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

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ClinicalTrials.gov Identifier: NCT03404856
Recruitment Status : Recruiting
First Posted : January 19, 2018
Last Update Posted : January 25, 2019
Sponsor:
Information provided by (Responsible Party):
Orpha Labs

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

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease, Type 14 Drug: ORL-1G - D-galactose Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Actual Study Start Date : October 31, 2017
Estimated Primary Completion Date : October 31, 2019
Estimated Study Completion Date : October 31, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Treatment with ORL-1G - D-galactose Drug: ORL-1G - D-galactose
Oral ORL-1G - D-galactose




Primary Outcome Measures :
  1. Improvement in liver function. [ Time Frame: 3 months after treatment starts ]
    Statistically significant decrease in plasma liver enzyme levels


Secondary Outcome Measures :
  1. Improvement in serum transferrin glycosylation pattern. [ Time Frame: 30 days after treatment starts ]
    Decreased serum level of hypoglycosylated transferrin.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03404856


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

Locations
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Turkey
Orpha Labs Recruiting
Ankara, Turkey, 06100
Contact: Study Coordinator Study Coordinator    +905377636241    business.development@orphalabs.com   
Sponsors and Collaborators
Orpha Labs

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Responsible Party: Orpha Labs
ClinicalTrials.gov Identifier: NCT03404856     History of Changes
Other Study ID Numbers: GALA-1
First Posted: January 19, 2018    Key Record Dates
Last Update Posted: January 25, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orpha Labs:
CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
Additional relevant MeSH terms:
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Glycogen Storage Disease
Metabolic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn