Clemastine Fumarate in the Treatment of Neurodevelopmental Delays in Williams Syndrome

Launched by QILU HOSPITAL OF SHANDONG UNIVERSITY · Mar 11, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating the use of Clemastine Fumarate to help children with Williams syndrome, a condition that can affect learning, movement, and social skills. The main goal is to see if this medication can improve brain function by fixing certain issues with the brain’s myelin, which is the protective layer around nerve fibers. The study is currently looking for participants who are between 3 and 6 years old and have been diagnosed with Williams syndrome through specific genetic testing.

To join the trial, children must have a confirmed GTF2I gene mutation and normal heart health. However, there are some reasons that would prevent a child from participating, such as having other gene mutations or certain health conditions. For those who are eligible, taking part in the trial means they will receive the medication and be closely monitored for both effectiveness and safety. This study is an important step in finding new treatment options for children with Williams syndrome.

Gender

ALL

Eligibility criteria

• 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 Criteria:
• • 1. WS patients with other gene mutations;
• • 2. Used antihistamines, monoamine oxidase inhibitors, barbiturates and sedatives, as well as drugs affecting cognitive behavior, limb movement, white matter myelin, and MRI within 2 months before enrollment;
• • 3. Patients with narrow-angle glaucoma, narrow peptic ulcer, pyloroduodenal obstruction, symptomatic prostatic hypertrophy and bladder neck obstruction; Accompanied by severe immunodeficiency disease;
• • 4. Allergic to Clomastine fumarate or other arylalkylamine antihistamines or any receptor;
• • 5. According to the recent interpretation of MRI and neuroradiology experts or WS, there are 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 diseases that may affect the interpretation of study findings or patient safety in WS's judgment;