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New treatment option for Cornelia De Lange Syndrome
Official title Cornelia De Lange Syndrome: Assessing Positive Effects of Lithium Treatment
ClinicalTrials.gov ID: NCT06789783
What this study is testing
What is Lithium Carbonate Capsule?
Lithium Carbonate Capsule is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for cornelia de lange syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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
- Written consent signed by parent/legal guardian/representative prior to the screening visit
You likely can't join if
- The patient is participating in another clinical trial
- QT interval prolongation, thyroid dysfunction, renal insufficiency, hepatic insufficiency, leukopenia, or other currently clinically significant...
- QTcF interval on ECG greater than 450 msec
- Severe diabetes mellitus or inherited metabolic disorder
See the full eligibility 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
- Written consent signed by parent/legal guardian/representative prior to the screening visit
- Ability to take the study drug provided in capsules or drops (for younger patients and those with swallowing difficulties) or combined with food/drinks
- Caregiver must be able to understand the instructions and participate knowingly in the study
- The patient is participating in another clinical trial
- QT interval prolongation, thyroid dysfunction, renal insufficiency, hepatic insufficiency, leukopenia, or other currently clinically significant medical disorders (as determined by the investigator), other than those...
- QTcF interval on ECG greater than 450 msec
- Severe diabetes mellitus or inherited metabolic disorder
The study team makes the final eligibility decision.
Where it's taking place
- Milan, MI, Italy
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Milan, MI, Italy. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.