Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14

Glycogen Storage Disease, Type 14 Clinical Trial

Official title:

Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03404856
• Other study ID #: GALA-1
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 31, 2017
• Est. completion date: October 31, 2019

Study information

• Verified date: January 2019
• Source: Orpha Labs
• Contact: Study Coordinator Study Coordinator
• Phone: +90 537 763 6241
• Email: business.development[@]orphalabs.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Study of ORL-1G in Patients With Glycogen Storage Disease Type 14

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: October 31, 2019
• Est. primary completion date: October 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

• Diagnosis of Glycogen Storage Disease Type 14.

• Less than 18 years old.

Exclusion Criteria:

• Diagnosis of any other disease that is not a manifestation of Glycogen Storage Disease Type 14.

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease, Type 14
• Metabolic Diseases

Intervention

Drug:
• ORL-1G - D-galactose
Oral ORL-1G - D-galactose

Locations

Country	Name	City	State
Turkey	Orpha Labs	Ankara

Sponsors (1)

Lead Sponsor	Collaborator
Orpha Labs

Country where clinical trial is conducted

Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement in liver function.	Statistically significant decrease in plasma liver enzyme levels	3 months after treatment starts
Secondary	Improvement in serum transferrin glycosylation pattern.	Decreased serum level of hypoglycosylated transferrin.	30 days after treatment starts