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Trial record 66 of 103 for:    Pompe Disease

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

Tracking Information
First Submitted Date September 13, 2017
First Posted Date September 15, 2017
Last Update Posted Date July 2, 2019
Actual Study Start Date November 1, 2017
Actual Primary Completion Date June 29, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 13, 2017)
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.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03285126 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 Pompe Gene Therapy- Screening for Eligibility
Official Title Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
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.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Adults with late on-set Pompe Disease.
Condition Pompe Disease (Late-onset)
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 Completed
Actual Enrollment
 (submitted: July 1, 2019)
19
Original Estimated Enrollment
 (submitted: September 13, 2017)
20
Actual Study Completion Date June 29, 2019
Actual Primary Completion Date June 29, 2019   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03285126
Other Study ID Numbers Pro00085445
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Duke University
Study Sponsor Duke University
Collaborators Not Provided
Investigators
Principal Investigator: Dwight Koeberl Duke University
PRS Account Duke University
Verification Date June 2019