Cystic Fibrosis MedDRA version: 7.1 Level: LLT Classification code 10011762
Conditions
Interventions
Product Name: Recombinant truncated SPINT2 protease inhibitor
Product Code: AER002
Pharmaceutical Form: Nebuliser solution
Pharmaceutical form of the placebo: Nebuliser solution
Route of administratio
Sponsors
Aerovance Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female subjects 12 years of age or older 2. Subjects with mild to moderate CF defined as, FEV1 predicted between 47% and 84% with no exacerbation for 4 weeks 3. Subjects and/or parents or guardians who are able and willing to give written informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subjects who do not conform to the above inclusion criteria. 2. Subjects who have been on CF medication for less than 4 weeks (Part A) or less than 2 weeks (Part B). 3. Female subjects who are pregnant or breast-feeding, or of childbearing potential who are not using acceptable methods for contraception (reliable contraceptive measures include the following: systemic contraceptive [oral, implant, injections], diaphragm with intravaginal spermicide, cervical cap, intrauterine device, or condom with spermicide). 4. Subjects who have undergone lung transplant surgery. 5. Subjects who do not meet the following concomitant medication criteria: Part A: Subjects who have had changes in their CF medication (dose or medication type) in the 4 weeks prior to enrollment. Part B: Subjects who have had changes in their CF medication (dose or medication type) in the 2 weeks prior to Day 1 of subject dosing. 6. Subjects with any other clinically significant lung disease as determined by the investigator. 7. Any clinically significant disease or condition that may interfere with the compound or outcome of the study (e.g., metabolic conditions, renal, cardiac, or hepatic conditions) as determined by the investigator. 8. Subjects who cannot communicate reliably with the investigator. 9. Subjects who are unlikely to cooperate with the requirements of the study. 10. Subjects who have participated in an investigational study within 30 days prior to Day 1 of dosing.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the maximum safe and tolerable dose of AER 002 (Part A) and to assess the long-term safety and efficacy of this dose during 28 days of treatment (Part B). Efficacy will be measured primarily as improvement in forced expiratory volume in 1 second (FEV1). ;Secondary Objective: To determine the pharmacokinetic (PK) profile and maximum tolerable dose of AER 002 and its efficacy measured as change in symptoms (i.e., sputum volume and culture, cough, breathlessness, and daily lung function). ;Primary end point(s): 1. Change in baseline at 4 weeks of % predicted forced expiratory volume in one second (FEV1) 2. Adverse events (AE) | — |
Countries
United Kingdom
Outcome results
None listed