Clemastine Fumarate in the Treatment of Neurodevelopmental Delays in Williams Syndrome

Last updated: March 11, 2024
Sponsor: Qilu Hospital of Shandong University
Overall Status: Active - Recruiting

Phase

3

Condition

Williams Syndrome

Treatment

Clemastine Fumarate Tablets

fructose

Clinical Study ID

NCT06315699
QL000001
MR-37-24-002019
  • Ages 3-6
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Age 3-6 years old;
  2. Positive fluorescence in situ hybridization (FISH) test confirmed Williams syndrome;
  3. GTF2I gene mutation was detected by whole exon;
  4. Heart safety variables are normal (e.g. normal ECG, blood pressure 120-129/80-84)

Exclusion

Exclusion Criteria:

  1. WS patients with other gene mutations;
  2. Used antihistamines, monoamine oxidase inhibitors, barbiturates and sedatives, as wellas drugs affecting cognitive behavior, limb movement, white matter myelin, and MRIwithin 2 months before enrollment;
  3. Patients with narrow-angle glaucoma, narrow peptic ulcer, pyloroduodenal obstruction,symptomatic prostatic hypertrophy and bladder neck obstruction; Accompanied by severeimmunodeficiency disease;
  4. Allergic to Clomastine fumarate or other arylalkylamine antihistamines or anyreceptor;
  5. According to the recent interpretation of MRI and neuroradiology experts or WS, thereare obvious brain lesions that are not related to WS disease;
  6. Clinically significant metabolic, hematological, liver, immune, urinary, endocrine,neurological, pulmonary, psychiatric, skin, allergic, renal, or other major diseasesthat may affect the interpretation of study findings or patient safety in WS'sjudgment;

Study Design

Total Participants: 50
Treatment Group(s): 2
Primary Treatment: Clemastine Fumarate Tablets
Phase: 3
Study Start date:
March 20, 2024
Estimated Completion Date:
December 30, 2025

Study 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 an open-label study with 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.

Connect with a study center

  • Qilu Hospital of Shandong University

    Tainan, Shangdong 250012
    China

    Active - Recruiting

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