Study to Assess the Safety, Tolerability, PK and PD of ABX1100
Completed · Early Phase 1 · Has a placebo group
Conditions studied: Healthy, Late Onset Pompe Disease
In brief
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.
Key facts
- Study ID
- NCT06109948
- Run by
- Aro Biotherapeutics
- People needed
- 46
- Starts
- 2023-10-19
- Expected to finish
- 2026-02-13
- Last updated by the study team
- 2026-05-07
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
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