ABX1100 Safety and Tolerability for Healthy Subjects

Recruiting at 3 trial locations

Overseen By Aro Study Information Center

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Aro Biotherapeutics

Must be taking: Enzyme replacement therapy

Disqualifiers: Cardiovascular disease, Cancer, Diabetes, others

Compensation: Varies

Number of Visits: 5

46 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are currently receiving enzyme replacement therapy (ERT) for Late-Onset Pompe Disease, you must be on a stable dose for at least 6 months before joining Part C of the trial.

Eligibility Criteria

Healthy adults with a BMI of 18.0-32.0 and weight between 50-90 kg can join this trial. They must not get tattoos, piercings, or certain vaccinations during the study period and avoid strenuous activities before muscle biopsies. Women must use contraception if applicable, and men agree to do so with partners.

Inclusion Criteria

My BMI is between 18 and 32, and my weight is between 50kg and 90kg.

I agree not to get a COVID-19 vaccine 7 days before and after the study drug.

I am not pregnant or breastfeeding and agree to use effective birth control.

Exclusion Criteria

I have a significant health condition affecting my organs or systems.

I have a history of inherited or acquired muscle diseases.

I have no history of heart disease or abnormal heart tests.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose (SAD)

Participants receive a single ascending dose of ABX1100 or placebo in a double-blind, placebo-controlled, randomized design

1 day

1 visit (in-person)

Multiple Dose (MD) - Part B

Participants receive multiple doses of ABX1100 or placebo in a double-blind, placebo-controlled, randomized design

4 weeks

2 visits (in-person)

Multiple Dose (MD) - Part C

Late-onset Pompe Disease patients receive multiple doses of ABX1100 in an open-label design

4 weeks

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

8-12 weeks

Treatment Details

Interventions

• ABX1100

Trial Overview The trial is testing ABX1100, a new drug given through IV infusion compared to a placebo (a substance with no active drug). It's done in two parts: one where subjects receive a single dose and another where they receive multiple doses to evaluate safety and effects on the body.

Participant Groups

Group I: Part C Experimental Treatment 1 Intervention

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

Group II: Part B Multi-dose (active) Experimental Treatment 1 Intervention

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

Group III: Part A Cohort 1-4 Single Dose (active) Experimental Treatment 1 Intervention

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

Group IV: Part A Cohort 1-3 Single Dose (placebo) Placebo Group 1 Intervention

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

Group V: Part B Multi-dose (placebo) Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Aro Biotherapeutics

Lead Sponsor

Trials

1

Recruited

50+