Study of ORL-1G (D-galactose) in Patients With Glycogen S... | NCT03404856

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

NCT03404856

Sponsor

Orpha Labs

Status

Unknown status

Last Update Posted

Jan 25, 2019Actual

Enrollment

5Estimated

Phase

Phase 1Phase 2

Conditions

Glycogen Storage Disease, Type 14

Interventions

ORL-1G - D-galactose

Countries

Turkey (Türkiye)

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 31, 2017Actual

Primary Completion Date

Oct 31, 2019Estimated

Completion Date

Oct 31, 2019Estimated

First Submitted Date

Jan 13, 2018

First Submission Date that Met QC Criteria

Jan 13, 2018

First Posted Date

Jan 19, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 22, 2019

Last Update Posted Date

Jan 25, 2019Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

5Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Treatment with ORL-1G - D-galactose	Other		Drug: ORL-1G - D-galactose

Interventions

Name	Type	Description	Arm Group Labels	Other Names
ORL-1G - D-galactose	Drug	Oral ORL-1G - D-galactose	Treatment with ORL-1G - D-galactose

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

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

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Study Coordinator Study Coordinator	Contact	+90 537 763 6241		business.development@orphalabs.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Orpha Labs	Recruiting	Ankara		06100	Turkey (Türkiye)

IPD Sharing Statement Module

Plan to Share IPD

Undecided

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D006008	Glycogen Storage Disease
C567859	Glycogen Storage Disease XIV

Ancestor Terms

ID	Term
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided