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D-galactose for Congenital Disorder of Glycosylation (AVTX-801 Trial)

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

Subject is currently on a stable dose of D-galactose therapy.

Subject is aged 18 to 60 years.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 16 weeks

Awards & highlights

AVTX-801 Trial Summary

This trial will explore if D-galactose can help treat PGM1-CDG. Your participation is needed for potential progress.

Who is the study for?

Adults aged 18-60 with PGM1-CDG who are already on a stable D-galactose therapy can join. They must understand the study and agree to use effective birth control if necessary. People with certain metabolic disorders, liver failure, recent other trial participation, or severe reactions to galactose cannot participate.Check my eligibility

What is being tested?

The study is testing AVTX-801 for people with PGM1-CDG who take D-galactose. It aims to find the best dose and assess long-term safety of this treatment in adults currently using D-galactose.See study design

What are the potential side effects?

While specific side effects of AVTX-801 aren't listed here, similar treatments may cause digestive issues like diarrhea or vomiting, changes in urine composition (galactosuria), and increased liver glycogen storage.

AVTX-801 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am on a consistent dose of D-galactose therapy.

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I am between 18 and 60 years old.

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I have been diagnosed with PGM1-CDG through genetic testing.

AVTX-801 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 16 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~16 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 16 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Time (days) to rescue treatment from randomization

AVTX-801 Trial Design

1Treatment groups

Experimental Treatment

Group I: AVTX-801Experimental Treatment1 Intervention

1.5g/kg/day AVTX-801

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Eva Morava-KoziczLead Sponsor

3 Previous Clinical Trials

78 Total Patients Enrolled

Eva Morava-Kozicz, MD, PhDPrincipal InvestigatorMayo Clinic

8 Previous Clinical Trials

871 Total Patients Enrolled

Media Library

AVTX-801 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05402332 — Phase 2

Congenital Disorder of Glycosylation Research Study Groups: AVTX-801

Congenital Disorder of Glycosylation Clinical Trial 2023: AVTX-801 Highlights & Side Effects. Trial Name: NCT05402332 — Phase 2

AVTX-801 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05402332 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the cutoff age for enrollment in this experiment below fifty-five years?

"This clinical trial seeks volunteers aged 18 to 60; those younger and older have respective trials available for them, with 6 total in the program."

Answered by AI

Are there any dangers associated with utilizing AVTX-801 for treatment?

"AVTX-801 has been deemed moderately secure, receiving a score of 2. This is because it is currently in the second phase of clinical trials and there are reports that suggest safety but none to support its efficacy."

Answered by AI

Is this trial taking on new participants?

"Clinicaltrials.gov indicates that this research endeavour, which was initially issued on November 1st 2023 and last updated on August 25th 2023, is no longer recruiting participants. Nonetheless, 5 other trials can still be found actively enrolling people at the moment."

Answered by AI

Could I be considered a viable candidate for this research endeavor?

"This clinical trial is looking for 9 volunteers between the ages of 18 and 60 who have been diagnosed with PGM1-CDG. Further, these participants must meet a variety of additional requirements: they should be aged 6 months to 60 years; possess blood work data from before beginning unregulated D galactose therapy; comprehend and sign an informed consent document (or assent if applicable); not currently lactating nor pregnant; use double barrier contraception during participation in the study as well as 30 days after their last dose of medication; and men are expected to ensure that their partner also employs acceptable contraceptive practices throughout this period."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Evaluate Optimal Dosing and Long-term Safety of D-galactose in PGM1-CDG

Eva Morava-Kozicz 2024. "Evaluate Optimal Dosing and Long-term Safety of D-galactose in PGM1-CDG". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05402332.