Safety Study of CTX-100 Inhalation Solution (Formerly ETX-100)

A Phase 2a, Ascending Dose, Placebo-Controlled Study to Evaluate the Safety of CTX-100 (Formerly ETX-100) Inhalation Solution in Subjects With Chronic Obstructive Pulmonary Disease

The purpose of the study is to evaluate the safety of administering repeated doses of CTX-100 (formerly ETX-100) to subjects with smoking-related chronic obstructive pulmonary disease.

Study Overview

• Status: Completed
• Conditions: Chronic Obstructive Pulmonary Disease (COPD)
• Intervention / Treatment: Drug: CTX-100 (formerly ETX-100) (hyaluronic acid)

Detailed Description

The study primarily aims to establish safety and evaluate biochemical activity of CTX-100 (formerly ETX-100) with respect to elastin breakdown, but will also concurrently gather data that may point to efficacy. The preliminary efficacy results will be used to assist in the design of subsequent phase 2b clinical studies of longer duration to examine both efficacy and safety of CTX-100 as a therapy for chronic obstructive pulmonary disease.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85712
      • Arizona Research Associates
  • New York
    • New York, New York, United States, 10019
      • St. Luke's-Roosevelt Hospital Center
    • New York, New York, United States, 10010
      • VA Harbor Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 75 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Able and willing to provide written informed consent and comply with study requirements.
• Men or women aged 40 through 75 years at the time of consent.
• At least 10-pack years (average packs/day x number of years) of cigarette use, and either current smoker or ex-smoker.
• Diagnosis of COPD at screening consistent with National Institutes of Health guidelines.
• Evidence of emphysema on chest x-ray.
• A ratio of prebronchodilator FEV1 to forced vital capacity (FVC) of ≤ 70% at screening.
• FEV1 ≥ 50% (prebronchodilator) and ≤ 79% (postbronchodilator) of predicted normal at screening.
• Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI/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 spermicide, or IUD) during the study and for 1 month after the final dose of study drug.

Exclusion Criteria:

• Subjects with a measured DLCO of ≤ 50%, or unable to perform a reproducible DLCO.
• Subjects unable to perform 3 reproducible spirometry tests after 5 attempts.
• Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day 1).
• Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) at screening or within the previous 12 months.
• Use of supplemental oxygen therapy.
• Requirement for ventilatory support within the last year.
• Exacerbation requiring treatment with systemic corticosteroids within the last 3 months.
• History of lung 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 (including subjects who participated in the first phase of this study).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: DOUBLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo	Drug: CTX-100 (formerly ETX-100) (hyaluronic acid); 3 mL of either 0.01%, 0.03% CTX-100 (formerly ETX-100), or placebo will be dispensed into the PARI LC STAR nebulizer and inhaled using the PARI Ultra II compressors. Number of cycles: if the study drug (0.01% CTX-100 Inhalation Solution or placebo) bid is well tolerated in the first cohort, the second cohort of subjects will begin dosing with 0.03% CTX-100 Inhalation Solution or placebo in the same manner as the first cohort.; Other Names: Hyaluronic acid; CTX-100; ETX-100
ACTIVE_COMPARATOR: 0.01% CTX-100 (formerly ETX-100)	Drug: CTX-100 (formerly ETX-100) (hyaluronic acid); 3 mL of either 0.01%, 0.03% CTX-100 (formerly ETX-100), or placebo will be dispensed into the PARI LC STAR nebulizer and inhaled using the PARI Ultra II compressors. Number of cycles: if the study drug (0.01% CTX-100 Inhalation Solution or placebo) bid is well tolerated in the first cohort, the second cohort of subjects will begin dosing with 0.03% CTX-100 Inhalation Solution or placebo in the same manner as the first cohort.; Other Names: Hyaluronic acid; CTX-100; ETX-100
ACTIVE_COMPARATOR: 0.03% CTX-100 (formerly ETX-100)	Drug: CTX-100 (formerly ETX-100) (hyaluronic acid); 3 mL of either 0.01%, 0.03% CTX-100 (formerly ETX-100), or placebo will be dispensed into the PARI LC STAR nebulizer and inhaled using the PARI Ultra II compressors. Number of cycles: if the study drug (0.01% CTX-100 Inhalation Solution or placebo) bid is well tolerated in the first cohort, the second cohort of subjects will begin dosing with 0.03% CTX-100 Inhalation Solution or placebo in the same manner as the first cohort.; Other Names: Hyaluronic acid; CTX-100; ETX-100

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Assessment of vital signs, laboratory tests, carbon monoxide (CO) diffusing capacity, oxygen saturation, electrocardiograms (ECGs), physical examinations, and adverse events	1 month

Secondary Outcome Measures

Outcome Measure	Time Frame
Assessment of elastin breakdown as measured by levels of elastin crosslinking amino acids desmosine/isodesmosine (D/I) in induced sputum, plasma, and urine.	1 month

Collaborators and Investigators

• Sponsor: Turino, Gerard, M.D.
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
• Investigators: Principal Investigator: Gerard Turino, M.D., St. Luke's-Roosevelt Hospital Center

Study record dates

Study Major Dates

• Study Start: November 1, 2010
• Primary Completion (ACTUAL): March 1, 2013
• Study Completion (ACTUAL): March 1, 2013

Study Registration Dates

• First Submitted: September 22, 2009
• First Submitted That Met QC Criteria: October 9, 2009
• First Posted (ESTIMATE): October 12, 2009

Study Record Updates

• Last Update Posted (ACTUAL): December 5, 2019
• Last Update Submitted That Met QC Criteria: December 3, 2019
• Last Verified: December 1, 2019

More Information

Terms related to this study

• Keywords: Chronic Obstructive Pulmonary Disease (COPD)
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Lung Diseases, Obstructive; Pulmonary Disease, Chronic Obstructive; Physiological Effects of Drugs; Immunologic Factors; Protective Agents; Adjuvants, Immunologic; Viscosupplements; Hyaluronic Acid
• Other Study ID Numbers: C100-005; 1R01HL081489-01A1 (NIH)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES