Pompe Pregnancy Sub-Registry

Status Recruiting

Clinical Trial Summary

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.

Clinical Trial Description

Study Design Time Perspective: Retrospective and Prospective ;

Study Design

Related Conditions & MeSH terms

Disease
Glycogen Storage Disease
Glycogen Storage Disease Type II
Glycogen Storage Disease Type II (GSD-II)
Glycogenesis 2 Acid Maltase Deficiency
Pompe Disease (Late-onset)

Clinical Trial Details

NCT number	NCT00567073
Study type	Observational [Patient Registry]
Source	Sanofi
Contact	Trial Transparency email recommended (Toll free number for US &
Phone	800-633-1610
Email	Contact-Us@sanofi.com
Status	Recruiting
Phase
Start date	June 18, 2007
Completion date	January 31, 2034

View Details

See also
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	`NCT00455195` - Late-Onset Treatment Study Extension Protocol	Phase 4
Completed	`NCT00731081` - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®	N/A
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