Here are some experiences of patients and parents about participating in an enzyme replacement therapy trial.
You can find more general information about clinical trials at www.clinicaltrials.gov and www.clinicaltrialsregister.eu.
Selected Pompe related clinical trials
| Name and Link to clinicaltrials.gov | Duration | Information |
|
ClinicalTrials.gov Identifier: NCT01230801 |
12/2010-12/2012 | A Phase I, multicenter, open-label, dose-escalation study designed to evaluate the safety and pharmacokinetics of administration of ZC-701 (other name: BMN-701, GILT-tagged recombinant human GAA) by intravenous infusion every two weeks over a 12-week treatment period to subjects with late-onset Pompe disease.
Sponsor: BioMarin Pharmaceutical |
|
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease ClinicalTrials.gov Identifier: NCT00976352 |
09/2010-01/2013 |
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. Sponsor: University of Florida |
|
Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis (POPS) ClinicalTrials.gov Identifier: NCT00830583 |
01/2009-02/2011 |
An international consensual group recommends confirming the diagnosis of the Pompe disease after a dried blood spot (DBS) with a dosage of the enzymatic activity in other tissue. This strategy is currently used in the usual practice. The aim is evaluate the prevalence of the Pompe disease among patients with progressive limb girdle muscular weakness and/or axial deficiency, and/or respiratory insufficiency. The diagnosis will be confirmed using DBS. Sponsor: Centre Hospitalier Universitaire de Nice |
|
Immune Tolerance Induction Study ClinicalTrials.gov Identifier: NCT00701701 |
06/2008-06/2013 |
An exploratory, open-labeled study of patients with Pompe disease, who have previously received Myozyme treatment, to evaluate the efficacy, safety and clinical benefit of 2 ITI regimens in combination with Myozyme. Sponsor: Genzyme |
|
A Long Term Follow up Study in Late-onset Pompe Disease ClinicalTrials.gov Identifier: NCT00713245 |
05/2008-12/2018 |
This study is to observe the progression of disease in late-onset Pompe disease. Sponsor: National Taiwan University Hospital |
|
Growth and Development Study of Myozyme ClinicalTrials.gov Identifier: NCT00486889 |
12/2007-09/2020 |
The overall objective of this study is to evaluate the long-term growth and development of patients with infantile-onset Pompe disease with Myozyme before 1 year of age. Patients will be followed for 10-year period. Sponsor: Genzyme |
|
Late-Onset Treatment Study Extension Protocol ClinicalTrials.gov Identifier: NCT00455195 |
03/2007-11-2008 |
The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704. Sponsor: Genzyme |
|
ClinicalTrials.gov Identifier: NCT00158600 |
09/2005-09/2007 | The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo. Click here for results (clinicaltrials.gov) Click here for related publication (pubmed.gov) Sponsor: Genzyme |
| A Study of rhGAA in Patients With Late-Onset Pompe Disease
ClinicalTrials.gov Identifier: NCT00250939 |
02/2005-11/2006 |
The overall objective is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment. Sponsor: Genzyme |
|
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease ClinicalTrials.gov Identifier: NCT00059280 |
04/2003-09/2005 |
This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied. Sponsor: Genzyme |
|
rhGAA in Patients With Infantile-Onset Glycogen Storage Disease-II (Pompe Disease) ClinicalTrials.gov Identifier: NCT00053573 |
02/2003-11/2006 | This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.
Sponsor: Genzyme |
|
ClinicalTrials.gov Identifier: NCT00051935 |
01/2003-10/2003 |
This study is being conducted to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II. Sponsor: Genzyme |
|
ClinicalTrials.gov Identifier: NCT00025896 |
05/2001-11/2002 |
Patients diagnosed with Classical Infantile Pompe disease who have a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material-Positive or “CRIM (+)” patients), will be studied. Sponsor: Genzyme |
|
ClinicalTrials.gov Identifier: NCT00025896 |
05/2001-11/2002 | This study being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with Classical Infantile Pompe disease who have a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material-Positive or “CRIM (+)” patients), will be studied.
Sponsor: Genzyme |
Source: www.clinicaltrials.gov.
The above list may be outdated. Please check www.clinicaltrials.gov and www.clinicaltrialsregister.eu for up-to-date information.