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Pompe Disease clinical trials

View clinical trials related to Pompe Disease.

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NCT ID: NCT01451879 Completed - Pompe Disease Clinical Trials

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Start date: October 2008
Phase: N/A
Study type: Observational

Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme. Treatment with immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.

NCT ID: NCT01435772 Terminated - Pompe Disease Clinical Trials

Extension Study for Patients Who Have Participated in a BMN 701 Study

Start date: August 15, 2011
Phase: Phase 2
Study type: Interventional

This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.

NCT ID: NCT01410890 Completed - Pompe Disease Clinical Trials

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

PAPAYA
Start date: November 3, 2014
Phase: Phase 4
Study type: Interventional

- The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease. - A secondary objective of this study was to evaluate and explore the relationship between anti-recombinant human acid alpha-glucosidase antibody titers and the PK of alglucosidase alfa.

NCT ID: NCT01409486 Not yet recruiting - Pompe Disease Clinical Trials

Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry

LC-MS-MS
Start date: September 2011
Phase: N/A
Study type: Observational

The aim of the study is: to develop a comprehensive biochemical assay for detection of Pompe disease (glycogen storage disease type II), to be implemented in the Newborn screening program among the Israeli population.

NCT ID: NCT01380743 Completed - Pompe Disease Clinical Trials

Drug-drug Interaction Study

Start date: October 31, 2011
Phase: Phase 2
Study type: Interventional

This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.

NCT ID: NCT01230801 Completed - Pompe Disease Clinical Trials

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Start date: January 17, 2011
Phase: Phase 1/Phase 2
Study type: Interventional

A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.

NCT ID: NCT00976352 Completed - Pompe Disease Clinical Trials

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Start date: September 2010
Phase: Phase 1/Phase 2
Study type: Interventional

Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and profound muscle weakness. A wide spectrum of disease is characteristic and the most severe patients have cardiorespiratory failure, often fatal in the first two years of life. Researchers have developed a way to introduce the normal GAA gene into muscle cells with the expectation that the GAA protein will be produced at levels sufficient to reduce glycogen accumulation. This study will evaluate the safety of the experimental gene transfer procedure in individuals with GAA deficiency. The study will also determine what dose may be required to achieve improvement in measures of respiratory function.

NCT ID: NCT00713453 Recruiting - Pompe Disease Clinical Trials

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Start date: January 2007
Phase: N/A
Study type: Observational

This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy

NCT ID: NCT00713245 Recruiting - Pompe Disease Clinical Trials

A Long Term Follow up Study in Late-onset Pompe Disease

Start date: May 2008
Phase: N/A
Study type: Observational

This study is to observe the progression of disease in late-onset Pompe disease

NCT ID: NCT00701701 Terminated - Pompe Disease Clinical Trials

Immune Tolerance Induction Study

Start date: December 14, 2008
Phase: Phase 4
Study type: Interventional

An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who were then receiving Myozyme® therapy were enrolled into the study, and were followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme® alone). Eligible participants were followed for a minimum of 18 months on treatment or, if a participant was <6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.