Browse Studies — Arctuva

GSDIa Disease Monitoring Program

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administratio…

Glycogen Storage Disease Type Ia

Estimated

~$10 – $1,050

Ultragenyx Pharmaceutical IncNCT06636383

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

The goal of this study is to determine if the Lactate Plus meter is accurate compared to lab lactate levels, and to determine if the Accu chek guide glucometer…

Glycogen Storage Disease Type IAGlycogen Storage Disease Type IGlycogen Storage Disease Type IB+1 more

Estimated

~$10 – $750

Connecticut Children's Medical CenterNCT07459582

Phase 1

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant

This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with…

Glycogen Storage Disease Type Ia

Estimated

~$10 – $8,750

Beam Therapeutics Inc.NCT06735755

Are Portable Lactate Monitoring Devices Accurate in Patients With Glycogen Storage Disease Ia When Compared to Blood Serum Lactates?

The goal of this observational study is to determine if home lactate meters (both capillary and serum sample) are accurate, compared to lab serum lactate in a …

Glycogen Storage Disease Type Ia

Estimated

~$10 – $750

Connecticut Children's Medical CenterNCT06843330