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Virus Therapy

ACTUS-101 for Pompe Disease

Phase 1 & 2

Waitlist Available

Led By Edward C Smith, MD

Research Sponsored by Asklepios Biopharmaceutical, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic variants in the GAA gene

FVC within the range of 30% to less 90% (inclusive) of predicted in the upright position

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 78 weeks

Awards & highlights

Study Summary

This trial is testing a new drug, ACTUS-101, to see what effects it has on people. The trial will start with a small group of people, and then give increasing doses of the drug to see what happens.

Who is the study for?

This trial is for adults over 18 with Pompe disease, confirmed by specific tests and genetic markers. Participants must be able to walk at least 100 meters and have been on enzyme replacement therapy (ERT) for two years, with a stable dose in the last year. They should have certain lung function levels but can't join if they've used other investigational drugs recently, started or stopped respiratory training near enrollment, have certain infections or liver disease, are pregnant/nursing, need invasive ventilation, or had a live vaccine recently.Check my eligibility

What is being tested?

The trial is testing ACTUS-101 given through an IV. It's an open-label study where everyone knows what treatment they're getting. The doses will increase to see how much can be given safely. This is for people who already use standard treatments like ERT but still need help managing their condition.See study design

What are the potential side effects?

Since this summary doesn't include specific side effects of ACTUS-101 from the provided information, it's important to note that participants may experience reactions related to intravenous administration and potential unknown effects due to the experimental nature of the drug.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Pompe disease through a blood or skin test.

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My lung function is between 30% and 90% of what is expected.

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I am 18 years old or older.

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I can walk 100 meters or more with or without help like a cane.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 78 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~78 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 78 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of patient reported Serious Adverse Events (SAEs) (safety and tolerability)

Incidence of patient reported Treatment Emergent Adverse Events (TEAEs) (safety and tolerability)

Number of Participants With Abnormal Laboratory Values

Secondary outcome measures

Anti-rhGAA antibody formation

Glycogen content in muscle

Muscle GAA Bioactivity

+7 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort 3Experimental Treatment1 Intervention

A one-time intravenous infusion of ACTUS-101 (dose level 3)

Group II: Cohort 2Experimental Treatment1 Intervention

A one-time intravenous infusion of ACTUS-101 (dose level 2)

Group III: Cohort 1Experimental Treatment1 Intervention

A one-time intravenous infusion of ACTUS-101 (dose level 1)

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Asklepios Biopharmaceutical, Inc.Lead Sponsor

7 Previous Clinical Trials

426 Total Patients Enrolled

Edward C Smith, MDPrincipal InvestigatorDuke University

Media Library

AAV2/8-LSPhGAA (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03533673 — Phase 1 & 2

Pompe Disease Research Study Groups: Cohort 1, Cohort 2, Cohort 3

Pompe Disease Clinical Trial 2023: AAV2/8-LSPhGAA Highlights & Side Effects. Trial Name: NCT03533673 — Phase 1 & 2

AAV2/8-LSPhGAA (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03533673 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is recruitment still open for this experiment?

"Affirmative. Clinicaltrials.gov data indicate that this investigation, first published on December 17th 2018 is actively recruiting individuals. Enrollment requires 13 subjects from 3 distinct medical centres."

Answered by AI

How many participants are involved in this research endeavor?

"The study needs a total of 13 eligible participants. It will be conducted by Asklepios Biopharmaceutical, Inc., in various sites such as Duke University located in Durham, North carolina and the Icahn School of Medicine at Mount Sinai that is based out of New york City."

Answered by AI

What are the primary aspirations of this clinical experiment?

"The primary aim of this long-term trial, which is measuring progress over a 78 week period, is the incidence of patient reported Serious Adverse Events (SAEs) to gauge safety and tolerability. Secondary objectives include Quality of Life Measurements via NIH PROMIS testing, plasma GAA bioactivity evaluation compared to baseline levels, and ELISA administered Anti-GAA antibody monitoring."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

2018. "AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03533673.

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