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AVTX-801 for Congenital Disorder of Glycosylation
Phase 2
Waitlist Available
Led By Eva Morava-Kozicz, MD, PhD
Research Sponsored by Eva Morava-Kozicz
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Molecular diagnosis of SLC35A2-CDG
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
Study Summary
This trial tests a potential treatment to help people with a rare genetic disorder called SLC35A2-CDG. It will measure safety, effectiveness & comfort.
Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include digestive issues like diarrhea or constipation based on exclusion criteria related to past severe adverse events from oral galactose intake.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with SLC35A2-CDG.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in Alanine aminotransferase level
Change in Aspartate aminotransferase level
Change in the composite score of the Nijmegen Pediatric CDG Rating Scale
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Main study arm - AVTX-801Experimental Treatment1 Intervention
subjects will wash out from food grade D-galactose then be put on medical grade D-galactose
Group II: PlaceboPlacebo Group1 Intervention
placebo crossover
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Who is running the clinical trial?
Eva Morava-KoziczLead Sponsor
3 Previous Clinical Trials
67 Total Patients Enrolled
Eva Morava-Kozicz, MD, PhDPrincipal InvestigatorMayo Clinic
8 Previous Clinical Trials
860 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your hemoglobin level is less than 7 milligrams per deciliter.I have been diagnosed with hemolytic uremic syndrome.You are currently on a ketogenic diet.I have Aldolase-B deficiency.I have been diagnosed with SLC35A2-CDG.You had severe side effects from taking galactose by mouth in the past.You have a history of not being able to tolerate galactose, as determined by the doctor.Taking D-galactose supplements as part of your diet.I have galactosemia.
Research Study Groups:
This trial has the following groups:- Group 1: Main study arm - AVTX-801
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has the AVTX-801 treatment been granted clearance by the FDA?
"AVTX-801 has yet to receive any clinical evidence endorsing its efficacy, so it is assigned a score of 2 in regards to safety. This assessment was made by Power Inc., and based on the fact that this is still an early stage trial."
Answered by AI
Are there any spots available for new patients in this research endeavor?
"Sadly, clinicaltrials.gov suggests that this trial is not currently seeking new participants; the study was first published on January 1st 2024 and last amended on August 25th 2023. However, 5 other trials are actively recruiting at present."
Answered by AI
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