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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
Must not have
Hemolytic uremic syndrome
Aldolase-B deficiency
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 28 days
Awards & highlights
All Individual Drugs Already Approved

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.
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.
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 below
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I have been diagnosed with SLC35A2-CDG.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have been diagnosed with hemolytic uremic syndrome.
Select...
I have Aldolase-B deficiency.
Select...
I have galactosemia.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~28 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Bristol Stool Form Scale (BSFS)
Number of major motor seizure frequency
Number of vomiting episodes

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: AVTX-801, then PlaceboExperimental Treatment2 Interventions
Each treatment period is 24 weeks, with 6-week washout period in between.
Group II: Placebo, then AVTX-801Placebo Group2 Interventions
Each treatment period is 24 weeks, with 6-week washout period in between.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,385 Previous Clinical Trials
652,678 Total Patients Enrolled
Rare Diseases Clinical Research NetworkNETWORK
68 Previous Clinical Trials
19,199 Total Patients Enrolled
Children's Hospital of PhiladelphiaOTHER
731 Previous Clinical Trials
8,472,824 Total Patients Enrolled
Eva Morava-KoziczLead Sponsor
3 Previous Clinical Trials
66 Total Patients Enrolled
Eva Morava-Kozicz, MD, PhDPrincipal InvestigatorIcahn School of Medicine at Mount Sinai
8 Previous Clinical Trials
1,209 Total Patients Enrolled

Media Library

AVTX-801 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05402384 — Phase 2
CDG Research Study Groups: AVTX-801, then Placebo, Placebo, then AVTX-801
CDG Clinical Trial 2023: AVTX-801 Highlights & Side Effects. Trial Name: NCT05402384 — Phase 2
AVTX-801 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05402384 — Phase 2
~7 spots leftby Aug 2026
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