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

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03404869

Enrollment

Registered

2018-01-19

Start date

2015-03-31

Completion date

2019-06-01

Last updated

2019-01-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

CDG Ib

Keywords

CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME

Brief summary

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

Interventions

DRUGORL-1M - D-mannose

Oral ORL-1M

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Improvement in hypoglycemia, diarrhea and vomiting.	6 months after treatment started	Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

Secondary

Measure	Time frame	Description
Improved glycosylation pattern of serum transferrin.	30 days after treatment started	Normalized isoelectric focusing pattern of serum transferrin.

Countries

Turkey (Türkiye)

Contacts

Primary ContactStudy Coordinator Study Coordinator

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026