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
2 other identifiers
interventional
4
8 countries
29
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Oct 2020
Longer than P75 for phase_1
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 16, 2019
CompletedFirst Posted
Study publicly available on registry
September 18, 2019
CompletedStudy Start
First participant enrolled
October 1, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2032
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 1, 2032
November 27, 2024
November 1, 2024
11.5 years
September 16, 2019
November 25, 2024
Conditions
Outcome Measures
Primary Outcomes (3)
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Adverse events.
Up to 5 years
Occurrence of immune response against AAV capsid
Up to 5 years
Occurrence of immune response against GAA transgene
Up to 5 years
Study Arms (1)
SPK-3006
EXPERIMENTALAll participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
University of California Irvine Health
Orange, California, 92868, United States
Emory University School of Medicine
Atlanta, Georgia, 30329, United States
University of Kansas Medical Center Research Institute
Kansas City, Kansas, 66160, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
University of Utah
Salt Lake City, Utah, 84108, United States
Lysosomal and Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030, United States
Vancouver General Hospital
Vancouver, British Columbia, V5Z 1M9, Canada
The Ottawa Hospital
Ottawa, Ontario, K1Y 4E9, Canada
Montreal Neurological Hospital
Montreal, Quebec, H3A 2B4, Canada
Rigshospitalet
Copenhagen, 84 2100, Denmark
Centre Hospitalier Universitaire d'Angers
Angers, 49933, France
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Garches, 92380, France
Centre Hospitalier Régional Universitaire de Lille
Lille, 59037, France
Assistance Publique Hôpitaux de Marseille
Marseille, 13 13385, France
Nice University Hospital
Nice, 6000, France
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
München, D08033, Germany
Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
Messina, 98122, Italy
Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
Milan, 35 20122, Italy
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
Naples, 80138, Italy
UO Neurologia Azienda Ospedaliera Universitaria Pisana
Pisa, 56126, Italy
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
Torino, 10126, Italy
Erasmus Medical Center
Rotterdam, 3015 CE, Netherlands
New Queen Elizabeth Hospital Birmingham
Birmingham, GBR, B15 2WB, United Kingdom
The Royal Free London NHS Foundation Trust
London, GBR, NW3 2QG, United Kingdom
Salford Royal MHS Foundation Trust
Salford, M6 8HD, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Tahseen Mozaffar, MD
University of California Irvine Health
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 16, 2019
First Posted
September 18, 2019
Study Start
October 1, 2020
Primary Completion (Estimated)
April 1, 2032
Study Completion (Estimated)
April 1, 2032
Last Updated
November 27, 2024
Record last verified: 2024-11
Data Sharing
- IPD Sharing
- Will not share