Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE2
10 participants
INTERVENTIONAL
2027-01-31
2028-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
TRIPLE
Study Groups
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AVTX-801, then Placebo
Each treatment period is 24 weeks, with 6-week washout period in between.
AVTX-801
Medical grade D-galactose
dosage:2.0 g/kg/day
Placebo
Matching placebo
Placebo, then AVTX-801
Each treatment period is 24 weeks, with 6-week washout period in between.
AVTX-801
Medical grade D-galactose
dosage:2.0 g/kg/day
Placebo
Matching placebo
Interventions
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AVTX-801
Medical grade D-galactose
dosage:2.0 g/kg/day
Placebo
Matching placebo
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
1 Month
ALL
No
Sponsors
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National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Rare Diseases Clinical Research Network
NETWORK
Children's Hospital of Philadelphia
OTHER
Eva Morava-Kozicz
OTHER
Responsible Party
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Eva Morava-Kozicz
Principal Investigator
Principal Investigators
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Eva Morava-Kozicz, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Icahn School of Medicine at Mount Sinai
Locations
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Mayo Clinic Minnesota
Rochester, Minnesota, United States
Countries
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Central Contacts
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Other Identifiers
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19-005271
Identifier Type: -
Identifier Source: org_study_id
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