Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry

Completed

• Conditions: Dyslipoproteinemias; Hypercholesterolemia, Familial; Familial Hypercholesterolemia - Homozygous; Familial Combined Hyperlipidemia
• Interventions: Drug: Standard lipid lowering therapy; Drug: PCSK9 Inhibitor [EPC]

• Registration Number: NCT03110432
• Lead Sponsor: GWT-TUD GmbH

Brief Summary

This is a prospective German registry for patients with dyslipidemia with very high cardiovascular risk who principally meet the Gemeinsamer Bundesausschuss (G-BA) stipulations for Proprotein convertase subtilisin/kexin like type 9 inhibitor (PCSK9i) use, and are treated by office-based cardiologists or in lipid ambulances.

Detailed Description

The study is purely observational, and will document data from the patient charts only. Treatment of patients will not be changed by this study, and all clinical decisions (including on frequency of visits) will be upon the discretion of the physician. No blood samples must be taken solely for the purpose of the study.

The registry will include two types of centers: 1) office-based cardiologists and 2) specialized lipid ambulances (outpatient departments).

Data on patient disease and treatment history will be collected a first documentation (retrospectively).

The documentation time is 3 years per patient. After the baseline visit, patients are followed-up every 6 ± 2months (last visit at month 36). This interval is considered narrow enough not to miss important events (safety reporting, cardiovascular events, hospitalizations).

Patients with stable (maintenance) lipid-lowering therapy (including those with existing PCSK9i therapy) or those with any therapy changes (including newly initiated PCSK9i treatment) will be documented in this study. Compared to the former group with stable drug treatment, the latter group will likely have major LDL-C changes during the first few weeks, which will be accounted for by (retrospective) monthly documentation in the first 3 months (data will be documented at the 6-month visit.

The documentation periods will be substantially longer than in the controlled studies of the PCSK9i (endpoints were as early as 3 months), and thus will provide much-needed information about the long-term effects on LDL-C and other lipid parameters, safety, and drug retention rates. Longer follow-up periods would likely be compromised by high rates of (administrative) discontinuation rates.

Study Timeline

• Study First Submitted: 2017-03-31
• Study First Submitted QC: 2017-04-06
• Study First Posted: 2017-04-12
• Study Start: 2017-05-18
• Primary Completion: 2024-04-17
• Study Completion: 2024-04-17
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-06
• Last Update Posted: 2025-05-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1695

Inclusion Criteria

• • with familial, homozygous hypercholesterolemia, in whom pharmaceutical and diet options for lipid lowering have proved insufficient, or

  • with confirmed familial, heterozygous hypercholesterolemia under consideration of the total familial risk, or

  • with heterozygous familial or non- familial hypercholesterolemia or mixed dyslipidemia with

    • therapy refractory course
    • maximal dietary and pharmaceutical lipid lowering therapy - in any case documented over a 12-month period
    • unsatisfactorily lowered LDL-C value (and thus with an indication for LDL apheresis)
    • confirmed vascular disease
    • other risk factors for cardiovascular events

Exclusion Criteria

• Concurrent participation of the patient in a clinical randomised study.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Standard lipid lowering therapy	Standard lipid lowering therapy	Statins, ezetimibe, nicotinic acid, fibrates, cholestagel, omega-3 fatty acids (and any combinations of these agents)
PCSK9 Inhibitor [EPC]	PCSK9 Inhibitor [EPC]	Evolocumab or alirocumab.

Primary Outcome Measures

Name	Time	Method
LDL cholesterol goal achievement	3 years	\< 70 mg/dl

Secondary Outcome Measures

Name	Time	Method
LDL cholesterol reduction	up top 3 years	Compared to baseline
Number of treatment changes	up to 3 years	During the follow-up period

Trial Locations

Locations (3)

Nephrologisches Zentrum; 🇩🇪 Villingen-Schwenningen, Germany

Klinik und Poliklinik für Kardiologie, Universitätsklinikum; 🇩🇪 Leipzig, Germany

Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum; 🇩🇪 Tübingen, Germany