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Muscle Response to Enzyme Replacement Therapy in Pompe Disease

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ClinicalTrials.gov Identifier: NCT00713453
Recruitment Status : Recruiting
First Posted : July 11, 2008
Last Update Posted : March 24, 2015
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

Tracking Information
First Submitted Date July 9, 2008
First Posted Date July 11, 2008
Last Update Posted Date March 24, 2015
Study Start Date January 2007
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 21, 2015)
all morphology changes improvement [ Time Frame: 6 months ]
Original Primary Outcome Measures Not Provided
Change History Complete list of historical versions of study NCT00713453 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Muscle Response to Enzyme Replacement Therapy in Pompe Disease
Official Title Muscle Response to Enzyme Replacement Therapy in Pompe Disease
Brief Summary This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population patients with Pompe disease, prepare or on enzyme replacement therapy
Condition Pompe Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 9, 2008)
16
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • patients with Pompe disease, prepare or on enzyme replacement therapy
  • signed informed consent

Exclusion Criteria:

-

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers Yes
Contacts
Contact: Yin-Hsiu Chien, MD 886-2-23123456 ext 71937 chienyh@ntu.edu.tw
Listed Location Countries Taiwan
Removed Location Countries  
 
Administrative Information
NCT Number NCT00713453
Other Study ID Numbers 200704066R
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Taiwan University Hospital
Study Sponsor National Taiwan University Hospital
Collaborators Not Provided
Investigators
Principal Investigator: Yin-Hsiu Chien, MD National Taiwan University Hospital
PRS Account National Taiwan University Hospital
Verification Date March 2015