Trials / Conditions / Pompe Disease Infantile-Onset

Pompe Disease Infantile-Onset

10 registered clinical trials studyying Pompe Disease Infantile-Onset — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Enrolling By Invitation	The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Followi NCT07072676	LMU Klinikum	N/A
Recruiting	Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases NCT06833489	Assistance Publique Hopitaux De Marseille	N/A
Active Not Recruiting	Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug NCT05793307	GeneCradle Inc	Phase 1 / Phase 2
Recruiting	Registry of Patients Diagnosed With Lysosomal Storage Diseases NCT05619900	University of California, San Francisco	—
Completed	MSOT in Pompe Disease NCT05083806	University of Erlangen-Nürnberg Medical School	N/A
Recruiting	PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) NCT04532047	University of California, San Francisco	Phase 1
Unknown	Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease. NCT04755751	Rambam Health Care Campus	—
Terminated	A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Pa NCT01597596	Genzyme, a Sanofi Company	Phase 4
Completed	Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously NCT00763932	Genzyme, a Sanofi Company	Phase 2
Available	Expanded Access for ATB200/AT2221 for the Treatment of IOPD NCT04327973	Amicus Therapeutics	—