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

Applied Therapeutics

Status and phase: Completed; Phase 3; Phase 2

Conditions: Classic Galactosemia

Treatments: Other: Placebo; Drug: AT-007

Study type: Interventional

Funder types: Industry

Identifiers

NCT04902781

AT-007-1002

Details and patient eligibility

About

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).

Full description

This is a randomized, double-blind, placebo-controlled study in pediatric subjects with CG. Children with CG ages 2 through 17 years will be enrolled. The study is designed to assess the clinical benefit using validated, standardized Clinical Outcome Assessments (COAs) 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 validated, standardized COAs.

Open-label extension (OLE) is an active treatment extension for patients who received placebo treatment in Part B.

Enrollment: 47 patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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47 participants in 2 patient groups, including a placebo group

Experimental: AT-007

Experimental group

Description:

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

Treatment:

Drug: AT-007

Placebo

Placebo Comparator group

Description:

Placebo given orally

Treatment:

Other: Placebo