Cornelia De Lange Syndrome: Assessing Positive Effects of Lithium Treatment
Recruiting
Phase 2
Phase 3
Interventional Study
Cornelia De Lange Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 and older
- Sex
- Any
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 34 (estimated)
- Sponsor
- University of Milan · Other
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About This Trial
The study "Cornelia de Lange Syndrome: assessing positive effects of Lithium treatment - CLoSER" aims to evaluate the effectiveness on behavioral modifications of lithium carbonate therapy in patients with Cornelia de Lange syndrome (CdLS).
CdLS is a rare genetic disease caused by autosomal mutations dominant or X-linked. The prevalence of CdLS is estimated to be between 1:10,000-30,000 newborns,…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Age \> 4 years
* Body weight within the normal range in the reference range for CdLS, based on age and height
* Diagnosis of CdLS based on consensus clinical criteria and a confirmed mutation in the NIPBL gene
* Stable drug regimen for 4 weeks prior to starting the study
* Wri…
Contacts