Back to resultsStudy of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Primary Purpose
Glycogen Storage Disease, Type 14
Status
Unknown status
Phase
Phase 1
Locations
Turkey
Study Type
Interventional
Intervention
ORL-1G - D-galactose
Sponsored by
About this trial
This is an interventional treatment trial for Glycogen Storage Disease, Type 14 focused on measuring CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
Eligibility Criteria
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.
Sites / Locations
- Orpha LabsRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Treatment with ORL-1G - D-galactose
Arm Description
Outcomes
Primary Outcome Measures
Improvement in liver function.
Statistically significant decrease in plasma liver enzyme levels
Secondary Outcome Measures
Improvement in serum transferrin glycosylation pattern.
Decreased serum level of hypoglycosylated transferrin.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03404856
Brief Title
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Official Title
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Study Type
Interventional
2. Study Status
Record Verification Date
January 2019
Overall Recruitment Status
Unknown status
Study Start Date
October 31, 2017 (Actual)
Primary Completion Date
October 31, 2019 (Anticipated)
Study Completion Date
October 31, 2019 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Orpha Labs
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Study of ORL-1G in Patients With Glycogen Storage Disease Type 14
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease, Type 14
Keywords
CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment with ORL-1G - D-galactose
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
ORL-1G - D-galactose
Intervention Description
Oral ORL-1G - D-galactose
Primary Outcome Measure Information:
Title
Improvement in liver function.
Description
Statistically significant decrease in plasma liver enzyme levels
Time Frame
3 months after treatment starts
Secondary Outcome Measure Information:
Title
Improvement in serum transferrin glycosylation pattern.
Description
Decreased serum level of hypoglycosylated transferrin.
Time Frame
30 days after treatment starts
10. Eligibility
Sex
All
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Coordinator Study Coordinator
Phone
+90 537 763 6241
Email
business.development@orphalabs.com
Facility Information:
Facility Name
Orpha Labs
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator Study Coordinator
Phone
+905377636241
Email
business.development@orphalabs.com
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
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