AVTX-801 D-galactose Supplementation in SLC35A2-CDG

SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation Clinical Trial

Official title:

Evaluation of Efficacy and Safety of D-galactose Supplementation in SLC35A2-CDG, a Disorder of Hypogalactosylation

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05402384
• Other study ID #: 19-005271
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: January 2024
• Est. completion date: January 2025

Study information

• Verified date: August 2023
• Source: Mayo Clinic
• Contact: Clinical Genomics Research Team
• Phone: 507-293-1139
• Email: CGResearch[@]mayo.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a multicenter, open-label, withdrawal and treatment study assessing the efficacy, safety, and tolerability of CERC-801 in subjects with SLC35A2-CDG.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: January 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria

• Molecular diagnosis of SLC35A2-CDG

• A parent or legal guardian must be available and willing to provide consent on behalf of minor subjects or adult subjects who are unable to give informed consent due to developmental disabilities.

Exclusion Criteria

• Aldolase-B deficiency

• Galactosemia

• Hemolytic uremic syndrome

• Hemoglobin < 7 mg/dL

• Previously experienced severe AEs from oral galactose (severe diarrhea, vomiting, constipation, galactosuria, or increased liver glycogen storage)

• Other history of galactose intolerance as determined by the investigator

• Currently treated with ketogenic diet

• Current enrollment in another trial involving investigational compounds

• Dietary D-galactose supplementation

• Use of investigational compounds

• Pregnancy

Related Conditions & MeSH terms

• Congenital Disorders of Glycosylation
• SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation

Intervention

Drug:
• AVTX-801
Medical grade D-galactose
• Placebo
Placebo

Locations

Country	Name	City	State
United States	Mayo Clinic Minnesota	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Eva Morava-Kozicz

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in the composite score of the Nijmegen Pediatric CDG Rating Scale	Changes in the composite Nijmegen Pediatric CDG Rating Scale (NPCRS) score from post washout baseline to 12 months	12 months
Primary	Change in Alanine aminotransferase level	• Change in the key clinical laboratory parameters alanine aminotransferase [ALT], aspartate aminotransferase [AST]) from post-washout baseline to 12 months	12 months
Primary	Change in Aspartate aminotransferase level	• Change in the key clinical laboratory parameters alanine aminotransferase [ALT], aspartate aminotransferase [AST]) from post-washout baseline to 12 months	12 months

External Links

•   Mayo Clinic Clinical Trials