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

Pompe Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04093349
• Other study ID #: SPK-3006-101
• Secondary ID: 2019-001283-30
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: October 1, 2020
• Est. completion date: April 2032

Study information

• Verified date: October 2023
• Source: Spark Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 4
• Est. completion date: April 2032
• Est. primary completion date: April 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
Canada	Montreal Neurological Hospital	Montréal	Quebec
Canada	The Ottawa Hospital	Ottawa	Ontario
Canada	Vancouver General Hospital	Vancouver	British Columbia
Denmark	Rigshospitalet	Copenhagen
France	Centre Hospitalier Universitaire d'Angers	Angers
France	CHU Paris IdF Ouest - Hôpital Raymond Poincaré	Garches
France	Centre Hospitalier Régional Universitaire de Lille	Lille Cedex
France	Assistance Publique Hôpitaux de Marseille	Marseille
France	Nice University Hospital	Nice
Germany	Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München	Munchen
Italy	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	Milano
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
Netherlands	Erasmus Medical Center	Rotterdam
United Kingdom	New Queen Elizabeth Hospital Birmingham	Birmingham	GBR
United Kingdom	The Royal Free London NHS Foundation Trust	London	GBR
United Kingdom	Salford Royal MHS Foundation Trust	Salford
United States	Emory University School of Medicine	Atlanta	Georgia
United States	Lysosomal and Rare Disorders Research & Treatment Center	Fairfax	Virginia
United States	University of Kansas Medical Center Research Institute	Kansas City	Kansas
United States	University of Minnesota	Minneapolis	Minnesota
United States	University of California Irvine Health	Orange	California
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Barrow Neurological Institute	Phoenix	Arizona
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	University of Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Spark Therapeutics

Countries where clinical trial is conducted

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

Outcome

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

External Links

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