GM3 Synthase DeficiencyNCT02234024
Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder
A study testing Supplementation of dairy-derived concentrated gangliosides. in people with GM3 Synthase Deficiency, run by DDC Clinic - Center for Special Needs Children.
Trial summary
The purpose of this pilot project is to see if a supplemental form of dietary gangliosides can serve as a potential treatment for the rare metabolic condition called ganglioside GM3 synthase deficiency. Read more about this study
- Who is it for?
- Ages Up to 20 years
- Conditions the study is for
- GM3 Synthase Deficiency
- What is being tested?
- Supplementation of dairy-derived concentrated gangliosides.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Jan 2014to Dec 2020
- Phase
- Interventional
- 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 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical diagnosis of GM3 synthase deficiency
Who cannot join
- -
About this study
Official title: Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency
The purpose of this pilot project is to see if a supplemental form of dietary gangliosides can serve as a potential treatment for the rare metabolic condition called ganglioside GM3 synthase deficiency.
- Conditions
- GM3 Synthase Deficiency
- Being tested
- Supplementation of dairy-derived concentrated gangliosides.
- Medical subject headings
- Amish Infantile Epilepsy Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Supplementation of dairy-derived concentrated gangliosides.Dietary supplement
| Study group | Role | What they receive |
|---|---|---|
| Supplementation of gangliosides | Experimental | Supplementation of dairy-derived concentrated gangliosides Receives: Dietary Supplement: Supplementation of dairy-derived concentrated gangliosides. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Body length compared to normal pediatric growth curves.
Measured: Quarterly measures- change from baseline over 24 months.
Change in scores of standardized developmental assessments (Vineland & Batelle)
Measured: Quarterly measures - change from baseline over 24 months
Body Weight compared to normal pediatric growth curves
Measured: Quarterly measurements from baseline over 24 months
Head circumference compared to normal pediatric growth curves.
Measured: Quarterly measures from baseline over 24 months
Secondary outcomes (1)
Concentration of ganglioside GM3 in blood plasma
Measured: Six times per year over 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2014 (estimated)
- Study startJan 2014
- Primary completionDec 2020 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GM3 Synthase DeficiencyStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- DDC Clinic - Center for Special Needs Children
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Heng Wang, MD PhD, Principal investigator, DDC Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02234024
- Sponsor's protocol ID
- DDC-62314
- Status last verified
- Feb 2019
- First submitted
- 3 Sep 2014
- Last updated
- 7 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency
- ClinicalTrials.gov ID
- NCT02234024
- Acronym / sponsor ID
- DDC-62314
- Conditions
- GM3 Synthase Deficiency
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- DDC Clinic - Center for Special Needs ChildrenUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- Dec 2020 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 9 Sep 2014 (estimated)
- 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.
The purpose of this pilot project is to see if a supplemental form of dietary gangliosides can serve as a potential treatment for the rare metabolic condition called ganglioside GM3 synthase deficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Supplementation of gangliosides | Experimental | Dietary Supplement: Supplementation of dairy-derived concentrated gangliosides.Supplementation of dairy-derived concentrated gangliosides |
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Clinical diagnosis of GM3 synthase deficiency
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| Supplementation of dairy-derived concentrated gangliosides. | Dietary supplement | Supplementation of gangliosides |
Primary outcome
Body length compared to normal pediatric growth curves.
Time frame: Quarterly measures- change from baseline over 24 months.
Change in scores of standardized developmental assessments (Vineland & Batelle)
Time frame: Quarterly measures - change from baseline over 24 months
Body Weight compared to normal pediatric growth curves
Time frame: Quarterly measurements from baseline over 24 months
Head circumference compared to normal pediatric growth curves.
Time frame: Quarterly measures from baseline over 24 months
1 secondary outcome
Concentration of ganglioside GM3 in blood plasma
Time frame: Six times per year over 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2019.
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.
Heng Wang, MD PhD principal investigator
DDC Clinic
Questions about this trial
What is this clinical trial studying?
A study testing Supplementation of dairy-derived concentrated gangliosides. in people with GM3 Synthase Deficiency, run by DDC Clinic - Center for Special Needs Children.
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 1 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 7 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Body length compared to normal pediatric growth curves. (time frame: Quarterly measures- change from baseline over 24 months.). The study lists 4 primary outcomes in all. 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 7 Feb 2019.