Clemastine Fumarate for Treating Neurodevelopmental Delays in Williams Syndrome

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

This study is testing if a medication called Clemastine Fumarate can help young children with Williams syndrome improve their thinking, movement, and social skills.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 3 Years to 6 Years
• Sex: All
• Sponsor: Qilu Hospital of Shandong University (Academic / other)
• Locations: 1 site (Tainan, Shangdong)
• Trial ID: NCT06315699 on ClinicalTrials.gov

What this trial studies

This study evaluates the efficacy and safety of Clemastine Fumarate in children aged 3-6 years with Williams syndrome, focusing on cognitive, motor, and social impairments. It employs an open-label, randomized, cross-over, placebo-controlled design, where participants receive either the drug or a placebo in two cycles. The study aims to reverse brain myelin defects associated with the condition and assess the drug's mechanisms of action and safety profile.

Who should consider this trial

Good fit: Ideal candidates are children aged 3-6 years with a confirmed diagnosis of Williams syndrome and specific genetic mutations.

Not a fit: Patients with other gene mutations or significant comorbid conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve cognitive and social functioning in children with Williams syndrome.

How similar studies have performed: While this approach is novel in the context of Williams syndrome, similar studies targeting neurodevelopmental delays have shown promise.

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;

Where this trial is running

Tainan, Shangdong

• Qilu Hospital of Shandong University — Tainan, Shangdong, China (Recruiting)

Study contacts

• Principal investigator: cao aihua, post-doctoral — Qilu Hospital of Shandong University
• Study coordinator: cao aihua, post-doctoral

Conditions Williams Syndrome; Child; Neurodevelopmental Delay; clemastine fumarate; Williams syndrome; MRI