An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia Syndrome1002742410013317
- Registration Number
- NL-OMON51487
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 5
1. Must have given written informed consent (signed and dated) and any
authorizations
required by local law and be able to comply with all study requirements
2. Satisfactory completion of the ISIS 678354-CS3 index study (last dose as
scheduled at Week 49) with an acceptable safety profile, per Investigator
judgement
3. Willing to follow a diet comprising <= 20 g fat per day during the study
4. Satisfy the following:
a. Females: must be non-pregnant and non-lactating and either:
i. surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral
salpingectomy,
bilateral oophorectomy)
ii. postmenopausal (defined as 12 months of spontaneous amenorrhea in females
> 55 years of age or, in females <= 55 years, 12 months of spontaneous amenorrhea
without an alternative medical cause and follicle-stimulating hormone (FSH)
levels in the postmenopausal range for the laboratory involved)
iii. abstinent* or
iv. if engaged in sexual relations of child-bearing potential, agree to use a
highly
effective contraceptive method from the time of signing the informed consent
form until at least 30 weeks after the last dose of ISIS 678354
b. Males: Surgically sterile, abstinent* or if engaged in sexual relations with
a female of
child-bearing potential, agree to use a highly effective contraceptive method
from the
time of signing the informed consent form until at least 30 weeks after the
last dose of
ISIS 678354
* Abstinence is only acceptable as true abstinence, i.e., when this is in line
with the
preferred and usual lifestyle of the patient. Periodic abstinence (e.g.,
calendar,
ovulation, symptothermal, post-ovulation methods), declaration of abstinence for
the duration of a trial, and withdrawal are not acceptable methods of
contraception
5. The following concomitant medications will be allowed if dosing regimen is
expected to
remain constant through the end of the study (occasional or intermittent use of
over-the-counter (OTC) medications will be allowed at Investigator*s
discretion):
a. Statins, omega-3 fatty acids (prescription and OTC), fibrates, or other
lipid-lowering
medications. Patients taking OTC omega-3 fatty acids should make every effort to
remain on the same brand through the end of the study
b. Antidiabetic medications except glucagon-like peptide 1(GLP-1) agonist that
is disallowed
c. Antihypertensive medications
d. Oral anticoagulants (e.g., warfarin, dabigatran, rivaroxaban, and apixaban)
and
regular clinical monitoring is performed
e. Tamoxifen, estrogens or progestins
f. Atypical antipsychotic medications (e.g., olanzapine and clozapine)
1. 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).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety endpoints include a proportion of patients who show the following<br /><br>changes from Baseline to Week 53, week 105 and week 157:*<br /><br>• Decrease in platelet count by >= 30%<br /><br>• Decrease in platelet count by >= 50%<br /><br>• Platelet count value < 50,000 mm3<br /><br>• Major bleeding events<br /><br>• Clinically relevant non-major bleeding events<br /><br>• Decrease in estimated glomerular filtration rate (eGFR) by >= 30%<br /><br>• Decrease in eGFR by >= 50%<br /><br>• Urine protein-Creatine ratio (UPCR) >= 1000 mg/g<br /><br>• Urine albumin-creatine ratio (UACR) >= 500 mg/g<br /><br>• alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >= 5 ×<br /><br>upper limit of normal (ULN)<br /><br>• Total bilirubin >= 2.0 mg/dL<br /><br>• ALT or AST >= 3 × ULN and total bilirubin >= 2 × ULN<br /><br>* Lab values based on confirmed results<br /><br>In addition, rate of major adverse cardiovascular event (MACE) will be<br /><br>summarized.</p><br>
- Secondary Outcome Measures
Name Time Method