NCT03404869

Brief Summary

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2015

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 31, 2015

Completed
2.8 years until next milestone

First Submitted

Initial submission to the registry

January 13, 2018

Completed
6 days until next milestone

First Posted

Study publicly available on registry

January 19, 2018

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2019

Completed
Last Updated

January 25, 2019

Status Verified

January 1, 2019

Enrollment Period

4.2 years

First QC Date

January 13, 2018

Last Update Submit

January 22, 2019

Conditions

CDG Ib

Keywords

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

Outcome Measures

Primary Outcomes (1)

  • Improvement in hypoglycemia, diarrhea and vomiting.

    Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.

    6 months after treatment started

Secondary Outcomes (1)

  • Improved glycosylation pattern of serum transferrin.

    30 days after treatment started

Study Arms (1)

Treatment with ORL-1M - D-mannose

OTHER
Drug: ORL-1M - D-mannose

Interventions

ORL-1M - D-mannoseDRUG

Oral ORL-1M

Treatment with ORL-1M - D-mannose

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Orpha Labs

Ankara, 06100, Turkey (Türkiye)

RECRUITING

Related Publications (1)

  • 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: 35120836DERIVED

MeSH Terms

Conditions

Congenital disorder of glycosylation type 1B

Central Study Contacts

Study Coordinator Study Coordinator

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 13, 2018

First Posted

January 19, 2018

Study Start

March 31, 2015

Primary Completion

June 1, 2019

Study Completion

June 1, 2019

Last Updated

January 25, 2019

Record last verified: 2019-01

Locations

TU(1)