Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy
- Conditions
- Partial lipodystrophyMedDRA version: 21.1Level: PTClassification code 10053857Term: Partial lipodystrophySystem Organ Class: 10040785 - Skin and subcutaneous tissue disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2015-000493-35-IT
- Lead Sponsor
- IONIS PHARMACEUTICALS, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 125
1. Clinical diagnosis of FPL plus diagnosis of type 2 diabetes mellitus and
hypertriglyceridemia.
2.Diagnosis of Type 2 diabetes mellitus as defined by the ADASMCD in
2015 made at least 12 weeks prior to the screening visit
3. Hypertriglyceridemia is defined as Fasting TG = 500 mg/dL (= 5.7
mmol/L) at Screening and qualification visit.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 47
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3
1. A diagnosis of generalized lipodystrophy
2. A diagnosis of acquired partial lipodystrophy (APL)
3. Acute pancreatitis within 4 weeks of Screening
4. History within 6 months of Screening of acute or unstable cardiac
ischemia (myocardial infarction, acute coronary syndrome, new onset
angina), stroke, transient ischemic attack or unstable congestive heart
failure requiring a change in medication
5. Major surgery within 3 months of Screening
6. Platelet count 7. Have any other conditions in the opinion of the investigator which
could interfere with the patient participating in our completing the study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of ISIS 304801 (300 mg once weekly) for<br>reduction in severity of metabolic derangement in patients with FPL with<br>hypertriglyceridemia and uncontrolled diabetes ;Secondary Objective: To evaluate the safety and tolerability of ISIS 304801 in patients with <br>FPL.To further evaluate the role of serum TGs in modulating insulin<br>resistance in FPL patients and the impact of TGs reduction on adipose<br>tissue distribution in FPL patients ;Primary end point(s): The primary efficacy analysis will be the comparison of percent changes<br>from Baseline to the primary analysis time point in fasting TG between<br>ISIS 304801 300 mg once weekly group and placebo group in the Full<br>Analysis Set (FAS) ;Timepoint(s) of evaluation of this end point: The primary analysis time point is at the end of Month 3 where the value <br>is defined as the average of Week 12 and Week 13 fasting assessments.<br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The secondary endpoints include:<br>¿ Change from baseline in HbA1c<br>¿ Change from Baseline in liver volume and hepatic steatosis (as<br>assessed by MRI)<br>¿ Absolute change from Baseline in fasting TG<br>¿ Proportion of patients who achieve a = 40% reduction in fasting TG<br>¿ Change from Baseline in fasting plasma glucose<br>¿ Reduction in insulin use<br>;Timepoint(s) of evaluation of this end point: At the end of Month 3 where the value is defined as the average of Week <br>12 (Day 78) and Week 13 (Day 85) fasting assessments. The value at<br>Month 6 is defined as the average of Week 25 and Week 26 and the<br>value at Month 12 is defined as the average of Week 51 and Week 52<br>fasting assessments.