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Status unknownNCT03404856Updated Jan 25, 2019

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

A Phase 1/2 interventional study of ORL-1G - D-galactose in Glycogen Storage Disease, Type 14, sponsored by Orpha Labs. Status unknown at 1 site in Turkey. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2019-01-25.

Sponsored by Orpha Labs · Phase 1/2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2019), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 1/2
Study type
Interventional
Enrollment
5
Allocation
Not applicable
Ages
Up to 18 Years
Sex
All
01

Study summary

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

02

Conditions studied

  • Glycogen Storage Disease, Type 14

Keywords

  • CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
03

In context

Glycogen Storage Disease

36 studies on the registry are indexed under Glycogen Storage Disease; 13 are open to participants now.

This study's planned enrollment of 5 is below the median of 16 across 18 interventional studies indexed under Glycogen Storage Disease.

Browse Glycogen Storage Disease studies →

Lead sponsor

Orpha Labs is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Diagnosis of any other disease that is not a manifestation of Glycogen Storage Disease Type 14.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
5 participants (estimated)

Study arms

  • Other
    Treatment with ORL-1G - D-galactose

    Drug: ORL-1G - D-galactose

Interventions

  • DrugORL-1G - D-galactose

    Oral ORL-1G - D-galactose

06

What researchers measure

Primary outcomes

  1. Improvement in liver function.

    Statistically significant decrease in plasma liver enzyme levels

    Time frame: 3 months after treatment starts

Secondary outcomes

  1. Improvement in serum transferrin glycosylation pattern.

    Decreased serum level of hypoglycosylated transferrin.

    Time frame: 30 days after treatment starts

07

Study locations

1 of 1 sites recruiting
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Oct 2017 (actual). Primary completion is estimated for Oct 2019, 24 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 13, 2018
First posted
Jan 19, 2018
StartActual
Oct 31, 2017
Primary completionEst.
Oct 31, 2019
CompletionEst.
Oct 31, 2019
Last update
Jan 25, 2019
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 25, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03404856
Lead sponsor
Orpha Labs
Responsible party
Sponsor
First posted
Jan 19, 2018
Start date
Oct 31, 2017
Primary completion
Oct 31, 2019 (estimated)
Completion
Oct 31, 2019 (estimated)
Last update
Jan 25, 2019

Study contacts

Study Coordinator Study Coordinator
Contact
business.development@orphalabs.com
+90 537 763 6241

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.

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Discussion

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