Status:
UNKNOWN
Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Lead Sponsor:
LMU Klinikum
Conditions:
Pompe Disease (Late-onset)
Inclusion Body Myositis
Eligibility:
All Genders
18+ years
Brief Summary
The primary aim of this nationwide, explorative, cross-sectional study in Germany is to characterize the prevalence, severity and quality of musculoskeletal pain in adult patients with late-onset Pomp...
Detailed Description
In this nationwide, explorative, cross-sectional study in Germany, approx. 50 patients with genetically confirmed late-onset Pompe disease (LOPD) will be included into the study. In addition, 15 Patie...
Eligibility Criteria
Inclusion
- A patient must meet the following criteria to be eligible for this study:
- The patient is willing and able to provide signed informed consent.
- The patient is able and willing to perform study-related assessments.
- A) The patient is ≥18 years of age with acid α-glucosidase \[GAA\] enzyme deficiency, confirmed by GAA gene mutation analysis, or B) The patient has a histologically confirmed diagnosis of inclusion body myositis (IBM), or a genetically confirmed spinal muscular atrophy type 3 (SMA3) or a genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD).
Exclusion
- A patient who meets any of the following criteria will be excluded from this study.
- The patient is participating in another clinical study or using an investigational treatment.
- The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
- The patient has currently a severe depression, assessed by the Beck depression inventory fast screen (BDI-FS) with a score ≥ 4
Key Trial Info
Start Date :
March 31 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 3 2024
Estimated Enrollment :
95 Patients enrolled
Trial Details
Trial ID
NCT05272969
Start Date
March 31 2022
End Date
September 3 2024
Last Update
October 10 2023
Active Locations (1)
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1
Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen
Munich, Bavaria, Germany, 80336