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Active clinical trials for "Glycogen Storage Disease Type I"

Results 1-10 of 12

Efficacy and Safety of Empagliflozin in GSD-Ib Patients

Glycogen Storage Disease Type IB

Empagliflozin Treatment of GSD-1b patients

Recruiting11 enrollment criteria

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in...

Glycogen Storage Disease Type IA

The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.

Active17 enrollment criteria

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

Glucose 6 Phosphatase Deficiency

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

Not yet recruiting11 enrollment criteria

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IAVon Gierke's Disease (GSD Type Ia)

The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

Active5 enrollment criteria

The Use of Uncooked Sweet Manioc Starch to Treat Hepatic Glycogen Storage Diseases

Glycogen Storage Disease Type I

Hepatic Glycogen Storage Diseases are a group of 10 serious genetic diseases that present in childhood and are characterized more frequently by the occurrence of repetitive hypoglycemia and dyslipidemia. Regarding treatment, the most commonly used strategy is the frequent administration of uncooked cornstarch, in average, every 4 hours. Although this treatment is successful, the use of large amounts of cornstarch can lead to overweight and, especially, to the decrease in the quality of life of patients and caregivers, due to the need to use the starch during the night. The search for a treatment that is widely available and that can lead to the prolongation of the fasting time, can collaborate to improve the care of these patients. The main scientific question to be answered by this research is: does sweet manioc starch, a Brazilian product, safely prolong the fasting time (with normoglycemia) of the patients as already suggested in experimental models? Main objective: To evaluate the efficacy and safety of the use of uncooked Sweet Manioc Starch in the treatment of patients with hepatic Glycogen Storage Diseases, using as model the Glycogen Storage Diseases type Ia.

Completed2 enrollment criteria

Comparison of the Effect of a Novel Starch (Glycosade) Versus Gastrostomy Tube-Dextrose Infusion...

Glycogen Storage Disorder Type 1Hypoglycemia2 more

The objective of this demonstration project is to compare a novel long-acting starch, Glycosade, a hydrothermally processed high amylopectin maize starch, versus gastrostomy tube-dextrose infusion in maintaining euglycaemia overnight in children with GSD-I. Glycosade has been reported to increase the duration of euglycaemia. Its slow release and longer periods of normal blood sugar achieved would preclude the need for the overnight dextrose infusion and eliminate the need for the surgical insertion of a gastrostomy tube for this purpose. Glycosade also reportedly causes fewer gastrointestinal side effects, thus potentially improving compliance to therapy. The investigators intend to evaluate Glycosade in our patients and determine its efficacy on glucose control, on the length of normoglycemia achieved and to determine if there are reduced side effects in our patients with GSD-I. This will be accomplished by an open label study of Glycosade in GSD-I patients who consent to the protocol.

Completed3 enrollment criteria

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Glycogen Storage Disease Type I

This study will be an open-label, prospective, interventional feasibility pilot project to study the efficacy, safety, and tolerability of UX007 (triheptanoin) on reducing hypoglycemic events in patients with GSD I. Subjects will serve as their own control. Five (5) subjects who are treatment naïve to UX007 (triheptanoin) and are already on standard dietary therapy for GSDI will be enrolled. The primary objective is to evaluate the efficacy, safety, and tolerability of UX007 (triheptanoin) in patients with GSD I. The secondary objectives include evaluating the effect of UX007 (triheptanoin) on maintaining the duration of normoglycemia between meals based on glucose monitoring (Preventing and reducing the frequency of hypoglycemia); reduction/stabilization of the dose of cornstarch; and the prevention of increased liver steatosis based on ultrasound with elastography.

Completed8 enrollment criteria

Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type...

Glycogen Storage Disease Type IGlucose 6 Phosphatase Deficiency

Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin

Unknown status11 enrollment criteria

Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IA

Glycogen storage disease type Ia (GSDIa) subjects retain a limited capacity for endogenous glucose production (EGP). To date, the origin of residual EGP in GSDIa patients is unknown. Either increased glycogen debranching or lysosomal glycogen breakdown can account for residual EGP in GSDIa. Innovative treatments for GSDIa (e.g. AAV8-mediated gene therapy and mRNA therapy) are being developed.Therefore, longitudinal minimally-invasive monitoring of outcomes after therapeutic interventions in GSD Ia subjects becomes warranted. The primary objective is to test the feasibility of EGP quantification in adult GSDIa subjects by stable isotopes after a single oral [6,6-2H2]glucose dose. Secondary objectives are to compare EGP assessed by a single oral [6,6-2H2]glucose dose (a) in GSDIa patients versus matched healthy participants, (b) among GSDIa patients, (c) in the pre-prandial state versus the fed state, (d) in the controlled hospital setting versus the home setting. Data collected from the continuous glucose monitoring data will also be compared

Completed9 enrollment criteria

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

Glycogen Storage Disease Type IA

The primary objective of this study is to assess the percentage of time patients were in normal glucose control.

Completed4 enrollment criteria
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