A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT ID: NCT04093349
Last Updated: 2024-11-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
4 participants
INTERVENTIONAL
2020-10-01
2032-04-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
NCT03893240
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT01924845
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
NCT00268944
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
NCT06391736
VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease
NCT02898753
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
SPK-3006
adeno-associated viral (AAV) vector
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
SPK-3006
adeno-associated viral (AAV) vector
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Spark Therapeutics, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Tahseen Mozaffar, MD
Role: PRINCIPAL_INVESTIGATOR
University of California Irvine Health
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California Irvine Health
Orange, California, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Kansas Medical Center Research Institute
Kansas City, Kansas, United States
University of Minnesota
Minneapolis, Minnesota, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
University of Utah
Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States
Vancouver General Hospital
Vancouver, British Columbia, Canada
The Ottawa Hospital
Ottawa, Ontario, Canada
Montreal Neurological Hospital
Montreal, Quebec, Canada
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, United Kingdom
The Royal Free London NHS Foundation Trust
London, GBR, United Kingdom
Salford Royal MHS Foundation Trust
Salford, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2019-001283-30
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SPK-3006-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.