Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04902781
Other study ID # AT-007-1002
Secondary ID
Status Active, not recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date March 20, 2021
Est. completion date September 2023

Study information

Verified date February 2023
Source Applied Therapeutics, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


Description:

This is a randomized, double-blind, placebo-controlled study in pediatric subjects with CG. Three age groups (≥2 to ≤6-year olds, ≥7 to ≤12-year olds, and ≥13 to <18- year olds) will be enrolled. The study is designed to assess the clinical benefit (changes in how pediatric patients with CG feels and function, changes in ocular disorders, and changes in sexual maturation, growth and development) as well as the safety, PK, and PD (reduction of galactitol levels) of AT-007 in pediatric subjects with CG. The study consists of 2 main parts, Part A and Part B, and an open-label extension (OLE). Part A is an intra-patient dose escalation evaluating multiple ascending doses (MAD) of AT-007. Part B is designed to assess the clinical benefits of long-term administration of AT-007 (at the optimum dose identified in Part A) on how patients with CG feel and function. Open-label extension (OLE) is an active treatment extension for patients who received placebo treatment in Part B.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 47
Est. completion date September 2023
Est. primary completion date April 2023
Accepts healthy volunteers No
Gender All
Age group 2 Years to 17 Years
Eligibility Inclusion: - Male - Female non-pregnant - Female non-lactating subjects aged =2 to <18 years. - Diagnosis of Classic Galactosemia, confirmed by decreased GALT (galactose-1-phosphate uridyltransferase gut-associated lymphoid tissue )activity in erythrocytes, or a historical record of diagnosis of GALT (galactose-1-phosphate uridyltransferase gut-associated lymphoid tissue ) deficiency. Exclusion: - Male/Female with no significant health problems (other than classic Galactosemia) - No other disease that would preclude participation in the study.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
AT-007
Treatment with AT-007 given orally
Other:
Placebo
Placebo given orally

Locations

Country Name City State
United States University of Michigan Ann Arbor Michigan
United States Rare Disease Research Center Atlanta Georgia
United States Children's Hospital Colorado Aurora Colorado

Sponsors (1)

Lead Sponsor Collaborator
Applied Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Bayley Scales of Infant and Toddler Development Bayley Scales of Infant and Toddler Development (Bayley-4) showing a score of 40 -160 with higher values showing more development. Exploratory Endpoint Every 6 months in Part B
Primary National Institute of Health Toolbox Cognition Battery NIH Toolbox Cognition Battery is a multiple of tests to show Cognition and consists of tests of multiple constructs to yield individual ranking and demonstrate Cognitive Function Composite , Fluid Cognition Composite Sequence Memory, List Sorting, Pattern Comparison, and Crystallized Cognition Composite, Picture Vocabulary and Reading Recognition. This is standardized normal or 100 with a standard deviation of 10. Higher scores are better. Every 6 months in Part B
Primary National Institute of Health Toolbox Motor Battery NIH Toolbox Motor Battery is a multiple of tests to determine motor skills and is a comprehensive set of neuro-behavioral measurements that quickly assess cognitive, emotional, sensory, and motor functions from the convenience of an iPad. This is standardized normal or 100 with a standard deviation of 10. Higher scores are better. Every 6 months in Part B
Primary Vineland Adaptive Behaviour Scales Vineland Adaptive Behaviour Scales (Vineland-3) has a normal score of 100 and lower scores show developmental delay Every 6 months in Part B
Primary Oral and Written Language Scales Oral and Written Language Scales Every 6 months in Part B
Secondary Biomarker Measure This biomarker measure will be performed to assess the inhibition of galactitol Month 1- 3 in Part B
Secondary Biomarker Analysis This biomarker analysis will be performed to assess the levels of galactose and its other metabolites Month 1 -3 in Part B
Secondary Rate of Lens Opacity Onset and severity/progression of cataracts using the Lens Opacity System (LOCS) III, has a grading scale of 1-4, with 4 the most severe as assessed by the reviewer per standard criteria for anterior and posterior obstruction of the eye. Every 6 months in Part B
Secondary Sexual maturation in female patients as indicated by Tanner Stage Sexual maturation in female patients as indicated by Tanner Stage 1- 4 which is the ranking of physical development in children, adolescents and adults. This defines physical measurements of development based on external primary and secondary sex characteristics, such as the size of the breasts, and development of pubic hair. This is assessed by physical exam with Stage 1 per-pubescent and Stage 4 post pubescent. Every 6 months in Part B
Secondary Ovarian function in female patients determined by one hormonal test. Ovarian function in female patients is recorded by hormonal measures which can be one of the following;FSH (follicle stimulating hormone), estradiol and/or LH (luteinizing hormone) Every 6 months in Part B
Secondary Spiral Drawing Test Spiral Drawing Test is a multiple of tests showing drawing skills Every 6 months in Part B
Secondary Scale for Assessment & Rating of Ataxia Scale for Assessment & Rating of Ataxia (SARA) is 0-40, the higher the score the worse value Every 6 months in Part B
Secondary Behavioral Assessment System for Children Behavioral Assessment System for Children (BASC-3) has a normal range of 40 -060 with the higher score showing more difficulty. Every 6 months in Part B
See also
  Status Clinical Trial Phase
Active, not recruiting NCT05418829 - AT-007 in Adult Subjects With Classic Galactosemia (CG) Phase 3
Enrolling by invitation NCT03838016 - Preventing Speech and Language Disorders in Children With Classic Galactosemia N/A
Completed NCT04117711 - Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia Phase 1/Phase 2
Completed NCT03580122 - The Effect of Arginine on Classic Galactosemia Phase 2