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

Completed

• Conditions: congenital metabolic diseases; Peroxisomal biogenesis disorder; zellweger syndrome spectrum; 10064477; 10021605

• Registration Number: NL-OMON42020
• Lead Sponsor: Kinderhematologie

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 5

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The lab results of APTT, PT, FV, FVII, fibrinogen, d-dimer, thrombocytes and<br /><br>PIVKA will be shown descriptively and in scatter plots before and after iv<br /><br>vitamin K.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Calibrated Automated Thrombogram (CAT) (= thrombin generation assay) will also<br /><br>be presented desciptively. </p><br>