Not Yet RecruitingPhase 2

Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)

United States8 participantsStarted 2026-10-01

Plain-language summary

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-galactose.

Who can participate

Age range18 Years – 60 Years

SexALL

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Inclusion criteria

✓. Subject is aged 18 to 60 years.
✓. Subject has biologically and genetically proven PGM1-CDG.
✓. Subject is currently on a stable dose of D-galactose therapy.
✓. Non-pregnant, non-lactating female subjects of childbearing potential who are heterosexually active and non-sterile male subjects with female sexual partners of childbearing potential agree to use a highly effective method of contraception for the duration of the study, including the long-term safety follow-up period. A highly effective method of birth control is defined as one that results in a low failure rate (i.e., \<1% per year) when used consistently and correctly, such as oral/injectable/inserted/implanted/transdermal contraceptives, condom with diaphragm, condom with spermicide, diaphragm with spermicide, intrauterine hormone- releasing system, or intrauterine device (IUD), or sexual abstinence. Contraception is not required where at least 6 weeks have passed since sterilization, defined as females having undergone one of the following surgeries: hysterectomy, bilateral tubal ligation or occlusion, bilateral oophorectomy, or bilateral salpingectomy; and males who are vasectomized. Contraception is not required where females are postmenopausal (defined as 12 consecutive months of spontaneous amenorrhea and age ≥51 years).
✓. Subject/legally authorized representative (LAR) is able to understand and provide written informed consent, and assent (as applicable) to participate in this study.

Exclusion criteria

✕. Subject has aldolase B deficiency, galactosemia, hemolytic uremic syndrome, or severe anemia.
✕. In the site Principal Investigator's opinion, subject has a history of galactose intolerance that precludes the subject from participation in this study.
✕. In the site Principal Investigator's opinion, subject has previously experienced any of the following severe AEs from oral galactose:
✕. Severe diarrhea
✕. Severe, recurrent vomiting
✕. Constipation
✕. Galactosuria
✕. Increased liver glycogen storage.

What they're measuring

Proportion of participants with at least one PGM1-CDG related event

Timeframe: after each treatment period (treatment periods are 18 weeks)

Trial details

NCT IDNCT05402332

SponsorEva Morava-Kozicz

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-03-02

Contact for this trial

Mary Freeman, MS, CGC

212-659-1434 mary.freeman@mssm.edu

View on ClinicalTrials.gov More PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation trials