Back to resultsStudy of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II
Primary Purpose
Leukocyte Adhesion Deficiency, Type II
Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
L-fucose
Sponsored by
About this trial
This is an interventional treatment trial for Leukocyte Adhesion Deficiency, Type II
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Treatment with ORL-1F - L-fucose
Arm Description
Outcomes
Primary Outcome Measures
Decrease in infection frequency
Statistically significant decrease in infection frequency
Secondary Outcome Measures
Decrease in neutrophil count
Statistically significant decrease in absolute neutrophil count
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03354533
Brief Title
Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II
Official Title
Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II
Study Type
Interventional
2. Study Status
Record Verification Date
January 2019
Overall Recruitment Status
Completed
Study Start Date
January 1, 2012 (Actual)
Primary Completion Date
November 1, 2018 (Actual)
Study Completion Date
November 1, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Orpha Labs
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Study of ORL-1F in Patients With Leukocyte Adhesion Deficiency Type II
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukocyte Adhesion Deficiency, Type II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment with ORL-1F - L-fucose
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
L-fucose
Intervention Description
Oral ORL-1F
Primary Outcome Measure Information:
Title
Decrease in infection frequency
Description
Statistically significant decrease in infection frequency
Time Frame
12 months after treatment started
Secondary Outcome Measure Information:
Title
Decrease in neutrophil count
Description
Statistically significant decrease in absolute neutrophil count
Time Frame
30 days after treatment started
10. Eligibility
Sex
All
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II
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