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

Phase 1

• Conditions: Glycogen Storage Disease, Type 14
• Interventions: Drug: ORL-1G - D-galactose

• Registration Number: NCT03404856
• Lead Sponsor: Orpha Labs

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2017-10-31
• Study First Submitted: 2018-01-13
• Study First Submitted QC: 2018-01-13
• Study First Posted: 2018-01-19
• Status Verified: 2019-01
• Last Update Submitted: 2019-01-22
• Last Update Posted: 2019-01-25
• Primary Completion: 2019-10-31
• Study Completion: 2019-10-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment with ORL-1G - D-galactose	ORL-1G - D-galactose	-

Primary Outcome Measures

Name	Time	Method
Improvement in liver function.	3 months after treatment starts	Statistically significant decrease in plasma liver enzyme levels

Secondary Outcome Measures

Name	Time	Method
Improvement in serum transferrin glycosylation pattern.	30 days after treatment starts	Decreased serum level of hypoglycosylated transferrin.

Trial Locations

Locations (1)

Orpha Labs; 🇹🇷 Ankara, Turkey