Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry (PERI-DYS)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Dyslipoproteinemias
• Intervention / Treatment: Drug: Standard lipid lowering therapy; Drug: PCSK9 Inhibitor [EPC]

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 Type: Observational
• Enrollment (Actual): 1713

Contacts and Locations

Study Locations

• Germany
  • Leipzig, Germany
    • Klinik und Poliklinik für Kardiologie, Universitätsklinikum
  • Tübingen, Germany
    • Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum
  • Villingen-Schwenningen, Germany
    • Nephrologisches Zentrum

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 100 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with dyslipidemia and very high cardiovascular risk ("highest risk patients" according to G-BA stipulation for PCSK9i use)

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Standard lipid lowering therapy; Statins, ezetimibe, nicotinic acid, fibrates, cholestagel, omega-3 fatty acids (and any combinations of these agents)	Drug: Standard lipid lowering therapy; drug use according to the respective product labelling; Other Names: Statins, ezetimibe, nicotinic acid, fibrates, cholestagel, omega-3 fatty acids
PCSK9 Inhibitor [EPC]; Evolocumab or alirocumab.	Drug: PCSK9 Inhibitor [EPC]; drug use according to the respective product labelling; Other Names: Repatha, Praluent

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
LDL cholesterol goal achievement	< 70 mg/dl	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
LDL cholesterol reduction	Compared to baseline	up top 3 years
Number of treatment changes	During the follow-up period	up to 3 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in quality of life	by EuroQol 5 dimensions	up to 3 years

Collaborators and Investigators

• Sponsor: GWT-TUD GmbH
• Investigators: Principal Investigator: David Pittrow, MD, PhD, GWT-TUD GmbH, Germany; Study Chair: Andreas Birkenfeld, MD, PhD, Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum Tuebingen, Germany; Study Chair: Bernd Hohenstein, MD, PhD, Nephrologisches Zentrum Villingen-Schwenningen, Germany; Study Chair: Ulrich Laufs, MD, PhD, Klinik für Innere Medizin III, Universität des Saarlandes, Germany; Study Chair: Volker JJ Schettler, MD, PhD, Nephrologisches Zentrum Göttingen GbR, Germany; Study Chair: Elisabeth Steinhagen-Thiessen, MD, PhD, Lipid Clinic, Charité Universitaetsmedizin, Berlin, Germany; Study Chair: Klaus G Parhofer, MD, PhD, Ludwig Maximilian University, Medizinische Klinik und Poliklinik IV, Muenchen, Germany

Publications and helpful links

General Publications

• 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. 2022 Sep 30. doi: 10.1007/s10557-022-07386-0. Online ahead of print.

Study record dates

Study Major Dates

• Study Start (Actual): May 18, 2017
• Primary Completion (Estimated): March 31, 2024
• Study Completion (Estimated): June 30, 2024

Study Registration Dates

• First Submitted: March 31, 2017
• First Submitted That Met QC Criteria: April 6, 2017
• First Posted (Actual): April 12, 2017

Study Record Updates

• Last Update Posted (Estimated): February 23, 2024
• Last Update Submitted That Met QC Criteria: February 22, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Lipid Metabolism Disorders; Dyslipidemias; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Enzyme Inhibitors; Antimetabolites; Protease Inhibitors; Micronutrients; Anticholesteremic Agents; Hypolipidemic Agents; Lipid Regulating Agents; Vitamins; Serine Proteinase Inhibitors; Vitamin B Complex; Nicotinic Acids; Niacin; Colesevelam Hydrochloride; Ezetimibe; PCSK9 Inhibitors
• Other Study ID Numbers: PERI-DYS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No