Clinical trials: Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
This is a single-arm, open-label study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by SHR-1918 in patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- NCT06009393
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 26 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
Homozygous Familial Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
Homozygous Familial Hypercholesterolemia
Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
A multicenter, single arm, open label phase II clinical study evaluating the long-term efficacy and safety of SHR-1918 in homozygous familial hypercholesterolemia patients
- Trial name
- NCT07133815
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
Dyslipoproteinemias, Hypercholesterolemia, Familial
Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry
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…
- Conditions
- Dyslipoproteinemias, Hypercholesterolemia, Familial, Familial Hypercholesterolemia - Homozygous, Familial Combined Hyperlipidemia
- Trial name
- PERI-DYS (NCT03110432)
- Testing
- PCSK9 Inhibitor [EPC], Standard lipid lowering therapy
- Where
- 3 sites in Germany
- Volunteers
- 1,695 people
- Sponsor
- GWT-TUD GmbH
Familial Hypercholesterolemia, Familial Hypercholesterolemia - Heterozygous
Early Detection of Familial Hypercholesterolemia in Children
Heterozigous FH is an underdiagnosed disease in the paediatric population. Its early detection, would allow us to initiate lifestyle therapeutical changes and early pharmacological therapy if necessary.
- Conditions
- Familial Hypercholesterolemia, Familial Hypercholesterolemia - Heterozygous, Familial Hypercholesterolemia - Homozygous
- Trial name
- DECOPIN (NCT04370899)
- Testing
- lifestyle assessment
- Where
- 2 sites in Spain
- Volunteers
- 400 people
- Sponsor
- Institut Investigacio Sanitaria Pere Virgili
Homozygous Familial Hypercholesterolemia
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objectives of the study are: To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH) To evaluate the long-term safety and tolerability of…
- Trial name
- NCT03409744
- Testing
- evinacumab
- Where
- 38 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
This is an open-label study designed to evaluate the long-term safety and efficacy of evinacumab, a fully human ANGPTL3 antibody, in patients with homozygous familial hypercholesterolemia (HoFH), in a real-life setting in Canada.
- Trial name
- NCT05611528
- Testing
- Evinacumab
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Daniel Gaudet
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- NCT04233918
- Testing
- Evinacumab
- Where
- 11 sites in United States, Australia and 4 more
- Volunteers
- 20 people
- Sponsor
- Regeneron Pharmaceuticals
Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
- Trial name
- HAUSER-OLE (NCT02624869)
- Testing
- Evolocumab
- Where
- 46 sites in United States, Australia and 21 more
- Volunteers
- 163 people
- Sponsor
- Amgen
Homozygous Familial Hypercholesterolemia HoFH
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
- Trial name
- RAMAN (NCT03403374)
- Testing
- evolocumab
- Where
- 12 sites in India
- Volunteers
- 30 people
- Sponsor
- Amgen
Severe Familial Hypercholesterolemia
Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
- Trial name
- TAUSSIG (NCT01624142)
- Testing
- Evolocumab
- Where
- 44 sites in United States, Australia and 16 more
- Volunteers
- 300 people
- Sponsor
- Amgen
ASCVD, Elevated Cholesterol
Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C
The purpose of this extension study was to evaluate the efficacy, safety, and tolerability of long-term dosing of Inclisiran. The study was a global multicenter study.
- Conditions
- ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
- Trial name
- ORION-8 (NCT03814187)
- Testing
- Inclisiran Sodium
- Where
- 238 sites in United States, Canada and 11 more
- Volunteers
- 3,275 people
- Sponsor
- Novartis Pharmaceuticals
Homozygous Familial Hypercholesterolemia
A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia
A study to evaluate safety and efficacy of IBI306 in subjects with homozygous familial hypercholesterolemia.
- Trial name
- NCT04031742
- Testing
- IBI306
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Homozygous Familial Hypercholesterolemia (HoFH)
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia…
- Trial name
- NCT02651675
- Testing
- AAV directed hLDLR gene therapy
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 9 people
- Sponsor
- REGENXBIO Inc.
Homozygous Familial Hypercholesterolemia
Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks [Q4W]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH…
- Trial name
- NCT04034485
- Testing
- lerodalcibep, evolocumab
- Where
- 12 sites in United States, India and 4 more
- Volunteers
- 65 people
- Sponsor
- LIB Therapeutics LLC
Homozygous Familial Hypercholesterolemia (HoFH)
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease.
- Trial name
- NCT04080050
- Testing
- Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 8 people
- Sponsor
- REGENXBIO Inc.
Homozygous Familial Hypercholesterolemia
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double…
- Trial name
- NCT03933293
- Testing
- AK102, Statins, Ezetimibe
- Where
- 2 sites in China
- Volunteers
- 10 people
- Sponsor
- Akeso
Homozygous Familial Hypercholesterolemia
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- ORION-5 (NCT03851705)
- Testing
- Inclisiran Sodium for injection
- Where
- 13 sites in Hong Kong, Israel and 6 more
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Hypercholesterolemia, Familial Hypercholesterolemia
A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00704444
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 11,332 people
- Sponsor
- Organon and Co
Hypercholesterolemia, Familial Hypercholesterolemia
A 52-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05245)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00705211
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 1,794 people
- Sponsor
- Organon and Co