Testing KRRO-110 for Alpha-1 Anti-trypsin Deficiency
A Phase 1/2a, Single- and Multiple-dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KRRO 110 in Healthy Adult Volunteers and in Adult Participants With Alpha-1 Antitrypsin Deficiency (AATD) (REWRITE)
This study is testing a new drug called KRRO-110 to see if it is safe and how it works in people with Alpha-1 antitrypsin deficiency and healthy adults.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 64 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Korro Bio, Inc. Industry-sponsored |
| Locations | 3 sites (Melbourne, Victoria and 2 other locations) |
| Trial ID | NCT06677307 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety, tolerability, and pharmacokinetics of KRRO-110 in both healthy adults and patients with Alpha-1 antitrypsin deficiency (AATD). It includes a single and multiple-dose escalation approach to determine how the drug behaves in the body and its effects on individuals with the condition. Participants will be divided into cohorts based on their genotype and health status, allowing for a comprehensive assessment of the drug's impact.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 to 70 with a documented PiZZ genotype and low baseline AAT levels.
Not a fit: Patients with other significant health conditions or those outside the specified age and genotype criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for patients suffering from Alpha-1 antitrypsin deficiency.
How similar studies have performed: While this is a first-in-human study, similar approaches in gene therapy and protein replacement for genetic disorders have shown promise in other contexts.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Part A SAD cohort Inclusion Criteria (Healthy Volunteers) * Adult male or female participants, 18 to 65 years of age * Documented PiMM genotype * Participants who are willing to and able to provide signed written informed consent PiZZ Genotype (Part A SAD and Part B MAD cohorts) Inclusion Criteria (PiZZ Genotype) * Adult male or female participants 18 to 70 years of age (inclusive) * Documented PiZZ genotype * Baseline blood total AAT level \< 11 µM/L Exclusion Criteria: Exclusion Criteria (Healthy Volunteers) * Female participant of childbearing potential or male participant that is unable or unwilling to use an approved, reliable means of contraception * Body mass index (BMI) \> 32 or \< 18.5 kg/m2 * History or current clinical evidence of hepatic disease * Evidence of active infection * History of medical condition(s), eg, advance cardiac disease, current or recent malignancy, organ transplantation, or other illness * Serology result indicative of any exposure (past or active) to hepatitis C, hepatitis B, or human immunodeficiency virus (HIV) * Respiratory or other acute illness within 8 weeks * Tobacco use of any kind within 6 months Exclusion Criteria (PiZZ Genotype) * Female participant of childbearing potential or male participant that is unable or unwilling to use an approved, reliable means of contraception * BMI \> 32 or \< 18.5 kg/m2 or weight \> 90 kg * History of FEV1 \< 50% * History or current clinical evidence of advanced hepatic disease and/or pulmonary disease * Use of an experimental therapy except KRRO-110 within 6 months or 5 half-lives for the experimental therapy, whichever is greater * Tobacco use of any kind within 6 months * Use of conventionally dosed AAT augmentation therapy within 5 half-lives * Serology result consistent with exposure to HIV, or serology consistent with active hepatitis B or hepatitis C infection
Where this trial is running
Melbourne, Victoria and 2 other locations
- Nucleus Network Pty Ltd — Melbourne, Victoria, Australia (Recruiting)
- New Zealand Clinical Research — Auckland, New Zealand (Not_yet_recruiting)
- New Zealand Clinical Research — Christchurch, New Zealand (Not_yet_recruiting)
Study contacts
- Study coordinator: Study Director
- Email: clinicaltrials@korrobio.com
- Phone: 617-468-1823
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.