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AT-007 for Galactosemia

Phase 2 & 3

Waitlist Available

Research Sponsored by Applied Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up month 1, month 2, month 3, month 6, month 12, and month 18

Summary

This trial is testing a new medicine called AT-007 in children with Classic Galactosemia, a genetic disorder. The medicine aims to lower harmful levels of a substance called galactitol in their bodies. Researchers hope this will improve the children's health and development.

Who is the study for?

This trial is for boys and girls aged 2 to under 18 with Classic Galactosemia, confirmed by specific blood tests or past medical records. Participants must not have other significant health issues besides Galactosemia, and girls must not be pregnant or breastfeeding.

What is being tested?

The study is testing AT-007 against a placebo to see if it's safe and effective in reducing harmful substances in the body (galactitol levels) of kids with Classic Galactosemia. It will also look at how the body processes the drug (PK/PD).

What are the potential side effects?

Specific side effects are not listed, but generally, participants will be monitored for any adverse reactions to AT-007 compared to those receiving a placebo.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 1, month 2, month 3, month 6, month 12, and month 18

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~month 1, month 2, month 3, month 6, month 12, and month 18

This trial's timeline: 3 weeks for screening, Varies for treatment, and month 1, month 2, month 3, month 6, month 12, and month 18 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Global Statistical Test (GST)

Sensitivity Analysis of the Primary Endpoint (GST) with Cognition

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

Secondary study objectives

Archimedes Spiral Drawing Test

Behavioral Assessment System for Children-3 [Individual Components]

Galactitol Level

+7 more

Other study objectives

Bayley Scales of Infant and Toddler Development-4 [Exploratory]

Biomarker Analysis [Exploratory]

Vineland Adaptive Behavior Scales-3 [Exploratory]

Side effects data

From 2021 Phase 1 & 2 trial • 114 Patients • NCT04117711

17%

Infections/Infestations

100%

80%

60%

40%

20%

Study treatment Arm

Part A Cohort 1: AT-007 0.5mg/kg

Part B & C Cohort 2: AT-007 10mg/kg

Part D Cohort 1: AT-007 5mg/kg

Part A Cohort 2: AT-007 5mg/kg

Part A Cohort 4: AT-007 20mg/kg

Part A Cohort 3: AT-007 10mg/kg

Part A Cohort 5: 40mg/kg

Part B & C Cohort 3: AT-007 20mg/kg

Part A Placebo Comparator

Part D Placebo Comparator

Part B & C Cohort 1: AT-007 5mg/kg

Part B & C Cohort 4: AT-007 40mg/kg

Part B & C Placebo Comparator

Part D Cohort 2: AT-007 20mg/kg

Part D Extension Placebo Comparator

Part D Cohort 3: AT-007 40mg/kg

Part D Extension Cohort 1: 20mg/kg

Part D Extension Cohort 2: 40mg/kg

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Experimental: AT-007Experimental Treatment1 Intervention

AT-007 The starting dose in Part A will be 5 mg/kg for all age groups. For each age group, Part B of the study will not start until the optimum dose evaluated in Part A has been identified

Group II: PlaceboPlacebo Group1 Intervention

Placebo given orally

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

AT-007

2019

Completed Phase 2

~120

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