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

Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: OBSERVATIONAL

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

Brief Summary: The primary objective of this study is to collect whole blood from patients diagnosed with Glycogen storage disease type 1B, which will be used to support the investigation of potential therapies that address the genetic basis of this disease.

Detailed Summary:
Sponsor: Sanguine Biosciences

Current Primary Outcome: Bipspecimen collection from subjects or their parental guardians that are diagnosed with Glycogen Storage Disease Type Ib(GSD1b, von Gierke disease) for the purpose to analyze the biospecimens to support the investigation of potential therapies that address the genetic basis of this disease.
The primary outcome measure is the collection of biospecimens for quantitative analysis using PBMC processes to report cell viability of at least 5M cells per vial.

Original Primary Outcome: Bipspecimen collection from subjects or their parental guardians that are diagnosed with Glycogen Storage Disease Type Ib(GSD1b, von Gierke disease) for the purpose to analyze the biospecimens to support the investigation of potential therapies that address the genetic basis of this disease.
The primary outcome measure is the collection of biospecimens for quantitative analysis using PBMC processes to report cell viability of at least 5M cells per vial.

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Sanguine Biosciences

Dates:
Date Received: June 14, 2023
Date Started: April 27, 2023
Date Completion: April 27, 2023
Last Updated: November 20, 2023
Last Verified: November 01, 2023