AVTX-801 for Congenital Disorder of Glycosylation
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called AVTX-801 for individuals with a rare genetic condition known as SLC35A2-CDG. The goal is to determine if the treatment reduces symptoms such as seizures, chronic vomiting, and digestive issues. Participants will receive both the treatment and a placebo (a substance with no active drug) at different times to compare effects. This trial suits those with a confirmed SLC35A2 genetic variant who experience symptoms affecting daily life, like frequent seizures or chronic digestive problems. Participants need a recent eye exam and, if unable to consent, a parent or guardian must provide consent. As a Phase 2 trial, researchers focus on measuring the treatment's effectiveness in an initial, smaller group of people.
Do I need to stop taking my current medications to join the trial?
The trial information does not specify whether you need to stop taking your current medications. However, if you are on a ketogenic diet or using investigational compounds, you may not be eligible to participate.
Is there any evidence suggesting that AVTX-801 is likely to be safe for humans?
Research has shown that AVTX-801, a treatment for certain genetic disorders, is generally well-tolerated. In an earlier study, healthy volunteers who took AVTX-801 experienced no serious side effects, suggesting it is relatively safe for humans. AVTX-801, a form of D-galactose (a type of sugar), has been tested for similar conditions with good safety results. While these findings are encouraging, ongoing studies continue to assess its safety in people with specific genetic disorders.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Congenital Disorder of Glycosylation, which often involve dietary supplements and supportive care, AVTX-801 offers a new approach by targeting the underlying glycosylation process. Researchers are excited about AVTX-801 because it acts on a specific pathway to improve glycosylation, potentially addressing the root cause of the disorder rather than just managing symptoms. This novel mechanism could lead to more effective and lasting improvements in patient health.
What evidence suggests that AVTX-801 might be an effective treatment for SLC35A2-CDG?
Research shows that AVTX-801, a type of D-galactose supplement, might help treat SLC35A2-CDG, a rare genetic disorder. Studies suggest that D-galactose can improve symptoms by enhancing the body's ability to process sugars. In patients with similar conditions, D-galactose has eased symptoms. Although specific data for SLC35A2-CDG is limited, the treatment's mechanism suggests potential benefits. Participants in this trial will receive AVTX-801 during one treatment period and a placebo during another, with each period lasting 24 weeks and a 6-week washout period in between. This treatment has received special recognition for its potential in treating rare childhood diseases, highlighting its promise.12346
Who Is on the Research Team?
Eva Morava-Kozicz, MD, PhD
Principal Investigator
Icahn School of Medicine at Mount Sinai
Are You a Good Fit for This Trial?
This trial is for individuals with a rare genetic disorder called SLC35A2-CDG. Participants must have a confirmed molecular diagnosis of this condition. The study welcomes minors and adults with developmental disabilities if consent is provided by guardians. It excludes those with galactose intolerance, other specific metabolic conditions, pregnancy, low hemoglobin levels, severe reactions to oral galactose, or current participation in another drug trial.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AVTX-801 or placebo for 24 weeks, followed by a 6-week washout period, then crossover to the alternate treatment for another 24 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AVTX-801
- Placebo
Trial Overview
The study tests the effectiveness and safety of AVTX-801 (CERC-801), which is D-galactose supplementation compared to a placebo in patients with SLC35A2-CDG. This multicenter trial involves an initial open-label phase where all participants receive AVTX-801 followed by a withdrawal period to assess changes when the supplement is stopped.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Each treatment period is 24 weeks, with 6-week washout period in between.
Each treatment period is 24 weeks, with 6-week washout period in between.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Eva Morava-Kozicz
Lead Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Rare Diseases Clinical Research Network
Collaborator
Children's Hospital of Philadelphia
Collaborator
Citations
1.
trial.medpath.com
trial.medpath.com/clinical-trial/faaca73b82dad323/nct05402384-evaluation-efficacy-safety-d-galactose-slc35a2-cdgAVTX-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...
Treatment of congenital disorders of glycosylation
The present paper aims to provide a detailed overview of these exciting developments and the rising concepts that are used to treat these ultra-rare diseases.
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 ...
Cerecor Announces Positive Phase I Safety Data for CERC ...
CERC-801 has been granted Orphan Drug Designation and awarded Rare Pediatric Disease Designation by the FDA, granting eligibility for receipt ...
Evaluating the Efficacy and Safety of D-galactose in PGM1 ...
This is a clinical trial to evaluate the efficacy of AVTX-801 (D-galactose) on the clinical manifestations of PGM1-CDG in participants currently taking D- ...
Congenital Disorder of Glycosylation, Type in (CDG1N)
Congenital disorders of glycosylation (CDGs) are a group of inherited metabolic disorders caused by enzymatic defects in glycoprotein synthesis.
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