Not yet recruiting NCT05402384
AVTX-801 D-galactose Supplementation in SLC35A2-CDG
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AVTX-801, Placebo.
- Who it may be relevant to
- Registry conditions: SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation. Basic parameters: from 1 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Evaluation of Efficacy and Safety of D-galactose Supplementation in SLC35A2-CDG, a Disorder of Hypogalactosylation
Overview
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
Interventions
- Drug AVTX-801
Medical grade D-galactose dosage:2.0 g/kg/day - Drug Placebo
Matching placebo
Primary outcome measures
- Number of major motor seizure frequency [Time frame: 28 days]
- Number of vomiting episodes [Time frame: 28 days]
- Bristol Stool Form Scale (BSFS) [Time frame: average daily 28 days]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Crossover
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Mayo Clinic Minnesota — Rochester
Identifiers
NCT: NCT05402384 · 19-005271 · U54NS115198