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NCT03694561 · ClinicalTrials.gov registry record
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
A clinical trial of Pompe Disease and Pompe Disease (Late-onset), sponsored by Duke University.
- Active
- Registry status
- 20
- Enrollment target
- 1
- Study location
NCT03694561 is a study of Pompe Disease and Pompe Disease (Late-onset) that is active but no longer recruiting, run by Duke University. The registered enrollment target is 20 participants, below the 231-participant average among 14 other Pompe Disease trials with a reported enrollment target (91% lower). The trial reports 1 study location across 1 state.
The verdict
NCT03694561, a study of Pompe Disease and Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Duke University.
- ACTIVE NOT RECRUITING
- Registry status
- 20 participants
- Enrollment target
- 1
- Study location
Study Summary
This is an observational study with no study related treatment of interventions. The purpose of the study is to investigate and document disease specific clinical symptoms in newborns, infants and children with Pompe disease without cardiomyopathy identified in newborn screening(NBS). There will be baseline, months 6 and months 12 visits for infants and newborns (infants study). For children of ages 24 months to 54 months, there will be baseline, year 1 and year 2 visits (children study). The study has four goals: 1. To study and record disease specific clinical symptoms in newborns, infants and children with Pompe disease without cardiomyopathy (disease of the heart muscle) in the first year of life identified through newborn screening (NBS) 2. To devise an approach to characterize early musculoskeletal (muscles and joints) involvement in subjects with the "late-onset" GAA variant identified by NBS including ability to collect research information via virtual health platforms. 3. To determine criteria to start preventative therapies including enzyme replacement therapy (ERT) in patients with clinical features of Pompe disease identified via NBS 4. To document parental coping and anxiety/emotional distress overtime using quality of life questionnaires after a child is diagnosed with late onset Pompe disease via NBS
Conditions Studied
Interventions
- OTHER Observational
Study Locations (1)
North Carolina
- Duke University - Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2019-03-25 |
| Est. Completion | 2027-08-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03694561
The ClinicalTrials.gov registry entry for NCT03694561 describes a study currently listed as active not recruiting, categorized as an unspecified phase. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 231-participant average among 14 other Pompe Disease trials with a reported enrollment target (91% lower). The listed sponsor is Duke University, which has 1,725 total studies on file at ClinicalTrials.gov.
The record links to 3 conditions, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention - of which Observational is the first listed.
NCT03694561 reports 1 study location spanning 1 distinct geographic area - top geographies include North Carolina.
Frequently Asked Questions
What is clinical trial NCT03694561 about?
NCT03694561 is a clinical study titled "Developing a Management Approach for Patients With "Late-Onset" Pompe Disease". This is an observational study with no study related treatment of interventions. The purpose of the study is to investigate and document disease specific clinical symptoms in newborns, infants and children with Pompe disease without cardiomyopathy identified in newborn screening(NBS). There will be...
What is the current status of trial NCT03694561?
This trial is currently active not recruiting. The enrollment target is 20 participants. The study started on 2019-03-25. Estimated completion is 2027-08-31.
What conditions does trial NCT03694561 study?
This clinical trial studies the following conditions: Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency.
What interventions are being tested in trial NCT03694561?
The interventions under investigation include: Observational (OTHER).
Who is sponsoring clinical trial NCT03694561?
This trial is sponsored by Duke University, which has 1,725 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03694561 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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