Prospective Collection of Biospecimen in Pediatric Patients & Adult

See also
Status	Clinical Trial	Phase
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Completed	`NCT02054832` - Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch	N/A
Recruiting	`NCT05960617` - Efficacy and Safety of Empagliflozin in GSD-Ib Patients	Phase 2
Recruiting	`NCT04986735` - Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin

See also

Status

Clinical Trial

Phase

Enrolling by invitation

NCT03655223 - Early Check: Expanded Screening in Newborns

Completed

NCT02054832 - Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch

N/A

Recruiting

NCT05960617 - Efficacy and Safety of Empagliflozin in GSD-Ib Patients

Phase 2

Recruiting

NCT04986735 - Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT05915910
Study type	Observational
Source	Sanguine Biosciences
Contact
Status	Terminated
Phase
Start date	April 27, 2023
Completion date	March 13, 2024

Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms