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A Study of KB408 for Alpha-1 Antitrypsin Deficiency

This study is looking into KB408, a new treatment for Alpha-1 Antitrypsin Deficiency (AATD). KB408 is given through a nebulizer (a device that turns liquid medicine into a mist you can breathe in). The main goal of this study is to check the safety of KB408 and how well people tolerate it. Researchers will look at side effects, vital signs, heart activity (ECG), lung function (spirometry), and blood tests over 2 or 3 months. You may be able to join if you are 18 to 70 years old and have AATD with a PI*ZZ or PI*ZNull genotype. The study plans to enroll 15 participants. The current status of this study is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 15 participants to test different dose levels of KB408.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Safety and tolerability will be assessed for 2 months (for Cohorts 1, 2, 3) or 3 months (for Cohort 2b) after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06049082

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

Recruiting
PHASE1Ages 18–70InterventionalTreatment
Krystal Biotech, Inc.
~15 participants
Updated 2025-07-22 on ClinicalTrials.gov
What's tested:KB408 (Nebulization)

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
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
Measured over 2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)

Conditions

Alpha 1-Antitrypsin Deficiency

Explore related trials

Alpha-1 Antitrypsin DeficiencySERPINA1 Gene

Where it's being run

3 sites across 3 states
Florida1
South Carolina1
Texas1

Study leadership

  • David Sweet, MD, PhD · STUDY_DIRECTOR · Director of Clinical Development

Who to contact

David Sweet, MD, PhD
[email protected]
412-586-5830
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Exclusion

Platelet count \<100×10\^9/L
Hemoglobin \<9 g/dL
White blood cell count \<3 or \>15×10\^9/L
Sodium \<130 or \>150 mmol/L
Potassium \<3 or \>5.5 mmol/L
Carbon dioxide \<16 mmol/L
Creatinine \>2 mg/dL 15. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator. 16. Females who are pregnant or nursing. 17. Subject who is unwilling to comply with contraception requirements per protocol

What this trial measures

  • 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 results2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)

    Number of subjects with treatment related adverse events as assessed by NCI-CTCAE v5