AVTX-801 D-galactose Supplementation in SLC35A2-CDG

Eva Morava-Kozicz

Status and phase: Begins enrollment this month; Phase 2

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

Treatments: Drug: AVTX-801; Drug: Placebo

Study type: Interventional

Funder types: Other; NETWORK; NIH

Identifiers

NCT05402384

19-005271

U54NS115198 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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

Enrollment: 10 estimated patients

Sex: All

Ages: 1 month to 40 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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10 participants in 2 patient groups, including a placebo group

AVTX-801, then Placebo

Experimental group

Description:

Each treatment period is 24 weeks, with 6-week washout period in between.

Treatment:

Drug: Placebo

Drug: AVTX-801

Placebo, then AVTX-801

Placebo Comparator group

Description:

Each treatment period is 24 weeks, with 6-week washout period in between.

Treatment:

Drug: Placebo

Drug: AVTX-801