Clinical trial NCT06804980: DESIFOR-EXPAND (MHIF)
Official title: The DEterminig Statin Intolerance for Rosuvastatin Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Statin Therapy
Statin Adverse Reaction
Statin
Keywords: Statin Intolerance
lipid-lowering medication
LDL
LDL Cholesterol
Lead sponsor: Minneapolis Heart Institute Foundation
Sponsor class: OTHER
First posted: 2025-02-03
Enrollment: 100
Sex: ALL
Minimum age (years): 21
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: Each kit will include 3 28-day packs of placebo and 2 28-day packs of rosuvastatin, assigned at random. Each kit will have a unique identification number, which the pharmacy will record in a drug tracking log as it is dispensed. Within each kit, a unique identifying number will be present on each blister pack which can be used to determine the order of allocation of placebo and rosuvastatin.
Primary purpose: TREATMENT

Brief summary: The DESIFOR pilot study was conducted to determine the feasibility of utilizing an n-of-1 trial to facilitate tolerance of unblinded rosuvastatin in patients with prior statin intolerance

Detailed description: Recently developed lipid lowering therapies have been shown to significantly reduce LDL cholesterol and subsequent CVD risk (1, 2). However, they are expensive, and the cost-effectiveness of these medications remains controversial, especially in primary prevention. Novel lipid lowering therapies are frequently considered for individuals with statin intolerance, but payers frequently deny prescription for these medications due to lack of adequate proof of true statin intolerance. How to accurately determine statin intolerance remains a clinical conundrum for both patients and providers. A more scientific, definitive method to determine statin intolerance could potentially allow appropriate, cost-effective use of statin therapy in individuals found to not actually be statin intolerant as well as judicious use of novel medications in patients who are found to truly be statin intolerant.

The DESIFOR pilot study was conducted to determine the feasibility of utilizing an n-of-1 trial to facilitate tolerance of unblinded rosuvastatin in patients with prior statin intolerance. The DESIFOR pilot study was a single-center, randomized, double-blinded, n-of-1 trial in patients aged 21 to 79 years and eligible for statin therapy by current guidelines but not on therapy due to intolerance of \>2 statins. Using a double-blinded, N-of-1 randomized trial in patients with previous statin intolerance, \<10% of patients had data suggestive of true statin intolerance, and 2/3 of patients were subsequently able to tolerate 3-months of unblinded treatment with rosuvastatin (3). These data raise the hypothesis that an n-of-1 trial can be used to facilitate tolerance of unblinded rosuvastatin in patients with prior statin intolerance.

Eligibility criteria: Inclusion Criteria:

* Adult patients (≥ 21 years old) with a prior history of statin intolerance. Statin intolerance is defined by discontinuation of at least 2 different statin medications due to possible side effects. Patients can participate in the trial while on other lipid-lowering agents, such as ezetimibe and PSCK9 inhibitors, as long as the patient has been on the other lipid lowering therapy and tolerating it well for at least 1 month. For individuals with established ASCVD or multiple ASCVD risk factors, initiation of other lipid lowering therapy prior to participation in DESIFOR is encouraged.
* 2. At least 30 days since discontinued use of a statin

Exclusion Criteria:

* Women who are pregnant, nursing or attempting to become pregnant
* Individuals who experienced severe reactions in the past, including rhabdomyolysis, severe myositis, anaphylaxis
* Individuals who are not otherwise clinically indicated to take rosuvastatin 20 mg

Primary outcomes:
measure: Musculoskeletal symptom presence/absence
timeFrame: 9 months
measure: Statin Tolerance
timeFrame: 9 months
measure: Variables associated with statin intolerance
timeFrame: 9 months
Secondary outcomes:
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Minneapolis Heart Institute Foundation
state: Minnesota
Study officials:
References: