Ignite Creation Date:
2024-05-06 @ 7:31 PM
Last Modification Date:
2024-10-26 @ 3:09 PM
Study NCT ID:
NCT06049082
Status:
RECRUITING
Last Update Posted:
2024-05-06
First Post:
2023-09-15
Brief Title:
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Sponsor:
Krystal Biotech Inc
Organization:
Krystal Biotech Inc
Study Overview
Official Title:
A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Serpentine-1
Brief 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 PIZZ or PIZNull genotype Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts At the high dose two cohorts will be conducted in parallel to evaluate patients on and off IV augmentation therapy
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None