Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03114020
• Date of registration: 05/04/2017
• Prospective Registration: No
• Primary sponsor: Gerard Turino
• Public title: Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
• Scientific 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
• Date of first enrolment: March 22, 2017
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03114020
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

Canada, United States

Contacts

• Name: Gerard M Turino, MD
• Affiliation: Mount Sinai St Lukes

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Able and willing to provide written informed consent and comply with study
requirements

2. Men or women aged 18 through 80 years at the time of consent

3. Diagnosis of emphysema at screening consistent with National Institutes of Health
guidelines 19 GOLD COPD classification stages I, II or III

4. Evidence of emphysema on radiographic imaging.

5. A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of = 80% at
screening

6. FEV1 = 30% and = 79% (post-bronchodilator) of predicted normal at screening

7. Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis)
within normal limits or clinically acceptable to the PI and sponsor at screening

8. 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.

9. Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ
deficiency. Individuals with PiMZ deficiency are not allowed in the study.

10. Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT)
augmentation therapy at least 3 months before entering study.

Exclusion Criteria:

1. Subjects with measured DLCO of = 35%, or unable to perform a reproducible DLCO

2. Subjects unable to perform 3 reproducible spirometry tests after 8 attempts

3. Upper or lower respiratory tract infection within 2 weeks prior to screening and
baseline (day1)

4. Presence of clinically relevant abnormality on chest x-ray (other than evidence of
emphysema) within the previous 12 months

5. Use of supplemental oxygen therapy

6. Requirement for ventilator support within the last year

7. Exacerbation requiring treatment with systemic corticosteroids within the last 3
months

8. History of lung transplant or liver transplant.

9. Presence of clinically relevant abnormality on electrocardiogram (ECG)

10. Any medical condition that, in the investigator's judgment, would compromise study
participation or the evaluation of the study drug

11. Women who are pregnant or breastfeeding

12. Receipt of an investigational drug within 30 days prior to screening

13. Patients who are current smokers or have smoked within the last 3 months -



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Alpha 1-Antitrypsin Deficiency

Emphysema

Intervention(s)

Drug: Placebo Inhalation Solution

Drug: Hyaluronic Acid Inhalation Solution

Primary Outcome(s)

Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine [Time Frame: 28 days]

Assessment of pulmonary function tests [Time Frame: 28 days]

Secondary Outcome(s)

Assessment of St. George Respiratory Questionnaire [Time Frame: 28 days]

Secondary ID(s)

C100-008

Source(s) of Monetary Support

Please refer to primary and secondary sponsors