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IMPAACT P1063: Safety and Effectiveness of Atorvastatin in HIV Infected Children and Adolescents With Hyperlipidemia

Phase 1
Terminated
Conditions
HIV Infections
Hyperlipidemia
Interventions
Registration Number
NCT00663234
Lead Sponsor
International Maternal Pediatric Adolescent AIDS Clinical Trials Group
Brief Summary

Treatment of HIV with combination antiretroviral regimens frequently results in the suppression of HIV viral load, significant immune recovery, and delayed disease progression. However, treatment with these regimens, particularly protease inhibitors (PIs), has been associated with significant increases in cholesterol and triglycerides in HIV-infected adults and children. The purpose of this study was to evaluate the safety and effectiveness of escalating doses of atorvastatin, a FDA-approved drug which lowers cholesterol and triglyceride levels, in HIV-infected children receiving stable antiretroviral regimens.

Detailed Description

Antiretroviral regimens, particularly those containing PIs, often cause hyperlipidemia, which is an increase in the amount of fat (such as cholesterol and triglycerides) in the blood. These increases can lead to heart disease and pancreatitis. Although the mechanism by which PIs cause hyperlipidemia is not clearly understood, there are medications to combat this side effect. The primary purpose of this study was to evaluate the safety and effectiveness of escalating doses of atorvastatin, based on low-density lipoprotein cholesterol (LDL-C) levels, in HIV-infected children receiving stable antiretroviral therapy.

Participants were assigned to one of two groups based on age (10 to 14 years or 15 to 23 years) and were treated for a maximum of 48 weeks. The first six participants enrolled in the study were in the 15 to 23 year old age group. Once safety data through week 8 on these 6 participants was analyzed, the remaining participants were enrolled. All participants received atorvastatin in combination with a stable antiretroviral regimen. Each participant was followed independently according to a dose escalation algorithm for atorvastatin. Participants began dosing at 10 mg daily. If efficacy criteria were not met, dosing increased to 20 mg daily at week 8. Since dose escalations were done within subject, safety and efficacy rates were presented for the dose-escalation strategy overall and not for individual doses. Atorvastatin was provided by the study, but antiretrovirals were not.

Study visits occurred at study entry and weeks 4, 8, 12, 24, 36, and 48. Safety labs were collected at all study visits. Blood collection for lipid measurements occurred at weeks 4, 12, 24 and 48.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
28
Inclusion Criteria
  • A diagnosis of HIV-1 infection
  • CD4 % of at least 15 at screening
  • HIV-1 viral load of less than 10,000 copies/ml at screening
  • On a stable antiretroviral therapy regimen for at least 6 months
  • Tanner stage of 2 or higher
  • At least two LDL-C measurements of 130 mg/dL or higher over the 6 months prior to screening and after documented attempts at modifying diet and other risk factors. More information on this criterion can be found in the protocol.
  • Able to fast overnight for 8 hours
  • Negative pregnancy test at screening
  • Agree to use two appropriate forms of contraception (female participants). More information on this criterion can be found in the protocol.
Exclusion Criteria
  • Certain abnormal laboratory values
  • Any laboratory or unresolved clinical toxicity of Grade 3 or higher
  • Unlikely to remain on current antiretroviral therapy for at least six months after study entry
  • Use of statin, fibrate, or niacin within 3 months prior to study entry
  • Evidence of chronic ongoing myositis or history of myopathy or neuromuscular disorder
  • Symptomatic peripheral neuropathy within 6 months prior to study entry
  • Pharmacologic treatment for depression or other mental disorder excluding Attention Deficit Disorder within 30 days prior to study entry
  • Presence of an active CDC Stage C opportunistic infection or serious bacterial infection requiring therapy within 2 weeks prior to screening.
  • Chemotherapy for malignancy within 3 months prior to study entry
  • Hepatitis B Surface Antigen positive
  • Hepatitis C viremia
  • Insulin-dependent diabetes mellitus
  • Required treatment with an agent contraindicated with either atorvastatin or PIs. More information on this criterion can be found in the protocol.
  • Pregnant or breastfeeding

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Age 15 to 23AtorvastatinParticipants ages 15 to 23 years receiving oral atorvastatin for 48 weeks while on a stable antiretroviral regimen
Age 10 to 14AtorvastatinParticipants ages 10 to 14 years receiving oral atorvastatin for 48 weeks while on a stable antiretroviral regimen
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Experiencing at Least One Treatment-related Adverse Event (AE)Study entry to weeks 12, 24, and 48

AEs were graded by the clinicians according to the Division of AIDS (DAIDS) AE Grading Table (see references in the Protocol Section) as follows: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe, Grade 4=Potentially Life-Threatening, Grade 5=Death. Relationship to study treatment was determined by the core study team. The primary outcome measure includes any AE of grade 3 or higher and liver function tests (LFTs) of grade 2 or higher.

