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

• Register: ClinicalTrials.gov
• Last refreshed on: 10 July 2023
• Main ID: NCT03533673
• Date of registration: 13/04/2018
• Prospective Registration: Yes
• Primary sponsor: Asklepios Biopharmaceutical, Inc.
• Public title: AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
• Scientific title: A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease
• Date of first enrolment: December 17, 2018
• Target sample size: 7
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03533673
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Biological: ACTUS-101

Primary Outcome(s)

Number of Participants With Abnormal Laboratory Values [Time Frame: 78 weeks]

Incidence of patient reported Treatment Emergent Adverse Events (TEAEs) (safety and tolerability) [Time Frame: 78 weeks]

Incidence of patient reported Serious Adverse Events (SAEs) (safety and tolerability) [Time Frame: 78 weeks]

Secondary Outcome(s)

Muscle GAA Bioactivity [Time Frame: 78 weeks]

Muscle Status Testing - Gross Motor Function Measure [Time Frame: 78 weeks]

Anti-rhGAA antibody formation [Time Frame: 78 weeks]

Muscle Status Testing - 6 minute walk test [Time Frame: 78 weeks]

Muscle Status Testing - Quick Motor Function Test (QMFT) Measure [Time Frame: 78 weeks]

Glycogen content in muscle [Time Frame: 78 weeks]

Muscle Status Testing - Timed up and Go (TUG) [Time Frame: 78 weeks]

Muscle Status Testing - Gait, Stairs, Gower, Chair [Time Frame: 78 weeks]

Pulmonary Function Testing [Time Frame: 78 weeks]

Serum GAA Bioactivity [Time Frame: 78 weeks]

Secondary ID(s)

ACT-CS101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors