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

Spark Therapeutics

Status and phase

Active, not recruiting

Phase 2

Phase 1

Conditions

Pompe Disease (Late-onset)

Pompe Disease

Glycogen Storage Disease Type II

LOPD

Lysosomal Storage Diseases

Glycogen Storage Disease Type 2

Acid Maltase Deficiency

Treatments

Genetic: SPK-3006

Study type

Interventional

Funder types

Industry

Identifiers

NCT04093349

2019-001283-30 (EudraCT Number)

SPK-3006-101

Details and patient eligibility

About

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.

Enrollment

4 patients

Sex

All

Ages

18+ years old

Volunteers

No 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.