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ACTIVE NOT RECRUITING
NCT06109948
EARLY_PHASE1

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

Sponsor: Aro Biotherapeutics

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

46

Start Date

2023-10-19

Completion Date

2026-02

Last Updated

2025-12-26

Healthy Volunteers

No

Interventions

DRUG

ABX1100 injection for IV infusion

Centyrin protein-siRNA conjugate

DRUG

Placebo injection for IV infusion

placebo saline injection

Locations (5)

UCI

Orange, California, United States

Washington University School of Medicine

St Louis, Missouri, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc

Fairfax, Virginia, United States

MAGIC clinic

Calgary, Alberta, Canada

McMaster University

Hamilton, Ontario, Canada