An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema
- Conditions
- HAEHereditary angioedema1000533010002426
- Registration Number
- NL-OMON54255
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 7
1. Must give written informed consent to participate in the study (signed and
dated) and any authorizations required by law
2. Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17
with an acceptable safety and tolerability profile, per Sponsor and
Investigator judgement
3. Able and willing to participate in a 64-week study
4. Satisfy 1 of the following:
a. Females: Non-pregnant and non-lactating; surgically sterile (e.g., tubal
occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy),
post-menopausal (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), abstinent*,
or, if engaged in sexual relations of child-bearing potential, patient is using
an acceptable contraceptive method from time of signing the ICF until 24 weeks
after the last dose of ISIS 721744 administration
b. Males: Surgically sterile, abstinent* or if engaged in sexual relations with
a female of child-bearing potential, patient is utilizing an acceptable
contraceptive method (refer to Section 6.3.1) from the time of signing the ICF
until 24 weeks after the last dose of ISIS 721744 administration
* 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. Patients must have access to, and the ability to use, >= 1 acute
medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a
bradykinin receptor (BK)2-receptor antagonist) to treat angioedema attacks
1. Have any new condition or worsening of an existing condition or change or
anticipated change in medication or other reason, 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Endpoint: Incidence and severity of treatment-emergent adverse events<br /><br>(TEAE)</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Endpoints:<br /><br>- The time-normalized HAE attacks (monthly) by treatment<br /><br>- Plasma PKK levels, plasma proenzyme activation and cHK levels<br /><br>- Consumption of on-demand medications<br /><br>- AE-QoL questionnaire score<br /><br><br /><br>Safety Endpoints:<br /><br>- Laboratory tests<br /><br>- Electrocardiograms (ECGs)<br /><br>- Use of concomitant medications<br /><br>- Vital signs</p><br>