Clinical trial • Phase III • Rare Disease|Endocrinology
ISIS 678354 SODIUM SALT for Familial chylomicronemia syndrome (FCS)
Phase III trial of ISIS 678354 SODIUM SALT for Familial chylomicronemia syndrome (FCS). open-label, none/not specified-controlled. 30 participants.
Overview
- Trial Therapeutic Area
- Rare Disease|Endocrinology
- Trial Disease
- Familial chylomicronemia syndrome (FCS)
- Trial Stage
- Phase III
- Drug Modality
- Oligonucleotide
Key dates
- Initial CTIS Submission Date
- 17-07-2024
- First CTIS Authorization Date
- 12-08-2024
Trial design
open-label, none/not specified-controlled Phase III trial across 14 sites in Sweden, France, Italy and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 30
- Trial Duration For Participant
- 1463
Eligibility
Recruits 30 Vulnerable population selected (isVulnerablePopulationSelected = true). Informed consent will be obtained using subject information sheets and informed consent forms (L1 ICF documents) provided in country-specific versions and multiple languages (examples in the record include English, Spanish, French, Italian, Dutch, Portuguese, Swedish, Slovak)..
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). Informed consent will be obtained using subject information sheets and informed consent forms (L1 ICF documents) provided in country-specific versions and multiple languages (examples in the record include English, Spanish, French, Italian, Dutch, Portuguese, Swedish, Slovak).
Inclusion criteria
- {"criterion_text":"- Satisfactory completion of the ISIS 678354 CS3 index study (last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement"}
- {"criterion_text":"- Willing to follow a diet comprising ≤ 20 g fat per day during the study"}
Exclusion criteria
- {"criterion_text":"- Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- The primary objective is to evaluate safety and tolerability of treatment with olezarsen","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Percent change in fasting TG from Baseline at Month 6 (average of Weeks 23, 25 and 27)","definition_or_measurement_approach":"Percent change in fasting TG from Baseline at Month 6 (average of Weeks 23, 25 and 27)"}
- {"endpoint_text":"- Percent change in fasting TG from Baseline at Month 12 (average of Week 51 and 53), Month 24 (average of Week 103 and Week 105) and Month 36 (average of Week 155 and Week 157), and Month 48 (average of Week 205 and 209)","definition_or_measurement_approach":"Percent change in fasting TG from Baseline at specified months using averages of specified weeks (Month 12: Weeks 51 & 53; Month 24: Weeks 103 & 105; Month 36: Weeks 155 & 157; Month 48: Weeks 205 & 209)"}
- {"endpoint_text":"- Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Proportion of patients with ≥40% reduction in fasting TG from Baseline measured at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Percent change in fasting apoB48 from Baseline at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Percent change in fasting apoB48 from Baseline measured at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Percent change in fasting apoC-III from Baseline at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Percent change in fasting apoC-III from Baseline measured at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Percent change in fasting non-HDL from Baseline at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Percent change in fasting non-HDL-C from Baseline measured at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Proportion of patients who achieve fasting TG 880mg/dL at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Proportion of patients achieving fasting TG ≤ 880 mg/dL at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105, 157 or 209)","definition_or_measurement_approach":"Adjudicated acute pancreatitis event rate during the Treatment Period defined across Weeks 1 through 53, 105, 157 or 209"}
- {"endpoint_text":"- Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 157 or 209), in patients with ≥ 2 events of adjudicated acute pancreatitis in 5 years prior to treatment with study drug in the index study","definition_or_measurement_approach":"Adjudicated acute pancreatitis event rate during Treatment Period (Weeks 1–53/105/157/209) in the subgroup with ≥2 adjudicated acute pancreatitis events in the 5 years prior to index-study treatment"}
- {"endpoint_text":"- Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 157 or 209) in patient with a prior history of pancreatitis within 10 years prior to Screening in the index study","definition_or_measurement_approach":"Adjudicated acute pancreatitis event rate during Treatment Period (Weeks 1–53/105/157/209) in patients with prior history of pancreatitis within 10 years prior to screening in the index study"}
- {"endpoint_text":"- Proportion of patients who achieve ≥ 70% reduction in fasting TG from Baseline at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Proportion of patients with ≥70% reduction in fasting TG from Baseline measured at Months 6, 12, 24, 36 and 48"}
- {"endpoint_text":"- Proportion of patients who achieve fasting TG ≤ 500 mg/dL at Month 6, 12, 24, 36, 48","definition_or_measurement_approach":"Proportion of patients achieving fasting TG ≤ 500 mg/dL at Months 6, 12, 24, 36 and 48"}
Recruitment
- Planned Sample Size
- 30
- Recruitment Window Months
- 50
- Consent Approach
- Informed consent obtained using subject information sheets and informed consent forms (L1 ICF documents). Country-specific ICFs and language versions are provided (documents listed include versions in English, Spanish, French, Italian, Dutch, Portuguese, Swedish, Slovak). A pregnant partner ICF and other ancillary subject information materials are also listed.
