SAV006-04
10 evidence topics · 3 sources
Objective
Pediatric efficacy evaluation
Location and study date
Listed study location and planned dates
| Attribute | Reported value |
|---|---|
| Listed country | “Germany” |
| Study start | “October 22, 2025” |
| Estimated completion | “December 31, 2027” |
Study design
Eligibility criteria
Specified age limits and diagnostic confirmation
Pulmonary gas-transfer criterion
| Measure | Screening threshold |
|---|---|
| Hemoglobin-adjusted DLCO percent predicted | “≤70%” |
Treatment
Registered inhaled treatment
| Treatment component | Quoted specification |
|---|---|
| Maximum daily dose | “300 µg microgram(s)” |
| Route | “INHALATION USE” |
| Maximum treatment duration | “48 Week(s)” |
Study outcomes
Rationale for using hemoglobin-adjusted DLCO percent predicted as the primary endpoint
Pediatric-specific endpoint validation
No evidence found.
Statistical analysis description
Number of participants (Planned and analyzed)
Planned enrollment
| Enrollment measure | Participants |
|---|---|
| Planned total | “5” |
Analyzed population
No evidence found.
Description of analysis sets
No evidence found.
Results
Participant disposition
No evidence found.
Baseline characteristics
No evidence found.
Efficacy results
No evidence found.
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
No evidence found.
Study limitations
Summary
The registered study has one open-label treatment group and a planned enrollment of five participants. No analyzed results are included in this report. Its design does not provide a concurrent placebo comparison and will provide limited precision for uncommon adverse events. The inclusion criteria specify age at least 6 years and less than 18 years, which is narrower than the older narrative description of ages 6 through 18.