Williams SyndromePhase 2NCT06315699
Clemastine Fumarate in the Treatment of Neurodevelopmental Delays in Williams Syndrome
A Phase 2 study testing Clemastine Fumarate Tablets and corn starch tablets in people with Williams Syndrome and Child, run by Qilu Hospital of Shandong University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Williams Syndrome, Child, Neurodevelopmental Delay
- What is being tested?
- Clemastine Fumarate Tablets and corn starch tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 1 siteChina
- Study dates
- 20 Mar 2024to 30 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 3-12 years old;
- Positive fluorescence in situ hybridization (FISH) test confirmed Williams syndrome;
- GTF2I gene mutation was detected by whole exon;
- Heart safety variables are normal (e.g. normal ECG, blood pressure 120-129/80-84)
Who cannot join
- WS patients with other gene mutations;
- 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;
- Patients with narrow-angle glaucoma, narrow peptic ulcer, pyloroduodenal obstruction, symptomatic prostatic hypertrophy and bladder neck obstruction; Accompanied by severe immunodeficiency disease;
- Allergic to Clomastine fumarate or other arylalkylamine antihistamines or any receptor;
- According to the recent interpretation of MRI and neuroradiology experts or WS, there are obvious brain lesions that are not related to WS disease;
- 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;
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized, Double-blind, Controlled Study of Clomastine Fumarate in the Treatment of Williams Syndrome
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.
Read the 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
- Being tested
- Clemastine Fumarate Tablets, corn starch tablets
- Keywords
- clemastine fumarate, Williams syndrome, MRI
- Medical subject headings
- Williams Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clemastine Fumarate TabletsDrug
clemastine fumarate (0.178 mg/kg/day), three months
corn starch tabletsDietary supplement
The dose was administered 2mg once daily in a double-blind random crossover method
| Study group | Role | What they receive |
|---|---|---|
| Clemastine | Experimental | Phase1: Clemastine，tablet，0.178 mg/kg/day，three months; Phase2 placebo (corn starch tablets) three months Receives: Drug: Clemastine Fumarate Tablets |
| corn starch tablets | Placebo comparator | Phase1: placebo (corn starch tablets) three months; Phase2: Clemastine，tablet，0.178 mg/kg/day，three months Receives: Dietary Supplement: corn starch tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Anisotropy Score (FA)
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Measured: baseline follow-up；D90; D194
Radial diffusion rate (RD)
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Measured: baseline follow-up；second month；fourth month
axial diffusivity (AD)
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Measured: baseline follow-up；D90；D194
mean diffusivity (MD)
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Measured: baseline follow-up；D90; D194
Peabody（Motion Estimation Timewarp）score
Assessing motion skills
Measured: baseline follow-up；second month；fourth month
Gesell Development Scale
Assessing neurodevelopment
Measured: baseline follow-up；second month；fourth month
quotients (IQs) of the WISC-IV
quantify intelligence quotients (IQs)
Measured: baseline; D90 ; D194
Secondary outcomes (7)
Differential pressure across valves
Measuring by Cardiac color ultrasound
Measured: baseline follow-up；second month；fourth month
Thyroid hormone value
Measured: baseline follow-up；second month；fourth month
Conners Parent Symptoms Questionnaire Score
Assessing adaptability
Measured: baseline follow-up；second month；fourth month
Vailand-3 scale
Assessing neurodevelopment
Measured: baseline follow-up；second month；fourth month
CSHQ Children's Sleep Habits Questionnaire Score
Assessing sleeping
Measured: baseline follow-up；second month；fourth month
SRS Score 2(Social Response Scale2) score
Assessing social skills
Measured: baseline follow-up；D90; D194
Score of the Chinese Communicative Development Inventories
Measured: baseline; D90; D194
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2024
- Study start20 Mar 2024
- Primary completion2 Mar 2025
- Study completion30 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Williams SyndromeStudies of the same condition, open and under way.
- More trials run by Qilu Hospital of Shandong UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Qilu Hospital of Shandong University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- cao aihua, post-doctoral, Principal investigator, Qilu Hospital of Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06315699
- Sponsor's protocol ID
- QL000001
- Other IDs
- MR-37-24-002019
- Status last verified
- Mar 2024
- First submitted
- 4 Mar 2024
- Last updated
- 19 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-blind, Controlled Study of Clomastine Fumarate in the Treatment of Williams Syndrome
- ClinicalTrials.gov ID
- NCT06315699
- Acronym / sponsor ID
- QL000001
- Conditions
- Williams Syndrome, Child, Neurodevelopmental Delay
- Keywords
- clemastine fumarate, Williams syndrome, MRI
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Qilu Hospital of Shandong UniversityUniversity, hospital or other
- Study start
- 20 Mar 2024 (actual)
- Primary completion
- 2 Mar 2025 (actual)
- Study completion
- 30 Dec 2025 (actual)
- First posted
- 18 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Clemastine | Experimental | Drug: Clemastine Fumarate TabletsPhase1: Clemastine，tablet，0.178 mg/kg/day，three months; Phase2 placebo (corn starch tablets) three months |
| corn starch tablets | Placebo comparator | Dietary Supplement: corn starch tabletsPhase1: placebo (corn starch tablets) three months; Phase2: Clemastine，tablet，0.178 mg/kg/day，three months |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age 3-12 years old;
- Positive fluorescence in situ hybridization (FISH) test confirmed Williams syndrome;
- GTF2I gene mutation was detected by whole exon;
- Heart safety variables are normal (e.g. normal ECG, blood pressure 120-129/80-84)
Key exclusion criteria (6 listed)
- WS patients with other gene mutations;
- 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;
- Patients with narrow-angle glaucoma, narrow peptic ulcer, pyloroduodenal obstruction, symptomatic prostatic hypertrophy and bladder neck obstruction; Accompanied by severe immunodeficiency disease;
- Allergic to Clomastine fumarate or other arylalkylamine antihistamines or any receptor;
- According to the recent interpretation of MRI and neuroradiology experts or WS, there are obvious brain lesions that are not related to WS disease;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clemastine Fumarate Tabletsclemastine fumarate (0.178 mg/kg/day), three months | Drug | Clemastine |
| corn starch tabletsThe dose was administered 2mg once daily in a double-blind random crossover method | Dietary supplement | corn starch tablets |
Primary outcome
Anisotropy Score (FA)
Time frame: baseline follow-up；D90; D194
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Radial diffusion rate (RD)
Time frame: baseline follow-up；second month；fourth month
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
axial diffusivity (AD)
Time frame: baseline follow-up；D90；D194
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
mean diffusivity (MD)
Time frame: baseline follow-up；D90; D194
Measuring by Magnetic resonance diffusion tensor imaging (DTI)
Peabody（Motion Estimation Timewarp）score
Time frame: baseline follow-up；second month；fourth month
Assessing motion skills
Gesell Development Scale
Time frame: baseline follow-up；second month；fourth month
Assessing neurodevelopment
quotients (IQs) of the WISC-IV
Time frame: baseline; D90 ; D194
quantify intelligence quotients (IQs)
7 secondary outcomes
Differential pressure across valves
Time frame: baseline follow-up；second month；fourth month
Thyroid hormone value
Time frame: baseline follow-up；second month；fourth month
Conners Parent Symptoms Questionnaire Score
Time frame: baseline follow-up；second month；fourth month
Vailand-3 scale
Time frame: baseline follow-up；second month；fourth month
CSHQ Children's Sleep Habits Questionnaire Score
Time frame: baseline follow-up；second month；fourth month
SRS Score 2(Social Response Scale2) score
Time frame: baseline follow-up；D90; D194
Score of the Chinese Communicative Development Inventories
Time frame: baseline; D90; D194
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2026.
- Individual participant dataSharing planned: No
Related trials for Williams Syndrome
RecruitingObservational study
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Rare Disorders, Undiagnosed Disorders
Recruiting
Characterization of the Natural History of Microduplication Syndrome 7q11.23
7q11.23 Microduplication Syndrome (7DUP), Autism Spectrum Disorder (ASD)
Enrolling by invitation
Parent and Infant Inter(X)Action Intervention (PIXI)
Fragile X Syndrome, Angelman Syndrome
RecruitingObservational study
Characterization and Natural History of Williams Syndrome and Other Chromosome 7q11.23 Variants
Williams Beuren Syndrome, Williams Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
cao aihua, post-doctoral principal investigator
Qilu Hospital of Shandong University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clemastine Fumarate Tablets and corn starch tablets in people with Williams Syndrome and Child, run by Qilu Hospital of Shandong University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Anisotropy Score (FA) (time frame: baseline follow-up；D90; D194). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2026.