NCT06109948

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

A Phase 1a/1b, Single Ascending Dose and Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABX1100 in Normal Healthy Volunteers and Late-onset Pompe Disease Patients

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 46 (estimated)
• Sponsor: Aro Biotherapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose (SAD) and multiple doses (MD) of ABX1100 administered intravenously to healthy participants and patients with LOPD. * Part A features a SAD study with a double-blind, placebo-controlled, randomized design in NHVs involving 3 cohorts (A1-A3). This Part also includes a single dose, open-labeled cohort (A4) in NHVs which will commence after cohorts A1-3. * Part B is a MD, double-blind, placebo-controlled, randomized design in NHVs. The MD Part B will commence after completion of Cohorts A1, A2 and A3 in the SAD Part A and SRC review of these 3 cohorts. * Part C opened for enrollment after the Safety Review Committee (SRC) review of safety, PK and PD data from both Part A and Part B. Part C is a MD, open-label design in patients with Late-Onset Pompe Disease.

Conditions

• Healthy
• Late Onset Pompe Disease

Interventions

Type	Name	Description
DRUG	ABX1100 injection for IV infusion	Centyrin protein-siRNA conjugate
DRUG	Placebo injection for IV infusion	placebo saline injection

Timeline

• Start date: 2023-10-19
• Primary completion: 2026-02-01
• Completion: 2026-02-01
• First posted: 2023-10-31
• Last updated: 2025-12-26

Locations

5 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study