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Status:

NOT_YET_RECRUITING

AVTX-801 D-galactose Supplementation in SLC35A2-CDG

Lead Sponsor:

Eva Morava-Kozicz

Collaborating Sponsors:

National Institute of Neurological Disorders and Stroke (NINDS)

Rare Diseases Clinical Research Network

Conditions:

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

Eligibility:

All Genders

1+ years

Phase:

PHASE2

Brief Summary

This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-CDG

Eligibility Criteria

Inclusion

  • Inclusion Criteria
  • Molecular confirmation of SLC35A2 genetic variant
  • Age \> 1 month
  • Presence of seizures, chronic vomiting, chronic constipation, or chronic diarrhea
  • 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.
  • Use of contraception in females \> age 8 years
  • Previously performed eye exam within last year
  • Exclusion Criteria
  • Aldolase-B deficiency
  • Galactosemia
  • Hemolytic uremic syndrome
  • Hemoglobin \< 7 mg/dL
  • LFTs \> 3x ULN
  • 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
  • Ongoing dietary D-galactose supplementation
  • Use of investigational compounds
  • Pregnancy

Exclusion

    Key Trial Info

    Start Date :

    January 1 2027

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ESTIMATED

    End Date :

    March 1 2028

    Estimated Enrollment :

    10 Patients enrolled

    Trial Details

    Trial ID

    NCT05402384

    Start Date

    January 1 2027

    End Date

    March 1 2028

    Last Update

    June 29 2025

    Active Locations (1)

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    Mayo Clinic Minnesota

    Rochester, Minnesota, United States, 55905