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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

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamis (PD) Study of ZC-701 in Patients With Late-Onset Pompe Disease

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
Collaborator: ZyStor Therapeutics, Inc.

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
Collaborator: National Heart, Lung, and Blood Institute (NHLBI)

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

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease ("LOTS")

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

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

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.
This is the famous study with John Crowley's (founder of Novazyme) children enrolled. See also the 2010 movie "Extraordinary Measures" starring Harrison Ford and Brendan Fraser.

Sponsor: Genzyme

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

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

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

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.


(Last Updated Sunday, 17 July 2011 10:59)

 
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