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Virus Therapy

Nebulized KB408 for Alpha-1 Antitrypsin Deficiency (Serpentine-1 Trial)

Phase 1

Recruiting

Research Sponsored by Krystal Biotech, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has a genetically confirmed diagnosis of AATD with a PI*ZZ genotype.

Cohort 3b: Subjects receiving AAT augmentation therapy must be willing to washout for at least 6 weeks (42 days) prior to Screening and be willing to remain off augmentation therapy for the duration of the study.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 months

Awards & highlights

Serpentine-1 Trial Summary

This trial is evaluating a new drug to help people with a genetic lung disorder who need extra support to breathe. It will be given as a nebulized spray and will be tested at three different doses.

Who is the study for?

Adults aged 18-70 with Alpha-1 Antitrypsin Deficiency and PI*ZZ genotype can join. They must be in good health except for AATD, not smoke, and agree to avoid certain medications. Those on augmentation therapy must stop it before joining the trial.Check my eligibility

What is being tested?

KB408 is a new treatment delivered through nebulization aiming to provide a functional SERPINA1 gene to those with AATD. The study will test three different doses of KB408 in adults, some of whom may also be receiving IV augmentation therapy.See study design

What are the potential side effects?

Potential side effects are not explicitly listed but could include reactions related to herpes simplex virus components used in the vector or issues from inhaling medication via nebulization.

Serpentine-1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been genetically diagnosed with AATD and have the PI*ZZ genotype.

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I am willing to stop my AAT therapy for 6 weeks before the study and not use it during the study.

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I am between 18 and 70 years old.

Serpentine-1 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome 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 results

Secondary outcome measures

To assess the effect of KB408 on plasma neutrophil elastase (NE) concentration

To assess the effect of KB408 on serum alpha-1 antitrypsin (AAT) concentration

To evaluate the effect of KB408 on AAT concentration in the lung

+1 more

Serpentine-1 Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort 3a/3b: High dose KB408Experimental Treatment1 Intervention

Single dose of KB408 (high dose)

Group II: Cohort 2: Mid dose KB408Experimental Treatment1 Intervention

Single dose of KB408 (mid dose)

Group III: Cohort 1: Low dose KB408Experimental Treatment1 Intervention

Single dose of KB408 (low dose)

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Krystal Biotech, Inc.Lead Sponsor

12 Previous Clinical Trials

418 Total Patients Enrolled

David Chien, MDStudy DirectorSenior Vice President of Clinical Development

6 Previous Clinical Trials

231 Total Patients Enrolled

Hubert Chen, MDStudy DirectorKrystal Biotech

3 Previous Clinical Trials

332 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the FDA given its stamp of approval to Cohort 1: Low dose KB408?

"The safety of Low Dose KB408 has been assessed as a 1 on our 3-point scale, owing to the fact that this is an early Phase I study with minimal supportive data."

Answered by AI

Does the eligibility criteria extend to individuals 45 and over for this trial?

"According to the eligibility requirements for this clinical trial, individuals aged 18 or above and 70 or below can register."

Answered by AI

Which type of individuals would benefit most from participating in this experiment?

"This research trial has a set of specific requirements for eligibility. Those with alpha-1 antitrypsin deficiency, aged between 18 and 70 years old may apply to join the study which is looking to recruit around 12 people in total."

Answered by AI

Are there vacancies within this research program for participants?

"According to clinicaltrials.gov, this medical study has ceased recruitment at the moment. Initially posted on January 1st 2024 and last modified on September 20th 2023, it is not currently looking for participants; however there are 15 other trials actively searching for volunteers."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06049082.