Geography
- Total Number Of Sites
- 14
- Total Number Of Participants
- 30
Sweden
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 12-08-2024
- Processing Time Days
- 5
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Sahlgrenska University Hospital-Vaestra Goetalandsregionen
- Department Name
- Molecular and Clinical Medicine
- Principal Investigator Name
- Stefano Romeo
- Principal Investigator Email
- stefano.romeo@wlab.gu.se
- Contact Person Name
- Stefano Romeo
- Contact Person Email
- stefano.romeo@wlab.gu.se
France
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 26-09-2024
- Processing Time Days
- 50
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- Endrocrinilogy
- Principal Investigator Name
- Bruno Verges
- Principal Investigator Email
- bruno.verges@chu-dijon.fr
- Contact Person Name
- Bruno Verges
- Contact Person Email
- bruno.verges@chu-dijon.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Endrocrinology
- Principal Investigator Name
- Sybil Charriere
- Principal Investigator Email
- sybil.charriere@chu-lyon.fr
- Contact Person Name
- Sybil Charriere
- Contact Person Email
- sybil.charriere@chu-lyon.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Nutrition, metabolic diseases and endocrinology
- Principal Investigator Name
- René Valero
- Principal Investigator Email
- rene.valero@ap-hm.fr
- Contact Person Name
- René Valero
- Contact Person Email
- rene.valero@ap-hm.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 02-09-2024
- Processing Time Days
- 26
- Number Of Sites
- 3
- Number Of Participants
- 6
Sites
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- Metabolic Diseases
- Principal Investigator Name
- Marcello Arca
- Principal Investigator Email
- marcello.arca@uniroma1.it
- Contact Person Name
- Marcello Arca
- Contact Person Email
- marcello.arca@uniroma1.it
- Site Name
- Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
- Department Name
- Internal Medicine
- Principal Investigator Name
- Angelo Baldassarre Cefalù
- Principal Investigator Email
- abaldassare.cefalu@unipa.it
- Contact Person Name
- Angelo Baldassarre Cefalù
- Contact Person Email
- abaldassare.cefalu@unipa.it
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Internal Medicine
- Principal Investigator Name
- Matteo Di Minno
- Principal Investigator Email
- matteo.diminno@unina.it
- Contact Person Name
- Matteo Di Minno
- Contact Person Email
- matteo.diminno@unina.it
Spain
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 12-08-2024
- Processing Time Days
- 5
- Number Of Sites
- 3
- Number Of Participants
- 8
Sites
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- Internal Medicine
- Principal Investigator Name
- Agustín Blanco Echevarría
- Principal Investigator Email
- agustin.blanco@salud.madrid.org
- Contact Person Name
- Agustín Blanco Echevarría
- Contact Person Email
- agustin.blanco@salud.madrid.org
- Site Name
- Hospital Clinic De Barcelona
- Department Name
- Endocrinology and Nutrition
- Principal Investigator Name
- Amanda Jiménez Pineda
- Principal Investigator Email
- ajimenez1@clinic.cat
- Contact Person Name
- Amanda Jiménez Pineda
- Contact Person Email
- ajimenez1@clinic.cat
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Internal Medicine
- Principal Investigator Name
- Ovidio Muñiz Grijalvo
- Principal Investigator Email
- cmunizg@gmail.com
- Contact Person Name
- Ovidio Muñiz Grijalvo
- Contact Person Email
- cmunizg@gmail.com
Slovakia
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 14-08-2024
- Processing Time Days
- 7
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Metabolické centrum Kataríny Rašlovej s.r.o.
