The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
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| ClinicalTrials.gov Identifier: NCT02658175 |
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Recruitment Status :
Completed
First Posted : January 18, 2016
Last Update Posted : December 24, 2020
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Sponsor:
Ionis Pharmaceuticals, Inc.
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
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Brief Summary:
An Open-Label Study of Volanesorsen (IONIS 304801) Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Familial Chylomicronemia Syndrome Lipoprotein Lipase Deficiency Hyperlipoproteinemia Type 1 | Drug: Volanesorsen | Phase 3 |
This is a multi-center, open-label study for Group 1: ISIS 304801-CS6 (index study) roll-over FCS patients, Group 2: ISIS 304801-CS16 (index study) roll-over FCS patients, and Group 3: FCS patients who did not participate in the ISIS 304801-CS6 or ISIS 304801-CS16 index studies. All patients will receive volanesorsen 300 mg once per week for 52 weeks. Patients will have the option of continuing dosing for an additional 52 weeks until an expanded access program is approved and available in their country.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 69 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | ISIS 304801-CS7 The APPROACH Open Label Study Volanesorsen (ISIS 304801) An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) |
| Actual Study Start Date : | December 31, 2015 |
| Actual Primary Completion Date : | January 15, 2020 |
| Actual Study Completion Date : | January 15, 2020 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Familial lipoprotein lipase deficiency
| Arm | Intervention/treatment |
|---|---|
| Experimental: Volanesorsen |
Drug: Volanesorsen
300 mg volanesorsen
Other Names:
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Primary Outcome Measures :
- Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 117 Weeks ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Must give written informed consent to participate in the study (signed and dated) and any authorization required by law
- Able and willing to participate in a 65-week study
Group 1 and 2:
- Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-CS16 (index studies) with an acceptable safety profile, per Sponsor and Investigator judgment
Group 3:
- Patients who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of Familial Chylomicronemia Syndrome (FCS) may enroll in the study.
- History of chylomicronemia
- A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
- Fasting triglycerides ≥ 750 mg/dL (8.4mmol/L) at Screening
Exclusion Criteria:
- Unwilling to comply with lifestyle requirements for the duration of the study
Group 1 and 2:
- 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.
Group 3:
- Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
- Active pancreatitis within 4 weeks of screening
- Acute Coronary Syndrome within 6 months of screening
- Major surgery within 3 months of screening
- Treatment with Glybera therapy within 2 years of screening
- Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02658175
Locations
| United States, California | |
| IONIS Investigative Site | |
| Huntington Beach, California, United States, 94143 | |
| IONIS Investigative Site | |
| San Francisco, California, United States, 94143 | |
| United States, Florida | |
| IONIS Investigative Site | |
| Boca Raton, Florida, United States, 33434 | |
| United States, Massachusetts | |
| IONIS Investigative Site | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Pennsylvania | |
| IONIS Investigative Site | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| United States, Texas | |
| IONIS Investigative Site | |
| Houston, Texas, United States, 77030 | |
| United States, Virginia | |
| IONIS Investigative Site | |
| Norfolk, Virginia, United States, 23510 | |
| United States, Washington | |
| IONIS Investigative Site | |
| Seattle, Washington, United States, 98104 | |
| Brazil | |
| IONIS Investigative Site | |
| Sao Paulo, Brazil, 04040-001 | |
| IONIS Investigative Site | |
| Sao Paulo, Brazil, CEP-05403-000 | |
| Canada, British Columbia | |
| IONIS Investigative Site | |
| Vancouver, British Columbia, Canada, V6Z1Y6 | |
| Canada, Quebec | |
| IONIS Investigative Site | |
| Chicoutimi, Quebec, Canada, G7H 7K9 | |
| IONIS Investigative Site | |
| Montreal, Quebec, Canada, H2W 1R7 | |
| Canada | |
| IONIS Investigative Site | |
| Quebec, Canada, G1V 4W2 | |
| France | |
| IONIS Investigative Site | |
| Paris, Cedex 13, France, 75013 | |
| IONIS Investigative Site | |
| Marseille Cedex 05, France, 13385 | |
| IONIS Investigative Site | |
| Nantes cedex 1, France, 44800 | |
| Germany | |
| IONIS Investigative Site | |
| Berlin, Germany, 13353 | |
| IONIS Investigative Site | |
| Cologne, Germany, 50937 | |
| Israel | |
| IONIS Investigative Site | |
| Safed, Israel, 13110 | |
| Italy | |
| IONIS Investigative Site | |
| Palermo, Italy, 90127 | |
| IONIS Investigative Site | |
| Roma, Italy, 00161 | |
| IONIS Investigative Site | |
| Rome, Italy, 00161 | |
| Netherlands | |
| IONIS Investigative Site | |
| Amsterdam-Zuidoost, Netherlands, 1105 AZ | |
| South Africa | |
| IONIS Investigative Site | |
| Cape Town, South Africa, 7925 | |
| Spain | |
| IONIS Investigative Site | |
| Barcelona, Spain, 08036 | |
| IONIS Investigative Site | |
| La Coruna, Spain, 15001 | |
| IONIS Investigative Site | |
| Madrid, Spain, 28007 | |
| IONIS Investigative Site | |
| Sevilla, Spain, 41013 | |
| IONIS Investigative Site | |
| Zaragoza, Spain, 50009 | |
| United Kingdom | |
| IONIS Investigative Site | |
| Birmingham, United Kingdom, B9 5SS | |
| IONIS Investigative Site | |
| London, United Kingdom, SE1 7EH | |
| IONIS Investigative Site | |
| Manchester, United Kingdom, M13 9WL | |
| IONIS Investigative Site | |
| Manchester, United Kingdom, M23 9LT | |
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
| Responsible Party: | Ionis Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT02658175 |
| Other Study ID Numbers: |
ISIS 304801-CS7 |
| First Posted: | January 18, 2016 Key Record Dates |
| Last Update Posted: | December 24, 2020 |
| Last Verified: | December 2020 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type I Hyperlipoproteinemias Hyperlipidemias Syndrome Disease Pathologic Processes |
Dyslipidemias Lipid Metabolism Disorders Metabolic Diseases Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |