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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03404869|
Recruitment Status : Unknown
Verified January 2019 by Orpha Labs.
Recruitment status was: Recruiting
First Posted : January 19, 2018
Last Update Posted : January 25, 2019
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|CDG Ib||Drug: ORL-1M - D-mannose||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Treatment with ORL-1M - D-mannose||
Drug: ORL-1M - D-mannose
- Improvement in hypoglycemia, diarrhea and vomiting. [ Time Frame: 6 months after treatment started ]Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.
- Improved glycosylation pattern of serum transferrin. [ Time Frame: 30 days after treatment started ]Normalized isoelectric focusing pattern of serum transferrin.
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.
|Ages Eligible for Study:||up to 18 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Diagnosis of CGD-1b.
- Less than 18 years old.
- Diagnosis of any other disease that is not a manifestation of CGD-1b.
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
|Contact: Study Coordinator Study Coordinator||+90 537 763 firstname.lastname@example.org|
|Ankara, Turkey, 06100|
|Contact: Study Coordinator Study Coordinator +905377636241 email@example.com|
|Responsible Party:||Orpha Labs|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Product Manufactured in and Exported from the U.S.:||No|
CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME