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

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03404856

Enrollment

Registered

2018-01-19

Start date

2017-10-31

Completion date

2019-10-31

Last updated

2019-01-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen Storage Disease, Type 14

Keywords

CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV

Brief summary

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

Interventions

DRUGORL-1G - D-galactose

Oral ORL-1G - D-galactose

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Improvement in liver function.	3 months after treatment starts	Statistically significant decrease in plasma liver enzyme levels

Secondary

Measure	Time frame	Description
Improvement in serum transferrin glycosylation pattern.	30 days after treatment starts	Decreased serum level of hypoglycosylated transferrin.

Countries

Turkey (Türkiye)

Contacts

Primary ContactStudy Coordinator Study Coordinator

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026