CompletedPhase 2/3

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

United States47 participantsStarted 2021-03-20

Plain-language summary

This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Classic Galactosemia (CG).

Who can participate

Age range2 Years – 17 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion: * Male * Female non-pregnant * Female non-lactating subjects aged ≥2 to \<18 years. * Diagnosis of Classic Galactosemia, confirmed by \<1% GALT (galactose-1-phosphate uridyltransferase) enzyme activity in erythrocytes, or a historical record of diagnosis of \<1% GALT enzyme activity. Exclusion: * Male/Female with no significant health problems (other than classic Galactosemia) * No other disease that would preclude participation in the study.

What they're measuring

Global Statistical Test (GST)

Timeframe: Month 6, Month 12 and Month 18

Sensitivity Analysis of the Primary Endpoint (GST) with Cognition

Timeframe: Month 6, Month 12 and Month 18

Sensitivity Analysis of the Primary Endpoint (GST) with Fine Motor Skills

Timeframe: Month 6, Month 12 and Month 18

Trial details

NCT IDNCT04902781

SponsorApplied Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-03-03

View on ClinicalTrials.gov More Classic Galactosemia trials