Nebulized KB407 for Cystic Fibrosis

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

- Two copies of a disease causing mutation in the CFTR gene

Subjects aged 18 years or older at the time of Informed Consent

Must not have

Treatment for Burkholderia cenocepacia, Burkholderia dolosa, or Nontuberculosis Mycobacteria infection within 3 months prior to the first dose

Subject who is unwilling to comply with contraception requirements per protocol

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 months

Awards & highlights

No Placebo-Only Group

Summary

This trial will study whether it is safe to give increasing doses of nebulized KB407 to adults with cystic fibrosis.

Who is the study for?

Adults with cystic fibrosis who understand and agree to the study's procedures can join. They must have a confirmed diagnosis, stable health, specific lung function levels, normal oxygen saturation without assistance, and two CFTR gene mutations. Excluded are those recently ill or hospitalized, vaccinated close to treatment start, on certain antibiotics recently or with conditions that could affect safety assessments.

What is being tested?

The trial is testing KB407 through nebulization to see how safe it is and how well people tolerate different doses. It's for adults with cystic fibrosis. Participants will receive ascending doses of KB407 via a nebulizer which turns liquid medication into a mist.

What are the potential side effects?

Specific side effects of KB407 aren't listed but generally include reactions related to inhaling medication such as coughing or wheezing; however, the trial primarily aims to assess these potential side effects.

Eligibility Criteria

Inclusion Criteria

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

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I have two CFTR gene mutations causing my condition.

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I am 18 years old or older.

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My lung function is between 50% and 100% of what is expected for my age, gender, and height.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't been treated for specific bacterial infections in the last 3 months.

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I am willing to follow the study's birth control requirements.

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I am not pregnant or nursing.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results

Secondary study objectives

To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1