A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04093349

Enrollment

Registered

2019-09-18

Start date

2020-10-01

Completion date

2032-04-30

Last updated

2024-11-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acid Maltase Deficiency, Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Pompe Disease, Pompe Disease (Late-onset)

Brief summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

Interventions

GENETICSPK-3006

adeno-associated viral (AAV) vector

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Provide written informed consent; * Males and Females ≥18 years of age with late-onset Pompe disease; * Received ERT for at least the previous 24 months * Have clinically moderate, late-onset Pompe disease characteristics; * Agree to use reliable contraception.

Exclusion criteria

* Active hepatitis B and/or C; * Significant underlying liver disease; * Human immunodeficiency virus (HIV) infection; * Prior hypersensitivity to rhGAA; * Pre-existing anti-AAV neutralizing antibody titers; * High titer antibody responses to rhGAA; * Requires any invasive ventilation or requires noninvasive ventilation while awake and upright; * Received any prior vector or gene transfer agent; * Active malignancy (except non-melanoma skin cancer); * History of liver cancer; * Pregnant or nursing women; * Any evidence of active infection at the time of SPK-3006 infusion.

Design outcomes

Primary

Measure	Time frame	Description
Occurrence of immune response against GAA transgene	Up to 5 years	—
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.	Up to 5 years	Adverse events.
Occurrence of immune response against AAV capsid	Up to 5 years	—

Countries

Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026