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

NCT ID: NCT03533673

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-17

Study Completion Date

2026-03-31

Brief Summary

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Open-label, ascending dose trial of ACTUS-101 administered intravenously.

Detailed Description

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This study will be a prospective, open-label trial designed to objectively assess the safety and bioactivity of ACTUS-101 in subjects diagnosed with Pompe disease, which is caused by a defect in acid α-glucosidase (GAA) gene. ACTUS-101 is intended to enable expression of a functional copy of the GAA gene in subject's hepatocytes.

Conditions

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Pompe Disease

Keywords

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Gene Therapy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

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

Group Type EXPERIMENTAL

ACTUS-101

Intervention Type BIOLOGICAL

Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Cohort 2

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

Group Type EXPERIMENTAL

ACTUS-101

Intervention Type BIOLOGICAL

Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Cohort 3

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

Group Type EXPERIMENTAL

ACTUS-101

Intervention Type BIOLOGICAL

Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Interventions

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ACTUS-101

Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic variants in the GAA gene,
* Age: Greater than or equal to 18 years at enrollment.
* Subjects are capable of giving written informed consent.
* Able to walk at least 100 meters on the 6MWT (with assistive devices permitted).
* FVC within the range of 30% to less 90% (inclusive) of predicted in the upright position.
* Subjects with a confirmed diagnosis of LOPD who have been treated with ERT for at least 104 weeks (inclusive) immediately preceding screening and receiving a stable dose of ERT for the 52-week period immediately preceding dosing.

Exclusion Criteria

* Invasive ventilation required or noninvasive ventilation required while awake and upright.
* FVC \<20% of predicted (supine).
* Received any live vaccination 2 months prior to study Day 1.
* Pregnant or nursing mothers.
* Serology consistent with exposure to HIV, or serology consistent with active hepatitis A, B or C infection. Any active liver disease.
* Active infection based upon clinical symptoms.
* Having started respiratory muscle strength training in the last 6 months prior to study day 1 or having discontinued respiratory muscle strength training in the 6-month period preceding study day 1, or having started respiratory strength training greater than 6 months prior to study day 1 and unwilling to continue for the first year of study participation.
* Received an investigational drug or participated in another interventional study within 90 days prior to Study Day 1. Additionally, subjects cannot participate in any other interventional clinical trial throughout the first 78 weeks after receiving ACTUS-101.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AskBio Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Duke University

Durham, North Carolina, United States

Site Status

Countries

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United States

References

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Smith EC, Hopkins S, Case LE, Xu M, Walters C, Dearmey S, Han SO, Spears TG, Chichester JA, Bossen EH, Hornik CP, Cohen JL, Bali D, Kishnani PS, Koeberl DD. Phase I study of liver depot gene therapy in late-onset Pompe disease. Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18.

Reference Type DERIVED
PMID: 36805083 (View on PubMed)

Other Identifiers

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ACT-CS101

Identifier Type: -

Identifier Source: org_study_id