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

Phase 2UnknownNCT03404869

Orpha Labs5 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

CDG Ib

Interventions

ORL-1M - D-mannoseDRUG

Oral ORL-1M

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Orpha Labs

Ankara, Turkey (Türkiye)

RECRUITING

Outcomes

Primary Outcomes

Improvement in hypoglycemia, diarrhea and vomiting.

Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

Time frame: 6 months after treatment started

Secondary Outcomes

Improved glycosylation pattern of serum transferrin.

Normalized isoelectric focusing pattern of serum transferrin.

Time frame: 30 days after treatment started

Central Contacts

Study Coordinator Study Coordinator

CONTACT

+90 537 763 6241 business.development@orphalabs.com

Data from ClinicalTrials.gov

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