NCT03404856

Brief Summary

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2017

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 31, 2017

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

January 13, 2018

Completed
6 days until next milestone

First Posted

Study publicly available on registry

January 19, 2018

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2019

Completed
Last Updated

January 25, 2019

Status Verified

January 1, 2019

Enrollment Period

2 years

First QC Date

January 13, 2018

Last Update Submit

January 22, 2019

Conditions

Glycogen Storage Disease, Type 14

Keywords

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

Outcome Measures

Primary Outcomes (1)

  • Improvement in liver function.

    Statistically significant decrease in plasma liver enzyme levels

    3 months after treatment starts

Secondary Outcomes (1)

  • Improvement in serum transferrin glycosylation pattern.

    30 days after treatment starts

Study Arms (1)

Treatment with ORL-1G - D-galactose

OTHER
Drug: ORL-1G - D-galactose

Interventions

ORL-1G - D-galactoseDRUG

Oral ORL-1G - D-galactose

Treatment with ORL-1G - D-galactose

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of Glycogen Storage Disease Type 14.
  • Less than 18 years old.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Orpha Labs

Ankara, 06100, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Glycogen Storage DiseaseGlycogen Storage Disease XIV

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Study Coordinator Study Coordinator

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 13, 2018

First Posted

January 19, 2018

Study Start

October 31, 2017

Primary Completion

October 31, 2019

Study Completion

October 31, 2019

Last Updated

January 25, 2019

Record last verified: 2019-01

Locations

TU(1)