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NCT01475825 · ClinicalTrials.gov registry record · Phase 3
A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
A Phase 3 study, sponsored by Kastle Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 309
- Enrollment target
NCT01475825: Completed Phase 3 study, sponsored by Kastle Therapeutics.
NCT01475825 is a Phase 3 study that has completed, run by Kastle Therapeutics. The registered enrollment target is 309 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (60% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01475825, a Phase 3 study, has completed, sponsored by Kastle Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 309 participants
- Enrollment target
Study Summary
Primary objective: Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo. Secondary Objectives: * Determine whether there are qualitative differences between the safety profiles of the 2 dosing regimens and placebo in Cohort 1, patients with HeFH with LDL-C levels ≥160 mg/dL and \<200 mg/dL plus the presence of CHD/risk equivalents (referred to as Cohort 2), and the overall study population * Determine whether there are qualitative differences between the tolerability of the 2 dosing regimens and placebo in Cohort 1, Cohort 2, and the overall study population * Further characterize the pharmacokinetics (PK) of the 2 dosing regimens in Cohort 1, Cohort 2, and the overall study population * Determine whether the 2 mipomersen dosing regimens significantly reduce atherogenic lipid levels in Cohort 2 compared to placebo * Obtain additional data regarding ongoing safety and efficacy of mipomersen in patients with FH and inadequately controlled LDL-C who complete the primary efficacy assessment visit (PET) in the Blinded Treatment Period and continue treatment in Open-Label Continuation Period
Primary Outcome
The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels ≥200 mg/deciliter (dL) plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents.
Interventions
- DRUG Placebo
- DRUG mipomersen sodium 200 mg
- DRUG mipomersen sodium 70 mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 309 participants |
| Start Date | 2011-12 |
| Est. Completion | 2015-12-29 |
| Phase | Phase 3 |
What the finished NCT01475825 record still lists
NCT01475825 is an interventional study that assigns participants to a tested intervention. The registered 309 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (60% lower).
The record links to 0 conditions, and to 3 interventions - of which Placebo is the first listed.
NCT01475825 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01475825 about?
NCT01475825 is a clinical study titled "A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol". Primary objective: Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presence of coronary heart...
What is the current status of trial NCT01475825?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 309 participants. The study started on 2011-12. Estimated completion is 2015-12-29.
What interventions are being tested in trial NCT01475825?
The interventions under investigation include: Placebo (DRUG), mipomersen sodium 200 mg (DRUG), mipomersen sodium 70 mg (DRUG).
Who is sponsoring clinical trial NCT01475825?
This trial is sponsored by Kastle Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01475825's enrollment target sits among peer trials
309 1109th of 2000 higher than 889 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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