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

Phase 2UnknownNCT03404856

Orpha Labs5 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Glycogen Storage Disease, Type 14

Interventions

ORL-1G - D-galactoseDRUG

Oral ORL-1G - D-galactose

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Orpha Labs

Ankara, Turkey (Türkiye)

RECRUITING

Outcomes

Primary Outcomes

Improvement in liver function.

Statistically significant decrease in plasma liver enzyme levels

Time frame: 3 months after treatment starts

Secondary Outcomes

Improvement in serum transferrin glycosylation pattern.

Decreased serum level of hypoglycosylated transferrin.

Time frame: 30 days after treatment starts

Central Contacts

Study Coordinator Study Coordinator

CONTACT

+90 537 763 6241 business.development@orphalabs.com

Data from ClinicalTrials.gov

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