NCT06315699

Clemastine Fumarate in the Treatment of Neurodevelopmental Delays in Williams Syndrome

Randomized, Double-blind, Controlled Study of Clomastine Fumarate in the Treatment of Williams Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Qilu Hospital of Shandong University · Academic / Other
• Sex: All
• Age: 3 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

This study focuses on therapeutic targets for cognitive, motor, and social impairments in Williams syndrome by reversing brain myelin defects caused by GTF2I. The primary objective of the study was to test and evaluate the initial efficacy and safety of Clomastine fumarate in the treatment of Williams syndrome.

Detailed description

The primary objective of this study was to evaluate the initial efficacy and safety of Clomastine fumarate in the treatment of Williams syndrome. The secondary objective is to study Clomastine fumarate in relation to mechanisms of action, safety, and/or pathological mechanisms. This study was a randomized, cross-over, placebo-controlled design. Each participant will be randomly assigned to two groups through baseline assessment (see study results), with Group A receiving the FDA-approved drug Clemastine at a weight-dependent dose (see dosing table below) for the first cycle and placebo for the second cycle. Group B will be treated with placebo for the first cycle and the FDA-approved drug Clemastine for the second cycle.

Conditions

• Williams Syndrome
• Child
• Neurodevelopmental Delay

Interventions

Type	Name	Description
DRUG	Clemastine Fumarate Tablets	clemastine fumarate (0.178 mg/kg/day), three months
DIETARY_SUPPLEMENT	corn starch tablets	The dose was administered 2mg once daily in a double-blind random crossover method

Timeline

• Start date: 2024-03-20
• Primary completion: 2025-03-02
• Completion: 2025-12-30
• First posted: 2024-03-18
• Last updated: 2026-03-19

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study