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

January 22, 2019 updated by: Orpha Labs
Study of ORL-1M in Patients With CDG-Ib

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
OTHER: Treatment with ORL-1M - D-mannose
Drug: ORL-1M - D-mannose
Oral ORL-1M

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Orpha Labs

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

March 31, 2015

Primary Completion (ANTICIPATED)

June 1, 2019

Study Completion (ANTICIPATED)

June 1, 2019

Study Registration Dates

First Submitted

January 13, 2018

First Submitted That Met QC Criteria

January 13, 2018

First Posted (ACTUAL)

January 19, 2018

Study Record Updates

Last Update Posted (ACTUAL)

January 25, 2019

Last Update Submitted That Met QC Criteria

January 22, 2019

Last Verified

January 1, 2019

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • PMID-1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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