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Status unknownNCT03404869Updated Jan 25, 2019

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

A Phase 1/2 interventional study of ORL-1M - D-mannose in CDG Ib, sponsored by Orpha Labs. Status unknown at 1 site in Turkey. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2019-01-25.

Sponsored by Orpha Labs · Phase 1/2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2019), so the status shown — last known as Recruiting — may be out of date.

From the registry’s dates

  • Registered 2 years 9 months after the study started (first participant enrolled Mar 2015, registered Jan 2018).
Phase
Phase 1/2
Study type
Interventional
Enrollment
5
Allocation
Not applicable
Ages
Up to 18 Years
Sex
All
01

Study summary

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

02

Conditions studied

  • CDG Ib

Keywords

  • CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME
03

In context

Lead sponsor

Orpha Labs is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Diagnosis of any other disease that is not a manifestation of CGD-1b.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
5 participants (estimated)

Study arms

  • Other
    Treatment with ORL-1M - D-mannose

    Drug: ORL-1M - D-mannose

Interventions

  • DrugORL-1M - D-mannose

    Oral ORL-1M

06

What researchers measure

Primary outcomes

  1. Improvement in hypoglycemia, diarrhea and vomiting.

    Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

    Time frame: 6 months after treatment started

Secondary outcomes

  1. Improved glycosylation pattern of serum transferrin.

    Normalized isoelectric focusing pattern of serum transferrin.

    Time frame: 30 days after treatment started

07

Study locations

1 of 1 sites recruiting
08

References and documents

Publications

  • 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. PubMed 35120836 ↗

Individual participant data

Plan to share: Undecided

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Mar 2015 (actual). Primary completion is estimated for Jun 2019, 50 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 13, 2018
First posted
Jan 19, 2018
StartActual
Mar 31, 2015
Primary completionEst.
Jun 1, 2019
CompletionEst.
Jun 1, 2019
Last update
Jan 25, 2019
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 25, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03404869
Lead sponsor
Orpha Labs
Responsible party
Sponsor
First posted
Jan 19, 2018
Start date
Mar 31, 2015
Primary completion
Jun 1, 2019 (estimated)
Completion
Jun 1, 2019 (estimated)
Last update
Jan 25, 2019

Study contacts

Study Coordinator Study Coordinator
Contact
business.development@orphalabs.com
+90 537 763 6241

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.

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Discussion

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Congenital disorder of glycosylation type 1B

Other studies from this sponsor

Orpha Labs

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