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

Part of paid clinical trials in Gainesville, Florida.

Sponsor
Krystal Biotech, Inc.
Study ID
NCT06049082
Phase
PHASE1
Status
Recruiting
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Conditions

  • Alpha 1-Antitrypsin Deficiency

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • KB408 (Nebulization) — DRUG
    Nebulized solution of KB408, a replication-defective HSV-1 vector expressing full length human SERPINA1

Study Details

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.

Key Dates

Start date
Feb 15, 2024
Status verified
Jul 2025
Primary completion
Dec 31, 2025
Completion
Dec 31, 2026

Study Design

Enrollment
15 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1: Low dose KB408
    Single dose of KB408 (low dose)
  • Experimental: Cohort 2: Mid dose KB408
    Single dose of KB408 (mid dose)
  • Experimental: Cohort 3: High dose KB408
    Single dose of KB408 (high dose)
  • Experimental: Cohort 2b: Mid dose KB408
    Multiple doses of KB408 (mid dose)

Primary Outcome Measure

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 [ Time Frame: 2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b) ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of Florida, GainesvilleGainesvilleFlorida32610
Allison Faunce
[email protected]
352-273-8666
Jorge Lascano, MD (PRINCIPAL_INVESTIGATOR)
Medical University of South CarolinaCharlestonSouth Carolina29425
Charlton Strange, MD
Charlton Strange, MD (PRINCIPAL_INVESTIGATOR)
Renovatio ClinicalThe WoodlandsTexas77380
Dustyn Tobin
[email protected]
713-703-2398
Ather J Siddiqi, MD (PRINCIPAL_INVESTIGATOR)

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University of Florida, Gainesville· Gainesville, FLMedical University of South Carolina· Charleston, SCRenovatio Clinical· The Woodlands, TX

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