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

NCT03404869 — Phase 1 / Phase 2, Unknown, 5 participants, Orpha Labs

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Registry	NCT03404869
Status	Unknown
Phase	Phase 1 / Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	5 (Estimated)
Sponsor	Orpha Labs (Industry)
Conditions	CDG Ib
Interventions	ORL-1M - D-mannose (Drug)
Dates	start 2015-03-31, primary completion 2019-06-01 (estimated), completion 2019-06-01 (estimated), first posted 2018-01-19, last updated 2019-01-25
Locations	1 site in Turkey (Türkiye)

-Summary

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

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+Arms and interventions

Arm	Type	Interventions	Description
Treatment with ORL-1M - D-mannose	Other	Drug: ORL-1M - D-mannose

ORL-1M - D-mannose (Drug): Oral ORL-1M

+Outcome measures

Primary outcomes

Improvement in hypoglycemia, diarrhea and vomiting. [6 months after treatment started]: Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

Secondary outcomes

Improved glycosylation pattern of serum transferrin. [30 days after treatment started]: Normalized isoelectric focusing pattern of serum transferrin.

+Eligibility

Sex: All, ages: any to 18 Years

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)

Facility	City	Country	Status
Orpha Labs	Ankara	Turkey (Türkiye)	Recruiting

+Publications

Results publication: Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1. PMID 35120836

My Notes

Public

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