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NCT07454343 · ClinicalTrials.gov registry record
ECD-Score: a Study on Erdheim-Chester Disease
A clinical trial of Erdheim-Chester Disease (ECD), sponsored by Meyer Children's Hospital IRCCS.
- Recruiting
- Registry status
- 1,000
- Enrollment target
- 7
- Study locations
NCT07454343 is a study of Erdheim-Chester Disease (ECD) that is actively recruiting participants, run by Meyer Children's Hospital IRCCS. The registered enrollment target is 1,000 participants. The trial reports 7 study locations across 7 states.
The verdict
NCT07454343, a study of Erdheim-Chester Disease (ECD), is actively recruiting participants, sponsored by Meyer Children's Hospital IRCCS.
- RECRUITING
- Registry status
- 1,000 participants
- Enrollment target
- 7
- Study locations
Study Summary
Erdheim-Chester disease (ECD) is a rare form of non-Langerhans cell histiocytosis that primarily affects adults but may also occur in pediatric patients. It is characterized by the accumulation of foamy histiocytes with a distinctive immunophenotype in multiple anatomical sites, most commonly the long bones, retroperitoneal and perirenal tissues, the heart, the central nervous system, and the pituitary gland. The disease shows marked clinical heterogeneity, ranging from localized and asymptomatic forms to severe manifestations with multiorgan involvement. From a pathogenetic perspective, ECD is mainly driven by gain-of-function mutations affecting the MAPK and PI3K-AKT pathways, particularly the BRAFV600E mutation, leading to aberrant activation of the MAPK and mTOR signaling pathways. The release of pro-inflammatory cytokines and chemokines plays a key role in systemic inflammation and tissue damage, resulting in significant complications and disability depending on the organs involved. Despite the significant efforts of international research in recent years, particularly given the extreme rarity of the disease (incidence below 5 cases per 10,000,000 adults per year), substantial knowledge gaps remain, especially with regard to the prediction of long-term outcomes, both in terms of survival and disability. Although some prognostic factors associated with survival have already been identified (such as central nervous system involvement), to date only limited-scale studies have systematically evaluated the prognosis of patients with ECD, focusing in particular on factors influencing organ-specific complications. Moreover, in clinical practice, several aspects that significantly affect patients' quality of life tend to be underestimated, partly due to the time required to perform comprehensive assessments using detailed questionnaires designed to quantify disease-related consequences, such as chronic disability, depression, and cognitive impairment. Nevertheless, th
Conditions Studied
Study Locations (7)
Maryland
- National Institute of Health - Bethesda
Minnesota
- Mayo Clinic - Rochester
New York
- Memorial Sloan Kettering Cancer Center - New York
Other
- Hopital Pitiè-Salpetriere - Paris
Fi
- Meyer Children's Hospital IRCCS, Firenze - Florence
Italy
- San Raffaele Hospital - Milan
Newcastel
- Newcastle Upon Tyne Hospitals NHS Foundation Trust - Newcastle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1,000 participants |
| Start Date | 2024-12-23 |
| Est. Completion | 2028-12 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07454343
The ClinicalTrials.gov registry entry for NCT07454343 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 1,000 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Meyer Children's Hospital IRCCS, which has 6 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Erdheim-Chester Disease (ECD) appearing as the primary indexed condition, and to 0 interventions.
NCT07454343 reports 7 study locations spanning 7 distinct geographic areas - top geographies include Maryland, Minnesota, New York.
Frequently Asked Questions
What is clinical trial NCT07454343 about?
NCT07454343 is a clinical study titled "ECD-Score: a Study on Erdheim-Chester Disease". Erdheim-Chester disease (ECD) is a rare form of non-Langerhans cell histiocytosis that primarily affects adults but may also occur in pediatric patients. It is characterized by the accumulation of foamy histiocytes with a distinctive immunophenotype in multiple anatomical sites, most commonly the lo...
What is the current status of trial NCT07454343?
This trial is currently recruiting. The enrollment target is 1,000 participants. The study started on 2024-12-23. Estimated completion is 2028-12.
What conditions does trial NCT07454343 study?
This clinical trial studies the following conditions: Erdheim-Chester Disease (ECD).
Who is sponsoring clinical trial NCT07454343?
This trial is sponsored by Meyer Children's Hospital IRCCS, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07454343 being conducted?
This trial has 7 study locations across Maryland, Minnesota, New York, Fi, Italy. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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