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

Leukocyte Adhesion Deficiency, Type II Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03354533
• Other study ID #: Ladtwo-1
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: January 1, 2012
• Est. completion date: November 1, 2018

Study information

• Verified date: January 2019
• Source: Orpha Labs
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: November 1, 2018
• Est. primary completion date: November 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Congenital Disorders of Glycosylation
• Leukocyte Adhesion Deficiency, Type II
• Tissue Adhesions

Intervention

Drug:
• L-fucose
Oral ORL-1F

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Orpha Labs

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Decrease in infection frequency	Statistically significant decrease in infection frequency	12 months after treatment started
Secondary	Decrease in neutrophil count	Statistically significant decrease in absolute neutrophil count	30 days after treatment started