ABX1100 Safety and Tolerability for Healthy Subjects

Recruiting at 6 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, ABX1100, to evaluate its safety and how the body processes it. The study includes healthy volunteers and individuals with Late-Onset Pompe Disease (LOPD), a muscle condition. Researchers will examine how different doses of ABX1100 affect participants over time. Healthy individuals who do not smoke and have no major health issues can join the initial phases, while those with LOPD who can walk 50 meters without a wheelchair are needed for the final phase. As an Early Phase 1 trial, participants will be among the first to receive this new treatment, contributing to foundational research on its safety and bodily processing.

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.

Is there any evidence suggesting that ABX1100 is likely to be safe for humans?

A previous study found ABX1100 to be safe for people. Researchers examined both healthy volunteers and patients with Late-Onset Pompe Disease (LOPD) to assess the medication's safety. The results were promising. Participants handled the treatment well, with no major side effects reported. ABX1100 is a new treatment designed to reduce harmful glycogen buildup in muscles, a problem for those with Pompe Disease. Although this trial is in its early stages, the safety evidence remains limited but encouraging. Those interested in joining might feel reassured knowing that early tests suggest the treatment is well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about ABX1100 because it offers a fresh approach for treating Late Onset Pompe Disease. Unlike current enzyme replacement therapies, ABX1100 is designed to be administered intravenously in a unique dosing schedule, potentially enhancing its effectiveness with fewer doses. Its novel mechanism of action may provide more efficient targeting of the underlying causes of the disease, offering hope for improved outcomes. This could be a game-changer for patients, reducing treatment frequency and possibly increasing quality of life.

What evidence suggests that ABX1100 might be an effective treatment for Late-Onset Pompe Disease?

Research shows that ABX1100 is a new treatment aimed at reducing harmful glycogen buildup in muscles, affecting people with Pompe disease. Early studies suggest it targets GYS1, an enzyme linked to this buildup, and may help ease symptoms of the disease. Although limited data from human trials exist, this approach appears promising. Scientists believe that by lowering glycogen levels, ABX1100 might improve muscle function and quality of life for patients. More studies, including this trial, are underway to confirm these effects. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. pompediseasenews.com pompediseasenews.com/news/enrollment-complete-phase-1b-trial-abx1100-lopd/

Enrollment complete in Phase 1b trial of ABX1100 for LOPD This part of the trial (NCT06109948) investigates the medication's safety, tolerability, and pharmacological profile in adults with LOPD.

2. arobiotx.com arobiotx.com/aro-bio-announces-start-of-first-in-human-study-with-abx1100

Aro Biotherapeutics Announces Start of First in Human ... ABX1100 is a first-in-class molecule representing a new approach to reducing toxic levels of glycogen that build up in muscles of patients with Pompe Disease.

3. withpower.com withpower.com/trial/phase-1-healthy-subjects-hs-9-2023-25569

ABX1100 Safety and Tolerability for Healthy Subjects 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 ...

4. biospace.com biospace.com/press-releases/aro-biotherapeutics-completes-enrollment-in-phase-1b-trial-of-targeted-sirna-therapy-abx1100-in-late-onset-pompe-disease-lopd

Aro Biotherapeutics Completes Enrollment in Phase 1b ... The trial is evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of ABX1100 in patients with LOPD who are currently ...

5. ucihealth.org ucihealth.org/clinical-trials/ASCC-24-23

A Study to Assess ABX1100 in Normal Healthy Volunteers and ... ... Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABX1100 in Normal Healthy Volunteers and Late-onset Pompe Disease Patients. A Study On: Pompe ...

6. arobiotx.com arobiotx.com/copy-of-aro-biotherapeutics-appoints

Aro Biotherapeutics Raises $13 Million and Establishes ... ... ABX1100, a novel treatment for late-onset Pompe disease (LOPD). ​. “Trial aims to establish safety and provide proof of concept for GYS1 inhibition as ...

7. clinicaltrials.ucbraid.org clinicaltrials.ucbraid.org/trial/NCT06109948

Study to Assess the Safety, Tolerability, PK and PD of ABX1100 Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending ...

8. checkrare.com checkrare.com/new-clinical-trial-for-late-onset-pompe-disease-begins/

New Clinical Trial For Late Onset Pompe Disease Begins A phase 1b trial of ABX1100 has been initiated in patients with late-onset Pompe disease, testing the safety and bioactivity of the treatment.