Glycogen Storage Disease, Type 14Phase 1/2NCT03404856
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
A Phase 1/2 study testing ORL-1G - D-galactose in people with Glycogen Storage Disease, Type 14, run by Orpha Labs at 1 site in Turkey (Türkiye).
Trial summary
Study of ORL-1G in Patients With Glycogen Storage Disease Type 14 Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Glycogen Storage Disease, Type 14
- What is being tested?
- ORL-1G - D-galactose
- 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 Oct 2017to 31 Oct 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 Glycogen Storage Disease Type 14.
- Less than 18 years old.
Who cannot join
- Diagnosis of any other disease that is not a manifestation of Glycogen Storage Disease Type 14.
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, business.development@orphalabs.com
About this study
Study of ORL-1G in Patients With Glycogen Storage Disease Type 14
- Conditions
- Glycogen Storage Disease, Type 14
- Being tested
- ORL-1G - D-galactose
- Keywords
- CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
- Medical subject headings
- Glycogen Storage Disease, Glycogen Storage Disease XIV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ORL-1G - D-galactoseDrug
Oral ORL-1G - D-galactose
| Study group | Role | What they receive |
|---|---|---|
| Treatment with ORL-1G - D-galactose | Other | Not described Receives: Drug: ORL-1G - D-galactose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in liver function.
Statistically significant decrease in plasma liver enzyme levels
Measured: 3 months after treatment starts
Secondary outcomes (1)
Improvement in serum transferrin glycosylation pattern.
Decreased serum level of hypoglycosylated transferrin.
Measured: 30 days after treatment starts
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 Oct 2017
- Primary completion31 Oct 2019 (estimated)
- Study completion31 Oct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease, Type 14Studies 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
- NCT03404856
- Sponsor's protocol ID
- GALA-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
- FDA-regulated device
- No
- Individual data sharing
- Undecided
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-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
- ClinicalTrials.gov ID
- NCT03404856
- Acronym / sponsor ID
- GALA-1
- Conditions
- Glycogen Storage Disease, Type 14
- Keywords
- CDG It; CDGIt PHOSPHOGLUCOMUTASE 1 DEFICIENCY PGM1 DEFICIENCY GLYCOGEN STORAGE DISEASE XIV; GSD14 GSD XIV
- 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 Oct 2017 (actual)
- Primary completion
- 31 Oct 2019 (estimated)
- Study completion
- 31 Oct 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-1G in Patients With Glycogen Storage Disease Type 14
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-1G - D-galactose | Other | Drug: ORL-1G - D-galactose |
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 Glycogen Storage Disease Type 14.
- Less than 18 years old.
Key exclusion criteria (1 listed)
- Diagnosis of any other disease that is not a manifestation of Glycogen Storage Disease Type 14.
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-1G - D-galactoseOral ORL-1G - D-galactose | Drug | Treatment with ORL-1G - D-galactose |
Primary outcome
Improvement in liver function.
Time frame: 3 months after treatment starts
Statistically significant decrease in plasma liver enzyme levels
1 secondary outcome
Improvement in serum transferrin glycosylation pattern.
Time frame: 30 days after treatment starts
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.
- Individual participant dataSharing planned: Undecided
Related trials for Glycogen Storage Disease, Type 14
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
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-1G - D-galactose in people with Glycogen Storage Disease, Type 14, 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 liver function. (time frame: 3 months after treatment starts). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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.