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13 recruiting studies matching your filters

A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult part…

Late-onset Pompe Disease

Estimated

~$10 – $8,750

Denali Therapeutics Inc.NCT07354724

Phase 1

A Single-Arm, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of AB-1009 in Adult Participants With Late-Onset Pompe Disease (LOPD)

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult …

Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD

Estimated

~$10 – $8,750

AskBio IncNCT07282847

Phase 2

A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…

Pompe Disease Registry

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to tra…

Glycogen Storage Disease Type IIPompe Disease

Estimated

~$10 – $1,125

Genzyme, a Sanofi CompanyNCT00231400

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 wom…

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

Estimated

~$10 – $1,125

Genzyme, a Sanofi CompanyNCT00567073

Phase 1

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety an…

MPS IMPS IIMPS IVA+6 more

Estimated

~$10 – $5,625

University of California, San FranciscoNCT04532047

A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and inf…

Pompe Disease

Estimated

~$10 – $8,750

Amicus TherapeuticsNCT06121011

Observational

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease

This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adult…

Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more

Estimated

~$10 – $6,250

University of Missouri-ColumbiaNCT07478172

Registry of Patients Diagnosed With Lysosomal Storage Diseases

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dis…

Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more

Estimated

~$10 – $675

University of California, San FranciscoNCT05619900

A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting

This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation, t…

Pompe DiseasePregnancy

Estimated

~$10 – $7,500

SanofiNCT05734521

Cognitive and Neurological Pathologies in Pompe Disease

The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain…

Pompe Disease

Estimated

~$10 – $750

Duke UniversityNCT04639336

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…

Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more

Estimated

~$10 – $1,125

xCuresNCT06539169