Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry

NCT ID: NCT03110432

Last Updated: 2025-05-09

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 1695 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-05-18
• Study Completion Date: 2024-04-17

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.

Conditions

Dyslipoproteinemias; Hypercholesterolemia, Familial; Familial Hypercholesterolemia - Homozygous; Familial Combined Hyperlipidemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Standard lipid lowering therapy

Statins, ezetimibe, nicotinic acid, fibrates, cholestagel, omega-3 fatty acids (and any combinations of these agents)

Standard lipid lowering therapy

Intervention Type: DRUG

drug use according to the respective product labelling

PCSK9 Inhibitor [EPC]

Evolocumab or alirocumab.

PCSK9 Inhibitor [EPC]

Intervention Type: DRUG

drug use according to the respective product labelling

Interventions

PCSK9 Inhibitor [EPC]

drug use according to the respective product labelling

Intervention Type: DRUG

Standard lipid lowering therapy

drug use according to the respective product labelling

Intervention Type: DRUG

Other Intervention Names

Repatha, Praluent; Statins, ezetimibe, nicotinic acid, fibrates, cholestagel, omega-3 fatty acids

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

GWT-TUD GmbH

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David Pittrow, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

GWT-TUD GmbH, Germany

Andreas Birkenfeld, MD, PhD

Role: STUDY_CHAIR

Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum Tuebingen, Germany

Bernd Hohenstein, MD, PhD

Role: STUDY_CHAIR

Nephrologisches Zentrum Villingen-Schwenningen, Germany

Ulrich Laufs, MD, PhD

Role: STUDY_CHAIR

Klinik für Innere Medizin III, Universität des Saarlandes, Germany

Volker JJ Schettler, MD, PhD

Role: STUDY_CHAIR

Nephrologisches Zentrum Göttingen GbR, Germany

Elisabeth Steinhagen-Thiessen, MD, PhD

Role: STUDY_CHAIR

Lipid Clinic, Charité Universitaetsmedizin, Berlin, Germany

Klaus G Parhofer, MD, PhD

Role: STUDY_CHAIR

Ludwig Maximilian University, Medizinische Klinik und Poliklinik IV, Muenchen, Germany

Locations

Klinik und Poliklinik für Kardiologie, Universitätsklinikum

Leipzig, , Germany

Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum

Tübingen, , Germany

Nephrologisches Zentrum

Villingen-Schwenningen, , Germany

Countries

Germany

References

Parhofer KG, Pittrow D, Birkenfeld AL, Fraass U, Hohenstein B, Siegert C, Klotsche J, Steinhagen-Thiessen E, Dexl S, Schettler VJJ, Laufs U. Determinants of lipid lowering therapy intensification in very high risk patients with dyslipidaemia eligible for PCSK9 monoclonal antibodies: 1-year outcomes of the PERI-DYS study. Acta Cardiol. 2025 Jul;80(5):475-486. doi: 10.1080/00015385.2025.2490381. Epub 2025 Apr 24.

Reference Type: BACKGROUND

PMID: 40270128 (View on PubMed)

Laufs U, Birkenfeld AL, Fraass U, Hohenstein B, Siegert C, Klotsche J, Steinhagen-Thiessen E, Pittrow D, Dexl S, Salmen S, Schettler VJJ, Parhofer KG; Collaborators in the PERI-DYS Study. Novel Insights into the Management of Patients with Very High Cardiovascular Risk Eligible for PCSK9 Inhibitor Treatment: Baseline Findings from the PERI-DYS Study. Cardiovasc Drugs Ther. 2024 Feb;38(1):119-129. doi: 10.1007/s10557-022-07386-0. Epub 2022 Sep 30.

Reference Type: BACKGROUND

PMID: 36178485 (View on PubMed)

Parhofer KG, Pittrow D, Birkenfeld AL, Fraass U, Hohenstein B, Siegert C, Klotsche J, Steinhagen-Thiessen E, Dexl S, Schettler VJJ, Laufs U. Treatment persistence, lipid lowering, and 3-year clinical outcomes in patients at very high cardiovascular risk on PCSK9 monoclonal antibodies. Clin Res Cardiol. 2025 Aug 4. doi: 10.1007/s00392-025-02719-z. Online ahead of print.

Reference Type: DERIVED

PMID: 40760109 (View on PubMed)

Other Identifiers

PERI-DYS

Identifier Type: -

Identifier Source: org_study_id