Is NCT06049082 recruiting in The Woodlands, TX?

Yes, NCT06049082 is currently recruiting participants at its The Woodlands, TX location. This alpha 1-antitrypsin deficiency clinical trial is actively enrolling qualified participants.

Is participation in this The Woodlands clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Alpha 1-Antitrypsin Deficiency Clinical Trial in The Woodlands, TX

Q: How do I join the alpha 1-antitrypsin deficiency clinical trial in The Woodlands?

To join this clinical trial in The Woodlands, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

About This Alpha 1-Antitrypsin Deficiency Study in The Woodlands

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.

Sponsor: Krystal Biotech, Inc.

Who Can Participate

Inclusion 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 instructions.

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: Subjects 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.

Exclusion Criteria

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 as measured at Screening).

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 interventional 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 the investigational product was demonstrated to be non-efficacious, is not exclusionary.

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 procedures and/or poses an additional risk to the assessment of safety of KB408.

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, is allowed.)

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 \<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

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in The Woodlands?

Yes, this clinical trial (NCT06049082) has an active research site in The Woodlands, TX that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Alpha 1-Antitrypsin Deficiency Treatment Options in The Woodlands, TX

If you're searching for alpha 1-antitrypsin deficiency treatment options in The Woodlands, TX, this clinical trial (NCT06049082) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our The Woodlands research site is actively enrolling participants for this clinical trial. You'll receive care from experienced alpha 1-antitrypsin deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all alpha 1-antitrypsin deficiency clinical trials near you to find additional studies recruiting in your area.

Alpha 1-Antitrypsin Deficiency Clinical Trial in The Woodlands, TX

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