- Department Name
- Ambulancia diabetológie a porúch látkovej premeny a výživy
- Principal Investigator Name
- Katarína Rašlová
- Principal Investigator Email
- metabolickecentrum@yahoo.com
- Contact Person Name
- Katarína Rašlová
- Contact Person Email
- metabolickecentrum@yahoo.com
Portugal
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 16-08-2024
- Processing Time Days
- 9
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Unidade Local De Saude Do Alto Ave E.P.E.
- Department Name
- Serviço de Gastrenterologia
- Principal Investigator Name
- Tiago Cúrdia Gonçalves
- Principal Investigator Email
- centroacademico@hospitaldeguimaraes.min-saude.pt
- Contact Person Name
- Tiago Cúrdia Gonçalves
- Contact Person Email
- centroacademico@hospitaldeguimaraes.min-saude.pt
- Site Name
- Unidade Local De Saude De Lisboa Ocidental E.P.E.
- Department Name
- Serviço de Endocrinologia
- Principal Investigator Name
- João Sequeira Duarte
- Principal Investigator Email
- jmsduarte@chlo.min-saude.pt
- Contact Person Name
- João Sequeira Duarte
- Contact Person Email
- jmsduarte@chlo.min-saude.pt
Netherlands
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 13-08-2024
- Processing Time Days
- 6
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Stichting Amsterdam UMC
- Department Name
- Vascular Medicine
- Principal Investigator Name
- Erik Sjoerd Gerard Stroes
- Principal Investigator Email
- e.s.stroes@amsterdamumc.nl
- Contact Person Name
- Erik Sjoerd Gerard Stroes
- Contact Person Email
- e.s.stroes@amsterdamumc.nl
Sponsor
Primary sponsor
- Full Name
- Ionis Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Pharmaceutical Product Development LLC
- Responsibilities
- code:4
- Name
- Medpace Finland Oy
- Responsibilities
- codes:1,10,12,13,15 (Maintenance of QA and QC of CRO systems and processes),2,4,5,8
- Name
- Almac Clinical Services Limited
- Responsibilities
- code:15 (Qualifed Person release of IMP)
- Name
- Bioclinica Inc.
- Responsibilities
- code:7
- Name
- Charles River Laboratories Montreal ULC
- Responsibilities
- code:4
- Name
- Versiti Wisconsin Inc.
- Responsibilities
- code:4
Third parties
- {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"codes:1,10,12,13,15 (Maintenance of QA and QC of CRO systems and processes),2,4,5,8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Versiti Wisconsin Inc.","duties_or_roles":"code:4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Switzerland","full_name":"Socar Research S.A.","duties_or_roles":"code:15 (Pancreatitis and MACE Adjudication Committee (PAC))","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"code:15 (Home health)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"code:15 (Qualifed Person release of IMP)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"code:7","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- ISIS 678354
- Active Substance
- ISIS 678354 SODIUM SALT
- Modality
- Oligonucleotide
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Maximum Dose
- 50 mg
- Investigational Product Name
- ISIS 678354
- Active Substance
- ISIS 678354 SODIUM SALT
- Modality
- Oligonucleotide
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Maximum Dose
- 80 mg
- Investigational Product Name
- ISIS 678354
- Active Substance
- ISIS 678354 SODIUM SALT
- Modality
- Oligonucleotide
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Maximum Dose
- 80 mg
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