A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110

Launched by KORRO BIO, INC. · Nov 5, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called KRRO-110 for people with Alpha-1 Antitrypsin Deficiency (AATD), a condition that can lead to lung and liver problems. The main goal of this study is to find out if KRRO-110 is safe and how well it works when given in single and multiple doses. The trial is currently looking for participants, including healthy adults and patients with AATD, aged between 18 and 70 years. To be eligible, healthy volunteers need to have a specific genetic profile called PiMM, while patients with AATD must have a different genetic profile known as PiZZ and a low level of a protein called AAT in their blood.

Participants in this trial can expect to receive either a single dose or multiple doses of KRRO-110 and will be monitored closely for any side effects or reactions. It's important to note that there are some criteria that might exclude potential participants, such as having a history of serious health issues or being pregnant. This trial is a first step in testing KRRO-110 in humans, and everyone involved will contribute to understanding how this treatment might help people with AATD in the future.

Gender

ALL

Eligibility 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 \< 35%
• • 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