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

NCT04093349 · tracked via the Priya Life Science USA tracker

Sponsor

Spark Therapeutics, Inc.

Phase

Phase 1/2

Started

2020-10-01

Last updated

2024-11-27

Condition(s) studied

Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesAcid Maltase Deficiency

Investigational drug(s) / intervention(s)

SPK-3006

SPK-3006: adeno-associated viral (AAV) vector

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

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.

Primary outcome measure(s)

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
Occurrence of immune response against GAA transgene — Up to 5 years

Trial sites (29)

Facility	City	Region
Barrow Neurological Institute	Phoenix	Arizona
University of California Irvine Health	Orange	California
Emory University School of Medicine	Atlanta	Georgia
University of Kansas Medical Center Research Institute	Kansas City	Kansas
University of Minnesota	Minneapolis	Minnesota
Oregon Health & Science University	Portland	Oregon
University of Pennsylvania	Philadelphia	Pennsylvania
University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
University of Utah	Salt Lake City	Utah
Lysosomal and Rare Disorders Research & Treatment Center	Fairfax	Virginia
Vancouver General Hospital	Vancouver	British Columbia
The Ottawa Hospital	Ottawa	Ontario
Montreal Neurological Hospital	Montreal	Quebec
Rigshospitalet	Copenhagen	Denmark
Centre Hospitalier Universitaire d'Angers	Angers	France
CHU Paris IdF Ouest - Hôpital Raymond Poincaré	Garches	France
Centre Hospitalier Régional Universitaire de Lille	Lille	France
Assistance Publique Hôpitaux de Marseille	Marseille	France
Nice University Hospital	Nice	France
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München	München	Germany
Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale	Messina	Italy
Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari	Milan	Italy
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II	Naples	Italy
UO Neurologia Azienda Ospedaliera Universitaria Pisana	Pisa	Italy
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette	Torino	Italy
Erasmus Medical Center	Rotterdam	Netherlands
New Queen Elizabeth Hospital Birmingham	Birmingham	GBR
The Royal Free London NHS Foundation Trust	London	GBR
Salford Royal MHS Foundation Trust	Salford	United Kingdom

More Spark Therapeutics, Inc. trials in the USA

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Active, not recruiting

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Active, not recruiting

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A Active, not recruiting

Other trials for the same condition

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease Active, not recruiting

Pompe Disease Registry Protocol Recruiting

Avalglucosidase Alfa Pregnancy Study Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04093349 on ClinicalTrials.gov ↗ ← All trials in the USA