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Atazanavir Used in Combination With Other Anti-HIV Drugs in HIV-Infected Infants, Children, and Adolescents

Phase 1
Completed
Conditions
HIV Infections
Interventions
Registration Number
NCT00006604
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

The purpose of this study was to find a safe and tolerable dose of the protease inhibitor (PI) atazanavir (ATV), with or without a low-dose boost of the PI ritonavir (RTV), when taken with other anti-HIV drugs in HIV infected infants, children, and adolescents.

Advancements in anti-HIV drugs for HIV infected children and adolescents have been hard to make, in part because these patients often do not take the drugs as prescribed. ATV may be a better option because it is available in the form of powder which children and adolescents may be more willing to take regularly. Using a low dose of RTV as a boosting agent for ATV may also increase the chances of virologic response of highly active antiretroviral treatment (HAART)-experienced patients. This study aimed to find safe and tolerable doses of ATV with or without low-dose RTV boost in infants, children, and adolescents. For this study, participants were enrolled in the United States and South Africa.

Detailed Description

Advancements in HAART for HIV-infected children and adolescents are hindered by patient nonadherence. The availability of a powder formulation and the once-daily dosing schedule make ATV an attractive agent for improved adherence in pediatric treatment regimens. This study was designed to provide pharmacokinetic (PK) data to guide dosing recommendations for ATV, when given concurrently with or without low-dose RTV boost, in infants, children, and adolescents. During the study, the safety and tolerance of ATV (with or without low-dose RTV) were closely monitored, and virologic efficacy data were obtained.

There were two parts to this study. Step I took place in the United States and South Africa, and were further divided into two sets of groups, Parts A and B. Part A participants received ATV only and Part B participants received ATV with low-dose RTV boost. All participants received ATV once a day with 2 other antiretroviral drugs (not provided by the study). In Part B only, participants received ATV with a low dose of RTV. Participants were placed into 1 of 8 groups (Groups 1 to 4 for Part A; Groups 5 to 8 for Part B) with respect to age and study drug formulation. Participants in Groups 1 and 5 were infants between ages 3 months and 1 day (91 days) and 2 years (less than or exactly 730 days) and took ATV in powder form. Participants in Groups 2, 3, 6, and 7 were children between 2 years and 1 day (731 days) old and 13 years old. Groups 2 and 6 received ATV in powder form, while Groups 3 and 7 received the capsule form. Patients in Groups 4 and 8 were adolescents between 13 years and 1 day old and 21 years old (not including the 22nd birthday) and took ATV in capsule form. As of 01/02/2008 a new group, 5A was opened for enrollment. Participants in Group 5A were between 3 months and 6 months old and took ATV in powder form plus a low-dose RTV booster.

For each group, enrollment started with five participants per group. All participants were evaluated for PK and safety criteria, adjusting the dose of ATV until one was found that passes both sets of criteria. Then five additional participants were enrolled, with enrollment continuing for each group once all participants within that group meet the PK criteria. For groups receiving RTV (Groups 5 to 8), additional criteria must be met for each dose of ATV studied. In addition to the PK and safety evaluations, 24-hour post-dose concentrations (Cmin) were monitored in the first 10 participants enrolled for a dose of ATV before more participants were enrolled and studied at that same dose. Note that in Protocol Version 5.0, South African (S.A.) sites were allowed to enroll patients in study groups 3,4,5,6,7,8. As a result, the study design has been modified to further stratify study groups 3, 4, 5, 6, 7, 8 (at the final recommended dose), by country, i.e., U.S.A. versus S.A., such that 10 evaluable study subjects will be accrued in parallel to each study group-country cohort.

Clinic visits will be every 4 weeks through Week 48, then every 8 weeks until the last participant to enroll in the study has reached Week 96 of his/her treatment. If, after 56 weeks, a participant has a toxic reaction to a nucleoside/tide reverse transcriptase inhibitor (NRTI) in their medication regimen, the regimen may be changed to a different NRTI. At every visit, participants will undergo a complete medical history and physical exam, cardiac conduction evaluation, and urine and blood collection. Participants of childbearing age will have a pregnancy test performed at each visit.

