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Pompe Gene Therapy- Screening for Eligibility

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03285126
Recruitment Status : Completed
First Posted : September 15, 2017
Last Update Posted : July 2, 2019
Sponsor:
Information provided by (Responsible Party):
Duke University

Study Description
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Brief Summary:

The purpose of this study is to determine eligibility for the future clinical trial of gene therapy in adults with late-onset Pompe disease.

This screening protocol will enroll up to 20 adults patients (>=18 yo) with late-onset Pompe disease. Study assessments include review of medical history, vital signs, physical examination, muscle function testing, lung function testing, blood and urine collection, serum pregnancy test and ECG.

The study results will be collected to perform descriptive statistical analysis and used to determine eligibility for the proposed clinical trial of gene therapy in the future.


Condition or disease
Pompe Disease (Late-onset)

Study Design
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Study Type : Observational
Actual Enrollment : 19 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
Actual Study Start Date : November 1, 2017
Actual Primary Completion Date : June 29, 2019
Actual Study Completion Date : June 29, 2019

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Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Eligibility for gene therapy. [ Time Frame: Up to 2 days ]
    The purpose of this study is to determine eligibility for the proposed clinical trial of gene therapy in adults with late-onset Pompe disease. Eligibility is measure by a composite score of blood tests, Electrocardiogram, Pulmonary Function Tests, and Muscle Status Testing.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adults with late on-set Pompe Disease.
Criteria

Inclusion Criteria

  • Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic variants in the GAA gene.
  • Age: at least 18 years at enrollment.
  • Subjects are capable of giving written consent.
  • Receiving ERT at a stable dose for at least 104 weeks.

Exclusion Criteria

  • Any condition that would interfere with participation in the study as determined by the principal investigator.
  • Pregnancy or nursing mothers.
  • History of active hepatitis B, hepatitis C, or cirrhosis
  • Receiving any investigational agent.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03285126


Locations
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United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27701
Sponsors and Collaborators
Duke University
Investigators
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Principal Investigator: Dwight Koeberl Duke University
More Information
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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT03285126    
Other Study ID Numbers: Pro00085445
First Posted: September 15, 2017    Key Record Dates
Last Update Posted: July 2, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases