Study of ORL-1F (L-fucose) in Patients With Leukocyte Adh... | NCT03354533

Studies
Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II

NCT03354533

Sponsor

Orpha Labs

Status

Completed

Last Update Posted

Jan 25, 2019Actual

Enrollment

4Actual

Phase

Phase 1Phase 2

Conditions

Leukocyte Adhesion Deficiency, Type II

Interventions

L-fucose

Countries

Not provided

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

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 1, 2012Actual

Primary Completion Date

Nov 1, 2018Actual

Completion Date

Nov 1, 2018Actual

First Submitted Date

Nov 21, 2017

First Submission Date that Met QC Criteria

Nov 24, 2017

First Posted Date

Nov 28, 2017Actual

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

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

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

4Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Treatment with ORL-1F - L-fucose	Other		Drug: L-fucose

Interventions

Name	Type	Description	Arm Group Labels	Other Names
L-fucose	Drug	Oral ORL-1F	Treatment with ORL-1F - L-fucose

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Decrease in infection frequency	Statistically significant decrease in infection frequency	12 months after treatment started

Secondary Outcomes

Measure	Description	Time Frame
Decrease in neutrophil count	Statistically significant decrease in absolute neutrophil count	30 days after treatment started

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Diagnosis of Leukocyte Adhesion Deficiency Type II.
Less than 18 years old.

Exclusion Criteria:

Diagnosis of any other disease that is not a manifestation of Leukocyte Adhesion Deficiency Type II.

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

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
C535755	Congenital disorder of glycosylation, type 2C

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D005643	Fucose

Ancestor Terms

ID	Term
D003837	Deoxy Sugars
D002241	Carbohydrates

Browse Leaves

Not provided

Browse Branches

Not provided