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Safety and Efficacy of Tenofovir DF in HIV-1 Infected Adolescents Failing Their Current Antiretroviral Therapy

Phase 3
Completed
Conditions
HIV Infections
Interventions
Drug: Placebo
Registration Number
NCT00352053
Lead Sponsor
Gilead Sciences
Brief Summary

The purpose of this study is to assess the safety and efficacy of tenofovir disoproxil fumarate (tenofovir DF; TDF) plus a genotype-guided optimized background regimen (OBR) compared to placebo plus OBR in the treatment of human immunodeficiency virus type 1 (HIV-1) infected antiretroviral treatment-experienced adolescents with plasma HIV-1 ribonucleic acid (RNA) levels greater than or equal to 1000 copies/mL.

Detailed Description

This is a 48-week, randomized, double-blind, placebo-controlled, multicenter study of the safety and efficacy of tenofovir DF as part of an optimized antiretroviral regimen in HIV-1 infected adolescents (12 years to \< 18 years of age) who are failing their current antiretroviral regimen and have HIV-1 RNA levels ≥ 1000 copies/mL at screening. Data from three consecutive 96-week study extensions have been used to evaluate the long-term efficacy, safety, and tolerability of open-label tenofovir DF as part of an antiviral regimen, providing data for up to 336 weeks of total drug exposure.

Pretreatment:

HIV-1 genotyping will be performed as part of the screening assessments to assist in the construction of an OBR, defined as at least 3, but no more than 5 antiretroviral agents, not including tenofovir DF or placebo.

Randomized Phase:

Participants will be randomized in a 1:1 ratio to receive either tenofovir DF + OBR, or placebo + OBR. The majority of efficacy and safety assessments will be performed at each clinic visit (Weeks 4, 8, 16, 24, 32, 40, and 48). At Week 24, participants who are adherent to study drug (in the opinion of the investigator), but do not demonstrate a ≥ 0.5 log10 copies/mL decrease from baseline in HIV-1 RNA, will be considered to be nonresponders and will be unblinded. Nonresponders randomized to the placebo group will be given the option to continue on study and receive open-label tenofovir DF with an appropriate background regimen determined by the investigator. Nonresponders randomized to the tenofovir DF treatment group will be discontinued from the study.

Extension Phases:

After completing 48 weeks of double-blind treatment with tenofovir DF or placebo, participants who have not reached 18 years of age, and who, in the opinion of the investigator, would derive clinical benefit from the use of open-label tenofovir DF, will be given the option to continue (or initiate) treatment with open-label tenofovir DF in the first of three 96 week study extension periods. Nonresponders who receive open-label tenofovir DF after Week 24 will also be considered eligible for the first study extension if they met the above criteria at Week 48.

After completing the first 96 week study extension, participants who have not reached 18 years of age, and who have shown ongoing clinical benefit from tenofovir DF, will be given the option to continue receiving open-label tenofovir DF for an additional 96 weeks or until tenofovir DF became commercially available in the country where the participants are enrolled, whichever occurs first.

After completing the second 96 week study extension, participants who have not reached 18 years of age, and who have shown ongoing clinical benefit from tenofovir DF, will be given the option to continue receiving open-label tenofovir DF for an additional 96 weeks or until tenofovir DF became commercially available in the country where the participants are enrolled, whichever occurs first.