Step II will only be open to South African subjects who are virologically responding to treatment when the last enrollee into either part of Step I (Part A or Part B) has completed 96 weeks of treatment (end of Step I) . All such participants will be given ATV in capsule form at the same dose they received at the end of Step I, as well as the other antiretrovirals they were receiving during Step I. Step II will continue until ATV is approved in South Africa and readily available by individual prescription, and participants will have a study visit every 12 weeks.

Note that the following ATV doses were independently evaluated for each group during the dose-finding stage based on the description above: Group 1 ATV Powder (310mg/m\^2, 620mg/m\^2); Group 2 ATV Powder (310mg/m\^2, 620mg/m\^2); Group 3 ATV Capsule (310mg/m\^2, 415mg/m\^2, 520mg/m\^2); Group 4 ATV Capsule (310mg/m\^2, 520mg/m\^2, 620mg/m\^2); Group 5 ATV Powder + RTV (310mg/m\^2); Group 6 ATV Powder +RTV (310mg/m\^2); Group 7 ATV Capsule + RTV (310mg/m\^2, 205mg/m\^2); Group 8 ATV Capsule + RTV (310mg/m\^2, 205mg/m\^2); Group 5A ATV Powder + RTV (310mg/m\^2). All these dosing groups are presented in Participant Flow groups to show the total number of participants enrolled, but only the participants enrolled at the final group doses are presented in the subsequent results.

The following groups satisfied the safety and PK guidelines specified in the protocol: Groups 3,4,6,7,8. Groups 5 and 5A did not satisfy the protocol-defined pharmacokinetic criteria. There was considerable inter-subject variability in systemic exposures in this age group, such that a dose escalation to 415mg/m\^2 may have resulted in ATV exposures greater than 90,000 ng\*hr/mL in some children. Thus, a further dose increase in Groups 5 and 5A was not attempted.

These are the final dose for each group: Groups 1 and 2 (Final dose was not established); Group 3 ATV Capsule (520mg/m\^2); Group 4 ATV Capsule (620mg/m\^2); Group 5 ATV Powder (310mg/m\^2) + RTV; Group 6 ATV Powder (310mg/m\^2) +RTV; Group 7 ATV Capsule (205mg/m\^2) + RTV; Group 8 ATV Capsule (205mg/m\^2) + RTV; Group 5A ATV Powder (310mg/m\^2) + RTV.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
195
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Step I: Group 2ATVGroup 2 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (powder) and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 620 mg/m\^2; Final Dose: Not Established
Step I: Group 6RitonavirGroup 6 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 7ATVGroup 7 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (capsule), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 205 mg/m\^2; Final Dose: 205 mg/m\^2
Step I: Group 4ATVGroup 4 enrolled participants between 13 years and 1 day of age and 21 years of age. They received ATV (capsule) and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 520 mg/m\^2, 620 mg/m\^2; Final Dose: 620 mg/m\^2
Step I: Group 1ATVGroup 1 enrolled participants between 91 days of age and 2 years of age. They received ATV (powder) and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 620 mg/m\^2; Final Dose: Not Established
Step I: Group 5aATVGroup 5a enrolled participants between 91 days of age and 180 days of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 5RitonavirGroup 5 enrolled participants between 91 days of age and 2 years of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 5aRitonavirGroup 5a enrolled participants between 91 days of age and 180 days of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 3ATVGroup 3 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (capsule) and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 415 mg/m2, 520 mg/m\^2; Final Dose: 520 mg/m\^2
Step I: Group 7RitonavirGroup 7 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (capsule), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 205 mg/m\^2; Final Dose: 205 mg/m\^2
Step I: Group 5ATVGroup 5 enrolled participants between 91 days of age and 2 years of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 6ATVGroup 6 enrolled participants between 2 years and 1 day of age and 13 years of age. They received ATV (powder), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2; Final Dose: 310 mg/m\^2
Step I: Group 8ATVGroup 8 enrolled participants between 13 years and 1 day of age and 21 years of age. They received ATV (capsule), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 205 mg/m\^2; Final Dose: 205 mg/m\^2
Step I: Group 8RitonavirGroup 8 enrolled participants between 13 years and 1 day of age and 21 years of age. They received ATV (capsule), ritonavir, and two NRTIs. ATV Dose Tested: 310 mg/m\^2, 205 mg/m\^2; Final Dose: 205 mg/m\^2
Primary Outcome Measures
NameTimeMethod
Number of Participants Who DiedFrom study entry up to week 96
Pharmacokinetic (PK) Parameter: Maximum Plasma Concentration (Cmax)Week 1 (Day 7) Intensive PK-24 hr (Pre-dose, 1, 2, 3, 4, 6, 8, and 12 hours post-dose and the following day at 24-hours post-dose)

Pharmacokinetics were determined by non-compartmental analysis and Maximum concentration (Cmax) was determined visually.

