hyaluronic Acid inhalation solution | Experimental | 3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days Receives: Drug: Hyaluronic Acid Inhalation Solution |
| Placebo Inhalation Solution | Placebo comparator | 3mL matching placebo inhalation solution BID for 28 days Receives: Drug: Placebo Inhalation Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine
measurement of biomarkers
Measured: 28 days
Assessment of pulmonary function tests
measurement of pulmonary function
Measured: 28 days
Secondary outcomes (1)
Assessment of St. George Respiratory Questionnaire
measurement of patient abilities
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2017
- Study start22 Mar 2017
- Primary completion12 Nov 2019
- Study completion12 Nov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EmphysemaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gerard Turino
- Responsible party
- Sponsor-investigator, Gerard Turino, Turino, Gerard, M.D.
- Investigators
- Gerard M Turino, MD, Principal investigator, Mount Sinai St Lukes
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03114020
- Sponsor's protocol ID
- C100-008
- Status last verified
- Apr 2020
- First submitted
- 5 Apr 2017
- Last updated
- 21 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema
- ClinicalTrials.gov ID
- NCT03114020
- Acronym / sponsor ID
- C100-008
- Conditions
- Emphysema, Alpha 1-Antitrypsin Deficiency
- Keywords
- Emphysema, COPD, Alpha-1 Antitrypsin Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Gerard Turino
- Study start
- 22 Mar 2017 (actual)
- Primary completion
- 12 Nov 2019 (actual)
- Study completion
- 12 Nov 2019 (actual)
- First posted
- 14 Apr 2017 (actual)
- 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.
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency
Detailed description
The study primarily aims to establish desmosine and isodesmosine concentrations in plasma, sputum and urine measured as markers of elastin degradation systemically in the lung and also markers of inflammation and fibrinogen. Assessment of vital signs, lab tests, carbon monoxide diffusing capacity, oxygen saturation, pulmonary function tests, ECGs, physical exams and adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hyaluronic Acid inhalation solution | Experimental | Drug: Hyaluronic Acid Inhalation Solution3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days |
| Placebo Inhalation Solution | Placebo comparator | Drug: Placebo Inhalation Solution3mL matching placebo inhalation solution BID for 28 days |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Able and willing to provide written informed consent and comply with study requirements
- Men or women aged 18 through 80 years at the time of consent
- Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III
- Evidence of emphysema on radiographic imaging.
- A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening
Key exclusion criteria (13 listed)
- Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO
- Subjects unable to perform 3 reproducible spirometry tests after 8 attempts
- Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1)
- Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months
- Use of supplemental oxygen therapy
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 |
|---|---|---|
| Hyaluronic Acid Inhalation SolutionAlso: active inhalation solutiontwice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution | Drug | Hyaluronic Acid inhalation solution |
| Placebo Inhalation SolutionTwice a day 3 ml of placebo inhalation solution | Drug | Placebo Inhalation Solution |
Primary outcome
Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine
Time frame: 28 days
measurement of biomarkers
Assessment of pulmonary function tests
Time frame: 28 days
measurement of pulmonary function
1 secondary outcome
Assessment of St. George Respiratory Questionnaire
Time frame: 28 days
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 21 Apr 2020.
- Individual participant dataSharing planned: No
Related trials for Emphysema
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A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
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Xenon MRI to Evaluate Changes to Lung Function Following Bronchoscopic Treatment of Air Trapping in Emphysema
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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.
Gerard M Turino, MD principal investigator
Mount Sinai St Lukes
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hyaluronic Acid Inhalation Solution in people with Emphysema and Alpha 1-Antitrypsin Deficiency, run by Gerard Turino at 6 sites in 2 countries.
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 10 inclusion and 13 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 21 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Canada. 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 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?
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?
Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine (time frame: 28 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 21 Apr 2020.