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NCT06049082 · ClinicalTrials.gov registry record · Phase 1
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
A Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by Krystal Biotech.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 3
- Study locations
NCT06049082: Recruiting Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by Krystal Biotech.
NCT06049082 is a Phase 1 study of Alpha-1 Antitrypsin Deficiency that is actively recruiting participants, run by Krystal Biotech. The registered enrollment target is 15 participants, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06049082, a Phase 1 study of Alpha-1 Antitrypsin Deficiency, is actively recruiting participants, sponsored by Krystal Biotech.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 3
- Study locations
Study Summary
The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\*ZZ or PI\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.
Primary Outcome
Number of subjects with treatment related adverse events as assessed by NCI-CTCAE v5
Conditions Studied
Interventions
- DRUG KB408 (Nebulization)
Study Locations (3)
Florida
- University of Florida, Gainesville - Gainesville
South Carolina
- Medical University of South Carolina - Charleston
Texas
- Renovatio Clinical - The Woodlands
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2024-02-15 |
| Est. Completion | 2026-12 |
| Phase | Phase 1 |
What NCT06049082 shows while recruiting
NCT06049082 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Alpha-1 Antitrypsin Deficiency appearing as the primary indexed condition, and to 1 intervention - of which KB408 (Nebulization) is the first listed.
NCT06049082 reports a single indexed study location in Florida, South Carolina, Texas.
Frequently Asked Questions
What is clinical trial NCT06049082 about?
NCT06049082 is a clinical study titled "A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency". The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to eva...
What is the current status of trial NCT06049082?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2024-02-15. Estimated completion is 2026-12.
What conditions does trial NCT06049082 study?
This clinical trial studies the following conditions: Alpha-1 Antitrypsin Deficiency.
What interventions are being tested in trial NCT06049082?
The interventions under investigation include: KB408 (Nebulization) (DRUG).
Who is sponsoring clinical trial NCT06049082?
This trial is sponsored by Krystal Biotech, which has 13 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06049082 being conducted?
This trial has 3 study locations across Florida, South Carolina, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06049082's enrollment target sits among peer trials
15 21st of 24 higher than 4 of 24 other Alpha-1 Antitrypsin Deficiency trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Alpha-1 Antitrypsin Deficiency trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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