Percentage of Participants Experiencing at Least One Adverse Event (AE)Study entry to weeks 12, 24, and 48

AEs were graded by the clinicians according to the Division of AIDS (DAIDS) AE Grading Table (see references in the Protocol Section) as follows: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe, Grade 4=Potentially Life-Threatening, Grade 5=Death. The primary outcome measure includes any AE of grade 3 or higher and liver function tests (LFTs) of grade 2 or higher.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria (Intention to Treat)Study entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria (Data Available)Study entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria (Per Protocol)Study entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria and Did Not Experience a Primary Safety Endpoint Attributable to Study DrugStudy entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria by Age GroupStudy entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percentage of Participants Who Met the LDL Cholesterol (LDL-C) Efficacy Criteria by NNRTI TreatmentStudy entry and weeks 4, 12, 24, and 48

Efficacy was defined as having LDL-C of 110 mg/dL or less or at least 30% decline in LDL-C from baseline to the specified week.

Percent Change in LDL Cholesterol (LDL-C) From Study EntryStudy entry and weeks 4, 12, 24, and 48
Percentage of Participants Experiencing at Least One Treatment-related Adverse Event (AE) by Age GroupStudy entry to weeks 12, 24, and 48

AEs were graded by the clinicians according to the Division of AIDS (DAIDS) AE Grading Table (see references in the Protocol Section) as follows: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe, Grade 4=Potentially Life-Threatening, Grade 5=Death. Relationship to study treatment was determined by the core study team. The primary outcome measure includes any AE of grade 3 or higher and liver function tests (LFTs) of grade 2 or higher.

Percentage of Participants Experiencing at Least One Adverse Event (AE) by Age GroupStudy entry to weeks 12, 24, and 48

AEs were graded by the clinicians according to the Division of AIDS (DAIDS) AE Grading Table (see references in the Protocol Section) as follows: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe, Grade 4=Potentially Life-Threatening, Grade 5=Death. The primary outcome measure includes any AE of grade 3 or higher and liver function tests (LFTs) of grade 2 or higher.

Secondary Outcome Measures
NameTimeMethod
Percent Change in Fasting Total Cholesterol (TC) From Study EntryStudy entry and weeks 4, 12, 24, and 48
Percent Change in Triglycerides (TG) From Study EntryStudy entry and weeks 4, 12, 24, and 48
Percent Change in HDL-cholesterol (HDL-C) From Study EntryStudy entry and weeks 4, 12, 24, and 48
Percent Change in Apolipoprotein A1 (Apo A-1) From Study EntryStudy entry and weeks 12, 24, and 48
Percent Change in Apolipoprotein B (Apo B) From Study EntryStudy entry and weeks 12, 24, and 48
Percent Change in High-sensitivity CRP (Hs-CRP) From Study EntryStudy entry and weeks 12, 24, and 48
Percent Change in Interleukin 6 (IL-6) From Study EntryStudy entry and weeks 12, 24, and 48
Percentage of Participants With Undetectable Plasma HIV-1 RNAStudy entry and weeks 12, 24, and 48

Undetectable is defined as plasma HIV-1 RNA below the lower limit of quantification of the assay used.

Trial Locations

Locations (11)

Univ. of Colorado Denver NICHD CRS (5052)

🇺🇸

Aurora, Colorado, United States

New York University NY (5012)

🇺🇸

New York, New York, United States

Chicago Children's CRS (4001)

🇺🇸

Chicago, Illinois, United States

Tulane University (5095)

🇺🇸

New Orleans, Louisiana, United States

Boston Medical Center Ped. HIV Program NICHD CRS (5011)

🇺🇸

Boston, Massachusetts, United States

Bronx-Lebanon Hospital IMPAACT CRS (6901)

🇺🇸

Bronx, New York, United States

Metropolitan Hospital (5003)

🇺🇸

New York, New York, United States

St. Jude/UTHSC CRS (6501)

🇺🇸

Memphis, Tennessee, United States

Texas Children's Hosp. CRS (3801)

🇺🇸

Houston, Texas, United States

Univ. of Miami Ped. Perinatal HIV/AIDS CRS (4201)

🇺🇸

Miami, Florida, United States

University of South Florida Tampa (5018)

🇺🇸

Tampa, Florida, United States

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