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New treatment option for Alpha 1-Antitrypsin Deficiency
Official title A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
ClinicalTrials.gov ID: NCT06049082
What this study is testing
What is KB408 (Nebulization)?
KB408 (Nebulization) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for alpha 1-antitrypsin deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70
You may be able to join if
- The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent...
- Subject is aged ≥18 to ≤70 years, at the time of informed consent.
- Subject has a genetically confirmed diagnosis of AATD with a PI\ ZZ or PI\ ZNull genotype.
- Cohort 2b and Cohort 3: people receiving AAT augmentation therapy must be willing to washout for at least 10 days prior to Screening and be willing...
- Cohort 2b and Cohort 3: Serum AAT level \<11 μM at Screening.
You likely can't join if
- Pulmonary function test with percent predicted forced expired volume in 1 second (ppFEV1) after inhalation of a bronchodilator is \<40% at Screening.
- Diffusing capacity of the lungs for carbon monoxide (DLCO) \<30 percent predicted (historical DLCO within 2 years prior to Screening without any...
- Known ongoing or history of clinically significant pulmonary impairment other than AATD.
- A pulmonary exacerbation within six weeks (42 days) of first dose.
- Initiation of any new chronic therapy or any change in ongoing therapy routine within 28 days of first dose.
- Participation in another treatment clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of...
See the full eligibility criteria
- The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and...
- Subject is aged ≥18 to ≤70 years, at the time of informed consent.
- Subject has a genetically confirmed diagnosis of AATD with a PI\ ZZ or PI\ ZNull genotype.
- Cohort 2b and Cohort 3: people receiving AAT augmentation therapy must be willing to washout for at least 10 days prior to Screening and be willing to remain off augmentation therapy for the duration of the study.
- Cohort 2b and Cohort 3: Serum AAT level \<11 μM at Screening.
- Willing to remain on a stable regimen of treatment during the study.
- Resting oxygen saturation ≥92% on room air at Screening.
- Clinically stable and in good general health, except for AATD, as determined by the Investigator.
- Pulmonary function test with percent predicted forced expired volume in 1 second (ppFEV1) after inhalation of a bronchodilator is \<40% at Screening.
- Diffusing capacity of the lungs for carbon monoxide (DLCO) \<30 percent predicted (historical DLCO within 2 years prior to Screening without any intervening change in clinical status since the measurement was taken, or...
- Known ongoing or history of clinically significant pulmonary impairment other than AATD.
- A pulmonary exacerbation within six weeks (42 days) of first dose.
- Initiation of any new chronic therapy or any change in ongoing therapy routine within 28 days of first dose.
- Participation in another treatment clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of first dose. Previous treatment with a genetic therapy for AATD, where...
- History of or listed for solid organ transplantation or has undergone major lung surgery (e.g., lobectomy) within 6 months of first dose.
- Any clinical condition or illness (including a history or current evidence of substance abuse or dependence) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related...
- An active oral herpes infection 30 days prior to the first dose.
- Clinically significant hepatic dysfunction defined as any one of the following:
- AST and ALT ≥3× upper limit of normal (ULN) at Screening
- Total bilirubin ≥2× ULN at Screening (unless associated with Gilbert's syndrome)
- Evidence of liver cirrhosis with clinical manifestations of portal hypertension (e.g., ascites, encephalopathy, variceal hemorrhage)
- History of cigarette smoking or any other tobacco use, or use of e-cigarettes or other recreational inhalant, within 6 months of Screening.
- Unwilling to refrain from smoking, e-cigarette use, or vaping throughout the duration of the study.
- A positive urine cotinine result that is consistent with active smoking at Screening. (A positive cotinine test due to nicotine replacement therapy for the purpose of smoking cessation, as attested by the Investigator...
- Abnormal hematology or chemistry testing at Screening as defined below, or any other clinically significant abnormalities that the Investigator believes may interfere with the assessment of safety of the study treatment.
- Platelet count \<100×10\^9/L
- Hemoglobin \<9 g/dL
- White blood cell count \ 15×10\^9/L
- Sodium \ 150 mmol/L
- Potassium \ 5.5 mmol/L
- Carbon dioxide \<16 mmol/L
- Creatinine \>2 mg/dL
- Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.
- Females who are pregnant or nursing.
- Subject who is unwilling to comply with contraception requirements per protocol
The study team makes the final eligibility decision.
Where it's taking place
- Gainesville, Florida, United States
- Charleston, South Carolina, United States
- The Woodlands, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Gainesville, Florida, United States; Charleston, South Carolina, United States; The Woodlands, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.