Pompe Disease Clinical Trial
Official title:
A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Verified date | March 2023 |
Source | Spark Therapeutics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.
Status | Completed |
Enrollment | 61 |
Est. completion date | October 27, 2020 |
Est. primary completion date | October 27, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations - Male or females =18 years of age - Currently on ERT using regular recombinant human GAA infusions for at least 18 months prior to screening - Documented history of clinically moderate late-onset Pompe disease. Exclusion Criteria: - History of HIV infection - Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright - Previously received SPK-3006 - Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks (vaccination studies are accepted) - Any concurrent clinically significant condition that would not allow the potential participant to complete the Day 1 examinations, or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study - Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol. |
Country | Name | City | State |
---|---|---|---|
France | Hôpital Raymond-Poincaré | Garches | Hauts-de-Seine |
France | Assistance Publique Hôpitaux de Marseille | Marseille | |
France | CHU Nice | Nice | |
Germany | Klinikum der Universität München | München | |
Italy | Università degli Studi di Messina | Messina | |
Italy | Universita degli Studi di Milano - Clinica Oculistica I | Milano | |
Italy | Università degli Studi di Napoli Federico II | Napoli | |
Italy | Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico | Pavia | |
Italy | Azienda Ospedaliero Universitaria Pisana | Pisa | |
Italy | Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino | Torino | |
Netherlands | Erasmus University Medical Center | Rotterdam | |
United Kingdom | Salford Royal NHS Foundation Trust | Salford | |
United States | University of Kansas Medical Center | Kansas City | Kansas |
United States | University of Minnesota Medical School | Minneapolis | Minnesota |
United States | University of California Irvine Health | Orange | California |
United States | Barrow Neurological Institute | Phoenix | Arizona |
United States | University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
Lead Sponsor | Collaborator |
---|---|
Spark Therapeutics |
United States, France, Germany, Italy, Netherlands, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Neutralizing Antibodies Titer to SPK-3006 capsid | The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit. | 1 day | |
Primary | Occurrence of Neutralizing Antibodies to SPK-3006 capsid | The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid. | 1 day | |
Secondary | Anti-GAA binding antibodies Titer | Anti-GAA binding antibodies titer is measured once prospectively at one site visit. | 1 day | |
Secondary | Occurrence of Anti-GAA binding antibodies across participants | The proportion of participants who have Anti-GAA binding antibodies. | 1 day | |
Secondary | Neutralizing antibodies to circulating GAA Titer | Neutralizing antibodies to circulating GAA titer is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent. | 1 day | |
Secondary | Occurrence of Neutralizing antibodies to circulating GAA | The proportion of participants who have neutralizing antibodies to circulating GAA. | 1 day | |
Secondary | GAA activity level | GAA activity level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent. | 1 day | |
Secondary | GAA antigen level | GAA antigen level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent. | 1 day |
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
|
||
Active, not recruiting |
NCT04093349 -
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
|
Phase 1/Phase 2 | |
Completed |
NCT02363153 -
Diet and Exercise in Pompe Disease
|
N/A |