Leukocyte Adhesion Deficiency, Type II
Conditions
Brief summary
Study of ORL-1F in Patients With Leukocyte Adhesion Deficiency Type II
Interventions
DRUGL-fucose
Oral ORL-1F
Sponsors
Orpha Labs
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Decrease in infection frequency | 12 months after treatment started | Statistically significant decrease in infection frequency |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Decrease in neutrophil count | 30 days after treatment started | Statistically significant decrease in absolute neutrophil count |
Outcome results
None listed