Observational prospective study to evaluate the efficacy of Vitamin K Intravenously on the coagulopathy in patients with peroxisomal biogenesis disorders (PBD)

Observational prospective study to evaluate the efficacy of Vitamin K Intravenously on the coagulopathy in patients with peroxisomal biogenesis disorders (PBD) - VitKId

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON42020

Enrollment

Registered

2015-03-18

Start date

2015-04-20

Completion date

Unknown

Last updated

2024-04-23

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

Conditions

congenital metabolic diseases Peroxisomal biogenesis disorder zellweger syndrome spectrum

Interventions

Before administration of vitamin K, blood will be collected for determination of baseline level of APTT, PT, FV, FVII, PIVKA, thrombocytes, d-dimer fibrinogen and Calibrated Automated Thrombogram (C

Eligibility

Age

2 Years to 17 Years

Inclusion criteria

Inclusion criteria: 1. Patients with PBD over five years of age 2. Coagulopathy with prolonged PT, low FVII concentration and increased PIVKA level due to vitamin K deficiency.

Exclusion criteria

Exclusion criteria: 1. No oral or written informed consent of the parents. 2. No coagulopathy as measured by PT.

Design outcomes

Primary

Measure	Time frame
The lab results of APTT, PT, FV, FVII, fibrinogen, d-dimer, thrombocytes and PIVKA will be shown descriptively and in scatter plots before and after iv vitamin K.	—

Secondary

Measure	Time frame
Calibrated Automated Thrombogram (CAT) (= thrombin generation assay) will also be presented desciptively.	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)