Status:
NOT_YET_RECRUITING
Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)
Lead Sponsor:
Eva Morava-Kozicz
Collaborating Sponsors:
Children's Hospital of Philadelphia
Conditions:
PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
Eligibility:
All Genders
18-60 years
Phase:
PHASE2
Brief 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.
Detailed Description
Participants who sign consent will be screened for enrollment, and if they meet all eligibility criteria, they will enter a 4-week Run-In period. During the Run-In period, participants will continue t...
Eligibility Criteria
Inclusion
- 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.
- Subject has any of the following:
- Liver failure
- ALT level \>8x ULN
- AST level \>8x ULN
- Use of investigational compounds within the previous 6 months or current enrollment in another trial involving investigational compounds.
- Subject is pregnant.
- Subject has hepatic impairment that would require a dose adjustment, defined by the site Principal Investigator.
- In the site Principal Investigator's opinion, subject is not able or willing to comply with the trial requirements.
Exclusion
Key Trial Info
Start Date :
February 2 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 2 2028
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT05402332
Start Date
February 2 2026
End Date
March 2 2028
Last Update
September 23 2025
Active Locations (1)
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1
Mayo Clinic Rochester
Rochester, Minnesota, United States, 55905