Pompe Disease (Late-onset) Clinical Trial
Official title:
A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase alfa or avalglucosidase alfa.
Study Design Time Perspective: Retrospective and Prospective ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00158600 -
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
|
Phase 3 | |
Recruiting |
NCT04174105 -
Gene Transfer Study in Patients With Late Onset Pompe Disease
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT04093349 -
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
|
Phase 1/Phase 2 | |
Completed |
NCT03285126 -
Pompe Gene Therapy- Screening for Eligibility
|
||
Recruiting |
NCT05083806 -
MSOT in Pompe Disease
|
N/A | |
Recruiting |
NCT05272969 -
Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
|
||
Active, not recruiting |
NCT03694561 -
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
|
||
Completed |
NCT00731081 -
Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
|
N/A | |
Completed |
NCT00455195 -
Late-Onset Treatment Study Extension Protocol
|
Phase 4 | |
Recruiting |
NCT05951790 -
Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease
|
N/A | |
Completed |
NCT01288027 -
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
|
Phase 4 | |
Completed |
NCT03893240 -
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
|
N/A | |
Withdrawn |
NCT04094948 -
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
|
Phase 2 | |
Completed |
NCT00250939 -
A Study of rhGAA in Patients With Late-Onset Pompe Disease
|
Phase 2 | |
Recruiting |
NCT03911505 -
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
|
Phase 3 | |
Recruiting |
NCT06150820 -
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
|
N/A | |
Completed |
NCT03729362 -
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
|
Phase 3 | |
Not yet recruiting |
NCT06178432 -
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
|
Early Phase 1 | |
Completed |
NCT00268944 -
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
|
Phase 3 | |
Active, not recruiting |
NCT04138277 -
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
|
Phase 3 |