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

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04093349
Study type	Interventional
Source	Spark Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	October 1, 2020
Completion date	April 2032

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT01942590` - Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy	Phase 1/Phase 2
Not yet recruiting	`NCT01409486` - Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry	N/A
Completed	`NCT00976352` - Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease	Phase 1/Phase 2
Recruiting	`NCT01665326` - Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Completed	`NCT01758354` - Newborn Screening Assay of Pompe's Disease	N/A
Recruiting	`NCT00231400` - Pompe Disease Registry Protocol
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Recruiting	`NCT04476550` - Clinical Specimen Collection From Pompe Disease Patients
Completed	`NCT01380743` - Drug-drug Interaction Study	Phase 2
Recruiting	`NCT05734521` - Avalglucosidase Alfa Pregnancy Study
Completed	`NCT02742298` - Pompe Disease QMUS and EIM	N/A
Terminated	`NCT02185651` - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction	Phase 1
Completed	`NCT02654886` - Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.	N/A
Completed	`NCT02405598` - Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease	Phase 4
Completed	`NCT00701129` - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease	Phase 4
Completed	`NCT01451879` - Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies	N/A
Completed	`NCT02240407` - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease	Phase 1
Completed	`NCT05073783` - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Completed	`NCT02363153` - Diet and Exercise in Pompe Disease	N/A
Completed	`NCT01410890` - Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease	Phase 4