Study of ORL-1M (D-mannose) in Patients With CDG-Ib

Phase 1

• Conditions: CDG Ib
• Interventions: Drug: ORL-1M - D-mannose

• Registration Number: NCT03404869
• Lead Sponsor: Orpha Labs

Brief Summary

Study of ORL-1M in Patients With CDG-Ib

Detailed Description

Not available

Study Timeline

• Study Start: 2015-03-31
• Study First Submitted: 2018-01-13
• Study First Submitted QC: 2018-01-13
• Study First Posted: 2018-01-19
• Status Verified: 2019-01
• Last Update Submitted: 2019-01-22
• Last Update Posted: 2019-01-25
• Primary Completion: 2019-06-01
• Study Completion: 2019-06-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Diagnosis of CGD-1b.
• Less than 18 years old.

Exclusion Criteria

• Diagnosis of any other disease that is not a manifestation of CGD-1b.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment with ORL-1M - D-mannose	ORL-1M - D-mannose	-

Primary Outcome Measures

Name	Time	Method
Improvement in hypoglycemia, diarrhea and vomiting.	6 months after treatment started	Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

Secondary Outcome Measures

Name	Time	Method
Improved glycosylation pattern of serum transferrin.	30 days after treatment started	Normalized isoelectric focusing pattern of serum transferrin.

Trial Locations

Locations (1)

Orpha Labs; 🇹🇷 Ankara, Turkey