Presentation of data:

After the randomized phase of the study, participants randomized to placebo during the randomized phase of the study and then switch to open-label tenofovir DF will have their baseline reset (defined as open-label baseline), and only outcome data collected after (on/after for adverse events (AEs)/concomitant medications) participants receive their first dose of open-label tenofovir DF will be included.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
87
Inclusion Criteria
  • Weight ≥ 35 kg
  • Documented laboratory diagnosis of HIV infection
  • Plasma HIV-1 RNA ≥ 1000 copies/mL
  • Prior antiretroviral treatment experience with at least 2 antiretroviral drug classes
  • Naive to tenofovir DF
  • Absence of K65R mutation on genotypic testing
Exclusion Criteria
  • Patients requiring didanosine in background regimen
  • Prior history of significant renal disease
  • Prior history of significant bone disease

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
OBR + Tenofovir DFTenofovir DFTenofovir DF administered orally, one tablet daily without regard to meals
OBR + Tenofovir DF PlaceboPlaceboPlacebo to match tenofovir DF administered orally, one tablet daily without regard to meals
Primary Outcome Measures
NameTimeMethod
Time-weighted Average Change From Baseline Through Week 24 (DAVG24) in Plasma HIV-1 RNABaseline to 24 Weeks

DAVG24 was defined as the time-weighted average between the first postbaseline value through the last value up to Week 24 minus the baseline value. DAVG24 was calculated using the trapezoidal rule with all available postbaseline data minus the baseline value.

Data for participants who discontinued the randomized (double-blind) phase of the study early were included up until the point of study discontinuation (missing data not imputed).

Secondary Outcome Measures
NameTimeMethod
Change From Baseline to Week 336 in CD4 CountBaseline to 336 weeks

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Time-weighted Average Change From Baseline Through Week 48 (DAVG48) in Plasma HIV-1 RNABaseline to 48 weeks

DAVG48 was defined as the time-weighted average between the first postbaseline value through the last value up to Week 48 minus the baseline value. DAVG48 was calculated using the trapezoidal rule with all available postbaseline data minus the baseline value.

Data for participants who discontinued the double-blind phase of the study early were included up until the point of discontinuation from the study (ie, missing data were not imputed).

Change From Baseline to Week 24 in HIV-1 RNABaseline to 24 weeks
Change From Baseline to Week 48 in HIV-1 RNABaseline to 48 weeks
Change From Baseline to Week 96 in HIV-1 RNABaseline to 96 weeks
Change From Baseline to Week 144 in HIV-1 RNABaseline to 144 weeks
Change From Baseline to Week 192 in HIV-1 RNABaseline to 192 weeks
Change From Baseline to Week 240 in HIV-1 RNABaseline to 240 weeks
Change From Baseline to Week 288 in HIV-1 RNABaseline to 288 weeks
Change From Baseline to Week 336 in HIV-1 RNABaseline to 336 weeks

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Change From Baseline to Week 144 in CD4 CountBaseline to 144 weeks
Change From Baseline to Week 288 in CD4 CountBaseline to 288 weeks
Change From Baseline to Week 24 in Cluster Determinant 4 (CD4) CountBaseline to 24 weeks
Change From Baseline to Week 48 in CD4 CountBaseline to 48 weeks
Change From Baseline to Week 96 in CD4 CountBaseline to 96 weeks
Change From Baseline to Week 24 in CD4 PercentageBaseline to 24 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Change From Baseline to Week 240 in CD4 PercentageBaseline to 240 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Change From Baseline to Week 336 in CD4 PercentageBaseline to 336 weeks

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 336Baseline to 336 weeks

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 288Week 288
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 24Week 24
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 96Week 96
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 144Week 144
Change From Baseline to Week 96 in CD4 PercentageBaseline to 96 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 144Baseline to 144 weeks
Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 192Baseline to 192 weeks
Change From Baseline to Week 192 in CD4 CountBaseline to 192 weeks
Change From Baseline to Week 240 in CD4 CountBaseline to 240 weeks
Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 240Baseline to 240 weeks
Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0log 10 Copies/mL From Baseline to Week 288Baseline to 288 weeks
Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 48Week 48
Change From Baseline to Week 48 in CD4 PercentageBaseline to 48 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Change From Baseline to Week 144 in CD4 PercentageBaseline to 144 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Change From Baseline to Week 192 in CD4 PercentageBaseline to 192 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Change From Baseline to Week 288 in CD4 PercentageBaseline to 288 weeks

CD4 percentage is the percentage of total lymphocytes that are CD4 cells.

Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 24Baseline to 24 weeks
Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 96Baseline to 96 weeks
Percentage of Participants With an HIV-1 RNA Decrease of ≥ 1.0 log10 Copies/mL From Baseline to Week 48Baseline to 48 weeks
Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 24Week 24
Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 96Week 96
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 336Week 336

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 144Week 144
Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 192Week 192
Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 240Week 240
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 48Week 48
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 240Week 240
Percentage of Participants With Virologic Failure Through Week 48Up to 48 weeks

Virologic failure was defined as either nonresponse or viral rebound.

* Nonresponse (failure to achieve response). Response was defined as either

* A ≥ 0.5 log10 copies/mL decrease in HIV-1 RNA from baseline at 2 consecutive visits, or

* HIV-1 RNA \< 400 copies/mL at 2 consecutive visits.

* Viral rebound was defined as either

* Participants who achieved a ≥ 0.5 log10 copies/mL decrease from baseline in plasma HIV-1 RNA at 2 consecutive visits, who then subsequently achieved plasma HIV-1 RNA values ≥ 1.0 log10 copies/mL above their on-study nadir (lowest value) and/or plasma HIV-1 RNA values ≥ the baseline value at 2 consecutive visits, or

* Participants who achieved plasma HIV-1 RNA levels of \< 400 copies/mL at 2 consecutive visits, and then subsequently had plasma HIV-1 RNA levels \> 1000 copies/mL at 2 consecutive visits.

The virologic failure rate was estimated from Kaplan-Meier product limit method by including all HIV-1 RNA data collected during the double-blind phase.

Percentage of Participants With HIV-1 RNA < 400 Copies/mL at Week 336Week 336

No analysis was performed because the last study participant discontinued after Week 294 and the study was closed.

Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 192Week 192
Percentage of Participants With HIV-1 RNA < 50 Copies/mL at Week 288Week 288

Trial Locations

Locations (18)

Centro de Doenças Infecciosas e Parasitárias

🇧🇷

Campo Grande - MS, Brazil

Hospital dos Servidores do Estado

🇧🇷

Rio de Janeiro, Brazil

Faculdade de Medicina - UFMG

🇧🇷

Belo Horizonte - MG, Brazil

Hospital das Clinicas da Universidade Federal do Parana - UFPR

🇧🇷

Curitiba - PR, Brazil

Hospital Infantil Joana de Gusmão

🇧🇷

Florianópolis - SC, Brazil

Hospital e Maternidade Celso Pierro

🇧🇷

Campinas - SP, Brazil

Hospital Materno Infantil Professor Fernando Figueira- IMIP

🇧🇷

Recife, Brazil

Universidade Federal de Sao Paulo

🇧🇷

Vila Clementino, Brazil

Instituto da Crianca do Hospital das Clinicas da FMUSP Depto de Pediatria

🇧🇷

Sao Paulo - SP, Brazil

Hospital del Nino

🇵🇦

Panama City, Panama

Hospital Infantil Nossa Senhora da Gloria Servico de Infectologia Pediatria

🇧🇷

Vitoria - ES, Brazil

Universidade Estadual de Campinas - UNICAMP

🇧🇷

Campinas - SP, Brazil

Santa Casa de Belo Horizonte

🇧🇷

Belo Horizonte - MG, Brazil

Hospital Municipal Sao Jose

🇧🇷

Joinville - SC, Brazil

Instituto de Infectologia Emilio Ribas

🇧🇷

Sao Paulo - SP, Brazil

Hospital Geral de Nova Iguacu Ambulatorio de DST e AIDS

🇧🇷

Rio de Jeneiro, Brazil

NEIMPE - Dept of Pediatrics Hospital das Clinicas FMRP-USP

🇧🇷

Sao Paulo, Brazil

Hospital Guilherme Alvaro

🇧🇷

Santos, Brazil

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