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

Phase 1

Completed

• Conditions: Leukocyte Adhesion Deficiency, Type II
• Interventions: Drug: L-fucose

• Registration Number: NCT03354533
• Lead Sponsor: Orpha Labs

Brief Summary

Study of ORL-1F in Patients With Leukocyte Adhesion Deficiency Type II

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-01
• Study First Submitted: 2017-11-21
• Study First Submitted QC: 2017-11-24
• Study First Posted: 2017-11-28
• Primary Completion: 2018-11-01
• Study Completion: 2018-11-01
• Status Verified: 2019-01
• Last Update Submitted: 2019-01-22
• Last Update Posted: 2019-01-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment with ORL-1F - L-fucose	L-fucose	-

Primary Outcome Measures

Name	Time	Method
Decrease in infection frequency	12 months after treatment started	Statistically significant decrease in infection frequency

Secondary Outcome Measures

Name	Time	Method
Decrease in neutrophil count	30 days after treatment started	Statistically significant decrease in absolute neutrophil count