Number of Participants Who Experienced a Safety Endpoint of Interest Attributed to ATVFrom study entry up to week 96

Total Bilirubin \>= 5.1xULN, ECG Events and Other Grade 3+ toxicities attributed to study treatment.

The AEs were graded by the clinicians according to the Division of AIDS (DAIDS) Toxicity 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 study team.

Pharmacokinetic (PK) Parameter: Area Under the Curve (AUC24h)Week 1 (Day 7) Intensive PK-24hr (Pre-Dose, 1, 2, 3, 4, 6, 8, and 12 hours post-dose and the following day at 24-hours post-dose)

Pharmacokinetics were determined by non-compartmental analysis and AUC0-24hr calculated by the linear trapezoidal method.

Pharmacokinetic (PK) Parameter: Minimum Plasma Concentration (C24)Week 1 (Day 7) Intensive PK-24hr (Pre-dose, 1, 2, 3, 4, 6, 8, and 12 hours post-dose and the following day at 24-hours post-dose)

Pharmacokinetics were determined by non-compartmental analysis. C24 determined visually, except in the instance when the patient re-dosed the study medication prior to the 24 hour blood draw or the 24 hour level was not obtained, in which case the C24 was calculated from the elimination rate (ke) and the last measured concentration.

Pharmacokinetic (PK) Parameter: Clearance (CL/F)Week 1 (Day 7) Intensive PK-24 hr (Pre-dose, 1, 2, 3, 4, 6, 8, and 12 hours post-dose and the following day at 24-hours post-dose)

Pharmacokinetics were determined by non-compartmental analysis and Apparent oral clearance (CL/F) was calculated as ATV dose divided by AUC0-24hr.

Secondary Outcome Measures
NameTimeMethod
Change in CD4 Percent From Baseline to Week 48Baseline, Week 48
Change in CD4 Count (Cells/mm^3) From Baseline to Week 48Baseline, Week 48
Change in CD4 Count (Cells/mm^3) From Baseline to Week 20Baseline, Week 20
Change in CD4 Percent From Baseline to Week 20Baseline, Week 20
Change in CD4 Percent From Baseline to Week 96Baseline, Week 96
Percentage of Participants With HIV RNA <400 Copies/mL at Week 24Week 24

Over the duration of the protocol, the assay used to determine Plasma HIV RNA levels was transitioned from the Amplicor HIV-1 Assay to the Amplicor HIV-1 Monitor 1.5 UltraSensitive Assay (Roche Molecular Systems, Branchburg, NJ) to finally the Abbott Real time HIV-1 RNA assay. The assays were performed according to the manufacturer's instructions in a laboratory accredited by the College of American Pathologists and certified by the NIH Virology Quality Assurance (VQA) in the United States, and VQA certified in South Africa.

Percentage of Participants With HIV RNA <400 Copies/mL at Week 96Week 96

Over the duration of the protocol, the assay used to determine Plasma HIV RNA levels was transitioned from the Amplicor HIV-1 Assay to the Amplicor HIV-1 Monitor 1.5 UltraSensitive Assay (Roche Molecular Systems, Branchburg, NJ) to finally the Abbott Real time HIV-1 RNA assay. The assays were performed according to the manufacturer's instructions in a laboratory accredited by the College of American Pathologists and certified by the NIH Virology Quality Assurance (VQA) in the United States, and VQA certified in South Africa.

