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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03404869
Recruitment Status : Recruiting
First Posted : January 19, 2018
Last Update Posted : January 25, 2019
Sponsor:
Information provided by (Responsible Party):
Orpha Labs

Brief Summary:
Study of ORL-1M in Patients With CDG-Ib

Condition or disease Intervention/treatment Phase
CDG Ib Drug: ORL-1M - D-mannose Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of ORL-1M (D-mannose) in Patients With CDG-Ib
Actual Study Start Date : March 31, 2015
Estimated Primary Completion Date : June 1, 2019
Estimated Study Completion Date : June 1, 2019

Arm Intervention/treatment
Treatment with ORL-1M - D-mannose Drug: ORL-1M - D-mannose
Oral ORL-1M




Primary Outcome Measures :
  1. Improvement in hypoglycemia, diarrhea and vomiting. [ Time Frame: 6 months after treatment started ]
    Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.


Secondary Outcome Measures :
  1. Improved glycosylation pattern of serum transferrin. [ Time Frame: 30 days after treatment started ]
    Normalized isoelectric focusing pattern of serum transferrin.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03404869


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

Locations
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Turkey
Orpha Labs Recruiting
Ankara, Turkey, 06100
Contact: Study Coordinator Study Coordinator    +905377636241    study.coordinator@orphalabs.com   
Sponsors and Collaborators
Orpha Labs

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Responsible Party: Orpha Labs
ClinicalTrials.gov Identifier: NCT03404869    
Other Study ID Numbers: PMID-1
First Posted: January 19, 2018    Key Record Dates
Last Update Posted: January 25, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Orpha Labs:
CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME