Familial HypercholesterolemiaFH-RegistryNCT06571630
PMMHRI - Familial Hypercholesterolemia Registry
An observational study of Familial Hypercholesterolemia, run by Polish Mother Memorial Hospital Research Institute at 1 site in Poland.
Trial summary
The registry is maintained at the Regional Centre for Rare Diseases, established in 2016, within Polish Mother's Memorial Hospital Research Institute. This facility diagnoses and treats over 80 distinct rare diseases in patients from across the country, including those with phenotypically or genetically confirmed… Read more about this study
- Who is it for?
- Ages 1 day and older
- Conditions the study is for
- Familial Hypercholesterolemia
- How many people
- 300 participants
- Where
- 1 sitePoland
- Study dates
- 1 Jan 2018to 31 Dec 2025
- How long will I take part?
- 1 Year
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All consecutive patients diagnosed with FH, including both children and adults,
Who may be able to join
- FH diagnosis
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
Poland1 site, 1 recruiting
- Recruiting
Polish Mother Memorial Research Institute (PMMHRI) in Lodz, Poland
Lodz, 93-338
Maciej Banach, MD,PhD,FNLA,FAHA,FESC,FASA, +48 42 2711124, maciej.banach@icloud.com
Joanna Lewek, MD, PhD, +48 422721593, joanna.lewek@iczmp.edu.pl
About this study
Official title: Polish Mother's Memorial Hospital Research Institute - Familial Hypercholesterolemia Registry
The registry is maintained at the Regional Centre for Rare Diseases, established in 2016, within Polish Mother's Memorial Hospital Research Institute. This facility diagnoses and treats over 80 distinct rare diseases in patients from across the country, including those with phenotypically or genetically confirmed familial hypercholesterolemia (FH).
Read the detailed description
All consecutive patients diagnosed with FH, including both children and adults, are being enrolled from December 2018 to May 2024. Each patient receives a consultation with a clinical geneticist and a dietitian, who provide recommendations for cascade screening among family members and promote a healthy and balanced diet. The study includes all index cases, with children also serving as index cases.
The PMMHRI FH registry is managed by physicians from the RCRD, who exclusively make all diagnostic and treatment decisions. The purpose of the registry is to investigate the clinical characteristics, diagnosis, management, achievement of therapeutic goals, and outcomes of FH patients. Inclusion criteria are based on phenotypic or genetic diagnosis of FH. Phenotypic diagnosis is conducted using the Dutch Lipid Clinic Network criteria for adults, and the Simon Broome criteria for children. During the baseline visit, comprehensive data collection includes all available lipidogram results, aiming to obtain naive LDL-C values, highest values, and treated values. All data collected during the enrollment period are included in the study.
- Conditions
- Familial Hypercholesterolemia
- Keywords
- Familial Hypercholesterolemia
- Medical subject headings
- Hyperlipoproteinemia Type II
What is being measured
The primary outcome is the main question the study is designed to answer.
Therapeutic goals achivement
For each patient, we collect data on the levels of all lipid profile fractions: such as total cholesterol [mg/dl], low density lipoprotein cholesterol - LDL [mg/dl], high density lipoprotein cholesterol HDL [mg/dl], triglycerides [mg/dl], non-HDL [mg/dl] and lipoprotein(a) during both dual and triple therapy. Additionally, we will assess the achievement of the therapeutic target as defined by the ESC guidelines. The therapeutic target for each patient will be evaluated individually based on their risk, in accordance with the ESC guidelines.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2024
- Study start1 Jan 2018
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Familial HypercholesterolemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 300 participants (estimated)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Polish Mother Memorial Hospital Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06571630
- Sponsor's protocol ID
- PMMHRI-FH-Registry
- Acronym
- FH-Registry
- Status last verified
- Aug 2024
- First submitted
- 8 Aug 2024
- Last updated
- 26 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Lewek J, Konopka A, Starostecka E, Penson PE, Maciejewski M, Banach M. Clinical Features of Familial Hypercholesterolemia in Children and Adults in EAS-FHSC Regional Center for Rare Diseases in Poland. J Clin Med. 2021 Sep 22;10(19):4302. doi: 10.3390/jcm10194302.
PubMed 34640319European Atherosclerosis Society Familial Hypercholesterolaemia Studies Collaboration. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study. Lancet. 2024 Jan 6;403(10421):55-66. doi: 10.1016/S0140-6736(23)01842-1. Epub 2023 Dec 12.
PubMed 38101429EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Global perspective of familial hypercholesterolaemia: a cross-sectional study from the EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Lancet. 2021 Nov 6;398(10312):1713-1725. doi: 10.1016/S0140-6736(21)01122-3. Epub 2021 Sep 7.
PubMed 34506743
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Polish Mother's Memorial Hospital Research Institute - Familial Hypercholesterolemia Registry
- ClinicalTrials.gov ID
- NCT06571630
- Acronym / sponsor ID
- FH-Registry / PMMHRI-FH-Registry
- Conditions
- Familial Hypercholesterolemia
- Keywords
- Familial Hypercholesterolemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Polish Mother Memorial Hospital Research InstituteUniversity, hospital or other
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 26 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The registry is maintained at the Regional Centre for Rare Diseases, established in 2016, within Polish Mother's Memorial Hospital Research Institute. This facility diagnoses and treats over 80 distinct rare diseases in patients from across the country, including those with phenotypically or genetically confirmed familial hypercholesterolemia (FH).
Detailed description
All consecutive patients diagnosed with FH, including both children and adults, are being enrolled from December 2018 to May 2024. Each patient receives a consultation with a clinical geneticist and a dietitian, who provide recommendations for cascade screening among family members and promote a healthy and balanced diet. The study includes all index cases, with children also serving as index cases.
The PMMHRI FH registry is managed by physicians from the RCRD, who exclusively make all diagnostic and treatment decisions. The purpose of the registry is to investigate the clinical characteristics, diagnosis, management, achievement of therapeutic goals, and outcomes of FH patients. Inclusion criteria are based on phenotypic or genetic diagnosis of FH. Phenotypic diagnosis is conducted using the Dutch Lipid Clinic Network criteria for adults, and the Simon Broome criteria for children. During the baseline visit, comprehensive data collection includes all available lipidogram results, aiming to obtain naive LDL-C values, highest values, and treated values. All data collected during the enrollment period are included in the study.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Target follow-up
- 1 Year
Who may qualify
- Ages
- 1 day and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All consecutive patients diagnosed with FH, including both children and adults,
Key inclusion criteria (1 listed)
- FH diagnosis
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Therapeutic goals achivement
Time frame: 5 years
For each patient, we collect data on the levels of all lipid profile fractions: such as total cholesterol [mg/dl], low density lipoprotein cholesterol - LDL [mg/dl], high density lipoprotein cholesterol HDL [mg/dl], triglycerides [mg/dl], non-HDL [mg/dl] and lipoprotein(a) during both dual and triple therapy. Additionally, we will assess the achievement of the therapeutic target as defined by the ESC guidelines. The therapeutic target for each patient will be evaluated individually based on their risk, in accordance with the ESC guidelines.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2024.
- Background: PubMed 34640319Lewek J, Konopka A, Starostecka E, Penson PE, Maciejewski M, Banach M. Clinical Features of Familial Hypercholesterolemia in Children and Adults in EAS-FHSC Regional Center for Rare Diseases in Poland. J Clin Med. 2021 Sep 22;10(19):4302. doi: 10.3390/jcm10194302.
- Background: PubMed 38101429European Atherosclerosis Society Familial Hypercholesterolaemia Studies Collaboration. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study. Lancet. 2024 Jan 6;403(10421):55-66. doi: 10.1016/S0140-6736(23)01842-1. Epub 2023 Dec 12.
- Background: PubMed 34506743EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Global perspective of familial hypercholesterolaemia: a cross-sectional study from the EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Lancet. 2021 Nov 6;398(10312):1713-1725. doi: 10.1016/S0140-6736(21)01122-3. Epub 2021 Sep 7.
- Individual participant dataSharing planned: Undecided
Related trials for Familial Hypercholesterolemia
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
RecruitingPhase 1
Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
Hypercholesterolemia, Hypertriglyceridemia
Active, not recruiting
Clinical Decision Support for Familial Hypercholesterolemia
Hypercholesterolemia, Familial, Clinical Decision Support
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Familial Hypercholesterolemia, run by Polish Mother Memorial Hospital Research Institute at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Maciej Banach, MD,PhD,FNLA,FAHA,FESC,FASA (+48 42 2711124, maciej.banach@icloud.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Therapeutic goals achivement (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06571630, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2024.