E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011762 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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).
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E.2.2 | Secondary objectives of the trial |
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).
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal 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.
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E.4 | Principal 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.
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Change in baseline at 4 weeks of % predicted forced expiratory volume in one second (FEV1) 2. Adverse events (AE)
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | Yes |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 8 |