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

Phase 2CompletedNCT03354533

Orpha Labs4 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Leukocyte Adhesion Deficiency, Type II

Interventions

L-fucoseDRUG

Oral ORL-1F

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Decrease in infection frequency

Statistically significant decrease in infection frequency

Time frame: 12 months after treatment started

Secondary Outcomes

Decrease in neutrophil count

Statistically significant decrease in absolute neutrophil count

Time frame: 30 days after treatment started

Data from ClinicalTrials.gov

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