Mayo Clinic Minnesota
Rochester, Minnesota, 55905, United States
Not Yet Recruiting
NCT Number: NCT05402384
AVTX-801 D-galactose Supplementation in SLC35A2-CDG
This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-CDG
Not Yet Recruiting
Trial opening soon.
Get NotifiedKey information
Age range
1 month and older
Sex eligibility
All sexes
Study type
Interventional
Phase
Phase 2
Primary location
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
- Molecular confirmation of SLC35A2 genetic variant
- Age > 1 month
- Presence of seizures, chronic vomiting, chronic constipation, or chronic diarrhea
- A parent or legal guardian must be available and willing to provide consent on behalf of minor subjects or adult subjects who are unable to give informed consent due to developmental disabilities.
- Use of contraception in females > age 8 years
- Previously performed eye exam within last year
Exclusion criteria
- Aldolase-B deficiency
- Galactosemia
- Hemolytic uremic syndrome
- Hemoglobin < 7 mg/dL
- LFTs > 3x ULN
- Previously experienced severe AEs from oral galactose (severe diarrhea, vomiting, constipation, galactosuria, or increased liver glycogen storage)
- Other history of galactose intolerance as determined by the investigator
- Currently treated with ketogenic diet
- Current enrollment in another trial involving investigational compounds
- Ongoing dietary D-galactose supplementation
- Use of investigational compounds
- Pregnancy
Treatment and study plan
AVTX-801
DrugMedical grade D-galactose
dosage:2.0 g/kg/day
Other names: D-Galactose
Placebo
DrugMatching placebo
Primary outcomes
-
Number of major motor seizure frequency
Time frame: 28 days
Seizure burden assessed by change from baseline in 28-day major motor seizure frequency
-
Number of vomiting episodes
Time frame: 28 days
Vomiting frequency in 28-day from baseline
-
Bristol Stool Form Scale (BSFS)
Time frame: average daily 28 days
Constipation/diarrhea assessed by scale improvement toward normal stool forms from baseline in average daily 28-day Bristol Stool form scale. The BSFS classifies stool into 7 groups, type 1 (hardest) to type 7 (softest). Type 3 and Type 4 would be more indicative of normal stool.
Study contacts
Contact information is provided by the study sponsor or research team.
Sponsors and collaborators
Lead sponsor
Eva Morava-Kozicz
Other
Collaborators
- Children's Hospital of Philadelphia
- National Institute of Neurological Disorders and Stroke (NINDS)
- Rare Diseases Clinical Research Network
Registry information
Official study title
Evaluation of Efficacy and Safety of D-galactose Supplementation in SLC35A2-CDG, a Disorder of Hypogalactosylation
Important dates
- Study start
- 2027
- Primary completion
- 2028
- Study completion
- 2028
- First posted
- Jun 2, 2022
- Registry last updated
- Jun 29, 2026
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.