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
- Are there results?
- None posted in the registry
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
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of ORL-1M (D-mannose) in Patients With CDG-Ib
- ClinicalTrials.gov ID
- NCT03404869
- Acronym / sponsor ID
- PMID-1
- Conditions
- CDG Ib
- Keywords
- CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Orpha LabsIndustry (pharma, biotech, devices)
- Study start
- 31 Mar 2015 (actual)
- Primary completion
- 1 Jun 2019 (estimated)
- Study completion
- 1 Jun 2019 (estimated)
- First posted
- 19 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study of ORL-1M in Patients With CDG-Ib
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with ORL-1M - D-mannose | Other | Drug: ORL-1M - D-mannose |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Diagnosis of CGD-1b.
- Less than 18 years old.
Key exclusion criteria (1 listed)
- Diagnosis of any other disease that is not a manifestation of CGD-1b.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ORL-1M - D-mannoseOral ORL-1M | Drug | Treatment with ORL-1M - D-mannose |
Primary outcome
Improvement in hypoglycemia, diarrhea and vomiting.
Time frame: 6 months after treatment started
Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.
1 secondary outcome
Improved glycosylation pattern of serum transferrin.
Time frame: 30 days after treatment started
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jan 2019.
- Derived from this study: PubMed 35120836Rebelo 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.
- Individual participant dataSharing planned: Undecided
Related trials for CDG Ib
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Study Coordinator Study Coordinator (+90 537 763 6241, business.development@orphalabs.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement in hypoglycemia, diarrhea and vomiting. (time frame: 6 months after treatment started). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03404869, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jan 2019.