Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome Clinical Trial

Official title:

Olezarsen (ISIS 678354) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT06360237
• Other study ID #: ISIS 678354
• Status: Available

Study information

• Verified date: April 2024
• Source: Ionis Pharmaceuticals, Inc.
• Contact: Ionis Pharmaceuticals Medical Information
• Phone: 1-833-644-6647 (833-MI-IONIS)
• Email: MedInfo[@]ionisph.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).

Description:

The Expanded Access Program (EAP) is intended to provide pre-approval access to olezarsen for eligible patients with FCS who have limited or no available treatment options. This program is open in the United States and operates under the individual patient (also referred to as single patient) IND expanded access route in which the patient's treating physician serves as the sponsor. Expanded access requests must be from the patient's treating physicians and submitted according to the instructions at https://www.ionispharma.com/patients/expanded-access-policy/

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Has a diagnosis of FCS as determined by the sponsoring physician. Ionis will review each application to determine eligibility based on documentation of validated genetic or clinical diagnosis.

o Documented loss of function mutations (homozygous, compound / double heterozygous) in genes such as LPL, GPIHBP1, APOA5, APOC2 or LMF1) or clinically validated diagnosis of FCS.

• Resides in and is a resident of the United States.

• Willing to follow a diet comprising =20 g fat per day.

Exclusion Criteria:

• Has any new or worsening of existing conditions which, in the opinion of the physician, would make the patient unsuitable for treatment with olezarsen.

• Olezarsen naïve patients with baseline platelet count <100x109/L at qualification.

• Estimated GFR (eGFR) <30 mL/min/1.73 m2.

• Secondary factors are the cause of triglyceride elevations.

• Is currently hospitalized in an acute emergency setting.

Related Conditions & MeSH terms

• Familial Chylomicronemia Syndrome
• Hyperlipoproteinemia Type I
• Syndrome

Intervention

Drug:
• Olezarsen
Olezarsen 80 mg administered once monthly by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

External Links

•   Related Info