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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 -3 in part b
Awards & highlights

Study Summary

This trial will study if AT-007 is safe and helpful for kids with Classic Galactosemia, a disorder where the body can't metabolize galactose.

Eligible Conditions
  • Classic Galactosemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 1- 3 in part b
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 1- 3 in part b for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
National Institute of Health Toolbox Cognition Battery
National Institute of Health Toolbox Motor Battery
Oral and Written Language Scales
+1 more
Secondary outcome measures
Behavioral Assessment System for Children
Biomarker Analysis
Biomarker Measure
+5 more
Other outcome measures
Bayley Scales of Infant and Toddler Development

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
~100

Find a Location

Who is running the clinical trial?

Applied Therapeutics, Inc.Lead Sponsor
4 Previous Clinical Trials
838 Total Patients Enrolled
Susan E Waisbren, PhDStudy ChairHarvard University
2 Previous Clinical Trials
20 Total Patients Enrolled
Jonathan W Mink, MDStudy ChairUniversity of Rochester

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people are signing up to participate in this experiment?

"This specific clinical trial is no longer recruiting patients. The study was posted on 3/20/2021 and the most recent update was 2/16/2022. Currently, there are 4 other trials related to classic galactosemia enrolling patients and 1 for AT-007."

Answered by AI

Which type of people would be best suited for this research project?

"This study is in need of 47 test subjects that have classic galactosemia and are aged 2-17. The following additional criteria must be met: Male, Female non-pregnant, Female non-lactating subjects aged ≥2 to <18 years., Male/Female with no significant health problems (other than classic Galactosemia), No other disease that would preclude participation in the study."

Answered by AI

Has this research been conducted before?

"Research on AT-007 started in 2021 with a clinical trial sponsored by Applied Therapeutics, Inc. The study had 47 participants and after its successful completion, AT-007 moved onto Phase 2 & 3 of drug approval. Currently, there is one active trial for AT-007 being conducted by Applied Therapeutics, Inc.."

Answered by AI

Are there any open appointment times for this test?

"This trial is no longer recruiting patients. The study was first posted on March 20th, 2021 and had its last update on February 16th, 2022. Currently, there are 4 clinical trials for classic galactosemia and 1 trial for AT-007 that are actively admitting patients."

Answered by AI

Does this research project have an age limit for participants?

"According to the age bracket that is eligible for this clinical trial, the minimum age requirement for enrollment is 2 years old while the maximum age limit is 17."

Answered by AI

What other medical research has been conducted using AT-007?

"Right now, there is 1 large-scale study and 1 smaller trial studying AT-007. Phase 3 trials are ongoing in Ann Arbor, Michigan as well as 2 other locations."

Answered by AI

Who else is applying?

What state do they live in?
Utah
How old are they?
< 18
What site did they apply to?
Children's Hospital Colorado
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
~12 spots leftby Mar 2025