Percentage of Participants With HIV RNA <400 Copies/mL at Week 48Week 48

Over the duration of the protocol, the assay used to determine Plasma HIV RNA levels was transitioned from the Amplicor HIV-1 Assay to the Amplicor HIV-1 Monitor 1.5 UltraSensitive Assay (Roche Molecular Systems, Branchburg, NJ) to finally the Abbott Real time HIV-1 RNA assay. The assays were performed according to the manufacturer's instructions in a laboratory accredited by the College of American Pathologists and certified by the NIH Virology Quality Assurance (VQA) in the United States, and VQA certified in South Africa.

Change in CD4 Count (Cells/mm^3) From Baseline to Week 96Baseline, Week 96

Trial Locations

Locations (36)

Rush Univ. Cook County Hosp. Chicago NICHD CRS

🇺🇸

Chicago, Illinois, United States

Children's Med. Ctr. Dallas

🇺🇸

Dallas, Texas, United States

Univ. of California San Francisco NICHD CRS

🇺🇸

San Francisco, California, United States

UAB Pediatric Infectious Diseases CRS

🇺🇸

Birmingham, Alabama, United States

Usc La Nichd Crs

🇺🇸

Alhambra, California, United States

University of California, UC San Diego CRS

🇺🇸

La Jolla, California, United States

Miller Children's Hosp. Long Beach CA NICHD CRS

🇺🇸

Long Beach, California, United States

UCLA-Los Angeles/Brazil AIDS Consortium (LABAC) CRS

🇺🇸

Los Angeles, California, United States

Howard Univ. Washington DC NICHD CRS

🇺🇸

Washington, District of Columbia, United States

South Florida CDTC Ft Lauderdale NICHD CRS

🇺🇸

Fort Lauderdale, Florida, United States

Columbus Regional HealthCare System, The Med. Ctr.

🇺🇸

Columbus, Georgia, United States

Johns Hopkins Univ. Baltimore NICHD CRS

🇺🇸

Baltimore, Maryland, United States

Ann & Robert H. Lurie Children's Hospital of Chicago (LCH) CRS

🇺🇸

Chicago, Illinois, United States

Univ. of Maryland Baltimore NICHD CRS

🇺🇸

Baltimore, Maryland, United States

Boston Medical Center Ped. HIV Program NICHD CRS

🇺🇸

Boston, Massachusetts, United States

WNE Maternal Pediatric Adolescent AIDS CRS

🇺🇸

Worcester, Massachusetts, United States

Nyu Ny Nichd Crs

🇺🇸

New York, New York, United States

Harlem Hosp. Ctr. NY NICHD CRS

🇺🇸

New York, New York, United States

SUNY Upstate Med. Univ., Dept. of Peds.

🇺🇸

Syracuse, New York, United States

DUMC Ped. CRS

🇺🇸

Durham, North Carolina, United States

Philadelphia IMPAACT Unit CRS

🇺🇸

Philadelphia, Pennsylvania, United States

St. Christopher's Hosp. for Children

🇺🇸

Philadelphia, Pennsylvania, United States

Texas Children's Hospital CRS

🇺🇸

Houston, Texas, United States

Children's Hosp. of the King's Daughters, Infectious Disease

🇺🇸

Norfolk, Virginia, United States

Childrens Hosp. of the Kings Daughters

🇺🇸

Norfolk, Virginia, United States

Seattle Children's Research Institute CRS

🇺🇸

Seattle, Washington, United States

Univ. Hosp. Ramón Ruiz Arnau, Dept. of Peds.

🇵🇷

Bayamon, Puerto Rico

San Juan City Hosp. PR NICHD CRS

🇵🇷

San Juan, Puerto Rico

Soweto IMPAACT CRS

🇿🇦

Johannesburg, Gauteng, South Africa

Shandukani CRS

🇿🇦

Johannesburg, Gauteng, South Africa

Univ. of Colorado Denver NICHD CRS

🇺🇸

Aurora, Colorado, United States

Rutgers - New Jersey Medical School CRS

🇺🇸

Newark, New Jersey, United States

St. Jude Children's Research Hospital CRS

🇺🇸

Memphis, Tennessee, United States

Tulane Univ. New Orleans NICHD CRS

🇺🇸

New Orleans, Louisiana, United States

Bronx-Lebanon Hospital Center NICHD CRS

🇺🇸

Bronx, New York, United States

Jacobi Med. Ctr. Bronx NICHD CRS

🇺🇸

Bronx, New York, United States

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