Clinical trials: Families
Familial Chylomicronemia
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT03544060
- Testing
- Volanesorsen
- Sponsor
- Akcea Therapeutics
NTRK Family Gene Mutation
Registry for Molecular Testing, Treatment and Outcome of Patients With Solid Tumors Harboring a NTRK Gene Fusion
The objective of this registry is to analyze treatment reality and outcome of patients with locally advanced or metastatic solid tumors harboring a fusion of NTRK1, NTRK2 or NTRK3
- Trial name
- REALTRK (NCT04557813)
- Where
- 26 sites in Germany, Switzerland
- Volunteers
- 88 people
- Sponsor
- iOMEDICO AG
Dyslipidemias, Familial Hypercholesterolemia
Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial…
- Conditions
- Dyslipidemias, Familial Hypercholesterolemia, Hypertriglyceridemia
- Trial name
- NCT03747224
- Testing
- ARO-ANG3, sterile normal saline (0.9% NaCl)
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia
Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease
LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy.
- Conditions
- Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia, Hypercholesterolemia
- Trial name
- NCT06112327
- Where
- 3 sites in New Zealand, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Verve Therapeutics, Inc.
Academic Support for Black Middle School Student Success in STEM
STEM As I Am Phase I
Black students experience significant achievement gaps that contribute to underrepresentation in high paying STEM fields and disparity in health and economic outcomes.
- Trial name
- STEM AIM (NCT07447661)
- Testing
- Technology-based educational intervention
- Volunteers
- 138 people, healthy volunteers welcome
- Sponsor
- Oregon Research Behavioral Intervention Strategies, Inc.
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
LOWER: Lomitapide Observational Worldwide Evaluation Registry
This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.
- Trial name
- LOWER (NCT02135705)
- Testing
- Lomitapide
- Where
- 71 sites in United States, Argentina and 6 more
- Volunteers
- 260 people
- Sponsor
- Amryt Pharma
Amyloidosis, Hereditary
ATTR Expanded Access Program (EAP) by Ionis
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
- Trial name
- NCT03400098
- Testing
- Inotersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Mediterranean Fever (FMF )
A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF
The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF).
- Trial name
- NCT06666335
- Testing
- Anakinra
- Sponsor
- Swedish Orphan Biovitrum
Amyotrophic Lateral Sclerosis
A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
- Trial name
- NCT03981536
- Testing
- AP-101
- Where
- 3 sites in Canada
- Volunteers
- 18 people
- Sponsor
- AL-S Pharma
Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG)
Compassionate Use of Evinacumab
Provide compassionate use of evinacumab
- Conditions
- Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG), Dysbetalipoproteinemia (DBL)
- Trial name
- NCT06500598
- Testing
- Evinacumab
- Sponsor
- Regeneron Pharmaceuticals
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Heterozygous Familial Hypercholesterolemia, Mixed Dyslipidemia
Efficacy and Safety of Fluvastatin in Children With Heterozygous Familial Hypercholesterolemia
The purpose of the study is to assess the safety and efficacy of fluvastatin in children diagnosed with heterozygous familial hypercholesterolemia
- Trial name
- NCT00171236
- Testing
- Fluvastatin
- Where
- Switzerland
- Volunteers
- 84 people
- Sponsor
- Novartis
Hypercholesterolemia, Familial Hypercholesterolemia
A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008)
The purpose of this study is to evaluate the efficacy and safety of enclitide chloride, an oral PCSK9 inhibitor, in lowering low-density lipoprotein cholesterol (LDL-C) in participants with hypercholesterolemia.
- Trial name
- NCT05261126
- Testing
- Enclitide Chloride
- Where
- 63 sites in United States, Germany and 6 more
- Volunteers
- 381 people
- Sponsor
- Merck Sharp & Dohme LLC
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Familial Hypercholesterolemia
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.
- Trial name
- NCT00943306
- Testing
- lomitapide
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 19 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Ewing Family of Tumors, Rhabdomyosarcoma
PK/PD of XM22 in Children With Ewing Family of Tumors or Rhabdomyosarcoma
This is a Phase I, open label study aimed at assessing the pharmacokinetics, pharmacodynamics, the efficacy, safety, and tolerability of a single injection of XM22 in children with Ewing family of tumors or rhabdomyosarcoma…
- Trial name
- NCT01585649
- Testing
- Lipegfilgrastim
- Where
- 18 sites in Bulgaria, Czechia and 4 more
- Volunteers
- 21 people
- Sponsor
- Merckle GmbH
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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
PFIC - Progressive Familial Intrahepatic Cholestasis
Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
Progressive familial intrahepatic cholestasis (PFIC) is a rare inherited liver disease that causes a build-up of bile acids in the liver.
- Trial name
- OvEC-PFIC (NCT07497724)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Ipsen