CDG IbPhase 1/2NCT03404869
Study of ORL-1M (D-mannose) in Patients With CDG-Ib
A Phase 1/2 study testing ORL-1M - D-mannose in people with CDG Ib, run by Orpha Labs at 1 site in Turkey (Türkiye).
Trial summary
Study of ORL-1M in Patients With CDG-Ib Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- CDG Ib
- What is being tested?
- ORL-1M - D-mannose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 31 Mar 2015to 1 Jun 2019
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of CGD-1b.
- Less than 18 years old.
Who cannot join
- Diagnosis of any other disease that is not a manifestation of CGD-1b.
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Orpha Labs
Ankara, 06100
Study Coordinator Study Coordinator, +905377636241, study.coordinator@orphalabs.com
About this study
Study of ORL-1M in Patients With CDG-Ib
- Conditions
- CDG Ib
- Being tested
- ORL-1M - D-mannose
- Keywords
- CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME
- Medical subject headings
- Congenital disorder of glycosylation type 1B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ORL-1M - D-mannoseDrug
Oral ORL-1M
| Study group | Role | What they receive |
|---|---|---|
| Treatment with ORL-1M - D-mannose | Other | Not described Receives: Drug: ORL-1M - D-mannose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in hypoglycemia, diarrhea and vomiting.
Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.
Measured: 6 months after treatment started
Secondary outcomes (1)
Improved glycosylation pattern of serum transferrin.
Normalized isoelectric focusing pattern of serum transferrin.
Measured: 30 days after treatment started
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2018
- Study start31 Mar 2015
- Primary completion1 Jun 2019 (estimated)
- Study completion1 Jun 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CDG IbStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Orpha Labs
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03404869
- Sponsor's protocol ID
- PMID-1
- Status last verified
- Jan 2019
- First submitted
- 13 Jan 2018
- Last updated
- 25 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- Individual data sharing
- Undecided
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