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

NCT06109948 | Completed Early Phase 1 DMC FDA Drug

• 1 other identifier: ABX1100-1001
• Study Type: interventional
• Target: 46
• Locations: 2 countries
• Sites: 5

Brief 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

Keywords

pompe; GYS1

Outcome Measures

Primary Outcomes (1)

• Number of subjects with treatment-emergent adverse events (TEAEs)

  Adverse Events will be graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) will be reported based on clinical laboratory tests, vital signs, physical examinations, electrocardiograms, and echocardiograms from the time informed consent is signed through 8 weeks after ABX1100 administration for Part A and through 12 weeks after ABX1100 administration for Part B, and up to 20 weeks for Part C. AEs will be considered to be treatment emergent (TEAE) if they occurred or worsened in severity after the first dose of study treatment.

  up to 20 weeks

Secondary Outcomes (6)

• Plasma pharmacokinetics as measured by Cmax

  0-8 hours after ABX1100 administration

• Plasma pharmacokinetics as measured by Tmax

  0-8 hours after ABX1100 administration

• Plasma pharmacokinetics as measured by AUC

  0-8 hours after ABX1100 administration

• Immunogenicity of AXB1100 as measured by anti-ABX1100 antibodies in serum

  Up to 16 weeks

• Muscle drug concentration

  Needle muscle biopsies at pre-dose, week 10 and week 6 or 16

Study Arms (5)

Part A Cohort 1-4 Single Dose (active)

EXPERIMENTAL

Subjects will receive 1 single IV dose of ABX1100 on Day 1.

Drug: ABX1100 injection for IV infusion

Part A Cohort 1-3 Single Dose (placebo)

PLACEBO COMPARATOR

Subjects will receive 1 single IV dose of placebo on Day 1.

Drug: Placebo injection for IV infusion

Part B Multi-dose (active)

EXPERIMENTAL

Subjects will receive 1 IV dose of ABX1100 on Day 1 and Day 29 each.

Drug: ABX1100 injection for IV infusion

Part B Multi-dose (placebo)

PLACEBO COMPARATOR

Subjects will receive 1 IV dose of placebo on Day 1 and Day 29 each.

Drug: Placebo injection for IV infusion

Part C

EXPERIMENTAL

Late onset Pompe Disease patients will receive 1 dose of ABX1100 at Day 1 and Day 29 respectively.

Drug: ABX1100 injection for IV infusion

Interventions

ABX1100 injection for IV infusion DRUG

Centyrin protein-siRNA conjugate

Part A Cohort 1-4 Single Dose (active); Part B Multi-dose (active); Part C

Placebo injection for IV infusion DRUG

placebo saline injection

Part A Cohort 1-3 Single Dose (placebo); Part B Multi-dose (placebo)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.
• 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.
• 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 or after the final study procedure, whichever is later.
• 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.
• Documented Acid alpha-glucosidase (GAA) deficiency and mutation analysis from blood, skin, or muscle tissue for confirmation of diagnosis of LOPD.

Sponsors & Collaborators

• Aro Biotherapeutics: lead

Study Sites (5)

UCI

Orange, California, 92868, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Lysosomal and Rare Disorders Research and Treatment Center, Inc

Fairfax, Virginia, 22030, United States

Location

MAGIC clinic

Calgary, Alberta, Canada

Location

McMaster University

Hamilton, Ontario, Canada

Location

MeSH Terms

Interventions

Infusions, Intravenous

Intervention Hierarchy (Ancestors)

Administration, Intravenous; Drug Administration Routes; Drug Therapy; Therapeutics; Infusions, Parenteral

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 19, 2023
• First Posted: October 31, 2023
• Study Start: October 19, 2023
• Primary Completion: February 13, 2026
• Study Completion: February 13, 2026
• Last Updated: May 7, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2); US (3)