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Study to Assess the Safety, Tolerability, PK and PD of ABX1100 | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGEARLY_PHASE1

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

NCT06109948•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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Body mass index (BMI) between 18.0 and 32.0 kg/m2, inclusive and weight between 50 and 90 kg, inclusive.
•Agree not to have a tattoo or body piercing until the end of the study.
•Agree not to receive COVID-19 vaccination from 7 days prior to first study drug administration until at least 7 days after the last study drug administration.
•Agree not to receive a vaccination (live attenuated vaccine) during the study and until 60 days after the study has ended (last study procedure).
•Willing to undergo needle muscle biopsies.
•Willing to avoid strenuous activities 48 hours before needle muscle biopsy and throughout the study.
•Female participants who are sexually active with a non-sterilized partner must be non-pregnant and non-lactating and agree to use a highly effective method of contraception.
•Males of childbearing potential must agree to use a highly effective method of contraception with female sexual partners of childbearing potential and not donate sperm during study participation and for 90 days after last administration of study drug ABX1100 or placebo.
•Male or female LOPD patients aged ≥ 18 years (inclusive) of age, at the time of informed consent.
•Body mass index (BMI) between 18.0 and 35.0 kg/m2, inclusive and weight \>50 kg.
+9 more criteria

Exclusion Criteria

•Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological or hematological disease or condition.
•History of any inherited or acquired skeletal muscle diseases (for example, Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), Limb-Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy, Spinal Muscular Atrophy (SMA), Polymyositis, Rhabdomyolysis and Inclusion Body Myositis (IBM).
•History of any inherited or acquired cardiac disease including congestive heart failure, ischemic heart disease, or arrhythmias; an abnormal ECG.
•History of cancer within past 5 years, with the exception of treated or excised skin basal cell carcinoma.
•Any clinically significant illness, medical/surgical procedure, or trauma within 4 weeks prior to the screening visit
•Presence of any significant physical or organ abnormality.
•Major surgery within 6 months prior to the start of the study.
•Current smoker, recent history of smoking and/or use of any nicotine-containing products (within past 6 months).
•A known history or positive test result for hepatitis B surface antigen (HBsAg), hepatitis C antibody or human immunodeficiency virus (HIV) infection.
•Currently participating in another investigational trial or have received any investigational drug within the past 30 days.
+13 more criteria

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

Key Dates

Start Date

October 19, 2023

Primary Completion Date

February 1, 2026

Completion Date

February 1, 2026

Last Updated

December 26, 2025

Enrollment

ESTIMATED participants

Conditions

HealthyLate Onset Pompe Disease

Interventions

ABX1100 injection for IV infusion

DRUG

Placebo injection for IV infusion

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 26, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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