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KB408 · Serpentine-1

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

Ensayo de Krystal Biotech, Inc. en Deficit de alfa-1-antitripsina.

Más ensayos de: Déficit de alfa-1-antitripsina.

Fase

Fase 1

Estado

Reclutando pacientes

Participantes

15

previstos

Centros

3

Fin del objetivo primario

dic 2025

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en feb 2024.

Qué mide

To evaluate safety and tolerability of KB408 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in vital signs, ECG, spirometry, and clinical laboratory test results (2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe 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.

Más ensayos de KRYS

Todos los de KRYS, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06049082), actualizada en jul 2025.