E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
RSV lower respiratory tract infection |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Virus Diseases [C02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061603 |
E.1.2 | Term | Respiratory syncytial virus infection |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
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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 evaluate the anti-viral effect and safety of different doses of inhaled ALX-0171 in subjects hospitalized for RSV lower respiratory tract infection. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the clinical activity, pharmacokinetic (PK) properties, pharmacodynamic (PD) effect and immunogenicity of different doses of inhaled ALX-0171. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject is a male or female infant or young child aged 28 days to < 2 years with gestational age ≥ 33 weeks.
- Subject is otherwise healthy, but is hospitalized for and clinically diagnosed with RSV lower respiratory tract infection.
- Subject has a positive RSV diagnostic test.
- Subject is expected to have to stay in the hospital for at least 24 hours.
- Parent(s)/legal guardian(s) provide written informed consent in accordance with locally approved consent process.
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E.4 | Principal exclusion criteria |
- Subject is known to have significant comorbidities
- Subject is known to be HIV-positive (or if the child is <6 months of age, the mother is known to be HIV-positive).
- Subject is known to be immunocompromised.
- Subject has or is suspected to have an active, clinically relevant concurrent infection
- Subject is critically ill and/or is expected to require invasive mechanical ventilation
- Subject is currently participating in any other study with investigational drug or has received an investigational drug within 4 weeks or 5 half-lives of the concerned drug (whichever is longer) prior to screening
- Subject was previously enrolled in a clinical study of ALX-0171
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E.5 End points |
E.5.1 | Primary end point(s) |
Anti-viral effect, as measured by the time needed for the viral load to drop below the assay quantification limit. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the in-hospital period, up to Day 14 after randomization |
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E.5.2 | Secondary end point(s) |
- Safety assessment (assessed by physical examination, AEs, laboratory assessments and vital signs) of different doses of ALX-0171
- Change from baseline in Global Severity Score
- Time to clinical response
- PK properties of ALX-0171
- Viral load profile over time
- Evaluation of serum anti-drug antibodies (ADA)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Throughout the study, up to Day 28 after randomization |
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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 | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Phase 2b dose-ranging study |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
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.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 42 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Brazil |
Chile |
Colombia |
Israel |
Malaysia |
Philippines |
South Africa |
Thailand |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 23 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 23 |