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Traboulsi Syndrome; a retrospective Dutch cohort study

Expanding the genotype and phenotype of Traboulsi Syndrome; a retrospective Dutch cohort study - Expanding the genotype and phenotype of Traboulsi Syndrome; a retrospective Dutch cohort study

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
NL-OMON
Registry ID
NL-OMON57533
Enrollment
8
Registered
2025-05-12
Start date
2025-07-01
Completion date
Unknown
Last updated
2025-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Connective tissue disorders and aneurysm

Interventions

Not applicable, given the retrospective cohort study design.

Sponsors

Erasmus MC, Universitair Medisch Centrum Rotterdam
Lead Sponsor

Eligibility

Inclusion criteria

Inclusion criteria: Patients with Traboulsi syndrome and a proven (likely) pathogenic homozygous or compound heterozygous variants in the ASPH-gene. Informed consent about re-use of the patient data is given.

Exclusion criteria

Exclusion criteria: Objection of the re-use of their data.

Design outcomes

Primary

MeasureTime frame
Better knowledge of the clinical phenotype/natural history. A pseudo-anonymized digital database will be prepared containing specific features concerning Traboulsi Syndrome which will be assessed per patient.

Secondary

MeasureTime frame
- Demographics, including sex, age at disease presentation, age at genetic diagnosis (if applicable)- Description of cardiac and/or vascular phenotype- Description of the ocular phenotype- Description of the respiratory phenotype- Description of the skeletal phenotype- Description of the facial appearance- Information about the hematological status- Description of other connective tissue disorder features- Information about pregnancy and delivery (if applicable)

Countries

Netherlands

Contacts

Public ContactI.M.B.H van de Laar

Erasmus MC, Universitair Medisch Centrum Rotterdam

ervo@erasmusmc.nl0107036915

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)