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Anti-HIV Drug Regimens and Treatment-Switching Guidelines in HIV Infected Children

Phase 2
Completed
Conditions
HIV Infections
Interventions
Drug: NRTIs (ABC, FTC, FTC/TDF, 3TC, 3TC/AZT, d4T, TDF, ddC, AZT)
Drug: PIs (AMP, IDV, LPV/r, NFV, SQV, RTV)
Drug: NNRTIs (EFV, NVP)
Registration Number
NCT00039741
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

Little is known about what treatment combinations are best for HIV infected children. This study examined the long-term effectiveness of different anti-HIV drug combinations in children and strategies for switching treatment if the first treatment does not work. The study enrolled children who had not previously taken anti-HIV medication. Participants in this study were recruited in the United States, South America and Europe.

Some European children may also enroll in a substudy that will observe changes in body fat in children taking anti-HIV medications.

Detailed Description

Antiretroviral therapy in children aims to prolong clinical and immunologic health. Currently, there are no data defining a particular highly active antiretroviral therapy (HAART) strategy as the optimal first-line therapy for children. This study evaluated the long-term efficacy of two HAART regimens used as initial therapy: 1) two nucleoside reverse transcriptase inhibitors (NRTIs) plus a protease inhibitor (PI), and 2) two NRTIs plus a nonnucleoside reverse transcriptase inhibitor (NNRTI). It also evaluated different strategies for switching therapy when the initial regimen fails. The long-term nature of this study should clarify whether early switching of therapy improves immunologic and virologic outcomes, or results in a more rapid exhaustion of treatment options. The study was conducted in the United States and in Europe.

Participants in this study had a CD4 cell count and viral load test during a screening visit. Participants had an entry visit that included blood and urine tests. Participants were then randomly assigned to one of four groups: Groups PI/1K and PI/30K received two NRTIs plus a PI; Groups NNRTI/1K and NNRTI/30K received two NRTIs plus an NNRTI. The medications allowed in the study were: abacavir, didanosine, emtricitabine, emtricitabine/tenofovir disoproxil fumarate, lamivudine, lamivudine/zidovudine, stavudine, tenofovir disoproxil fumarate, zalcitabine, and zidovudine (NRTIs); efavirenz and nevirapine (NNRTIs); efavirenz/emtricitabine/tenofovir disoproxil fumurate (NNRTI/NRTI); and amprenavir,atazanavir, darunavir, fosamprenavir calcium, indinavir, lopinavir/ritonavir, nelfinavir, saquinavir, ritonavir, and tipranavir (PIs). Note: Per the 06/28/05 amendment of this trial, emtricitabine, emtricitabine/tenofovir disoproxil fumarate, and tenofovir dioproxil fumarate were added to the list of medications that could be included in a participant's treatment regimen.

For participants whose initial regimen failed, or who experienced clinical disease progression (indicated by the development of a new CDC Category C diagnosis) or other clinical disease progression at or after Week 24 of first-line therapy, second-line therapy was strongly encouraged. (However, if poor adherence was suspected as a possible reason for an increase in HIV viral load, the site and the clinician were to try to improve patient adherence and obtain additional confirmatory viral load values within a five-week time frame.) In second-line therapy, participants who initially took NRTIs with a PI switched to NRTIs and an NNRTI. Participants who initially took NRTIs and an NNRTI switched to NRTIs and a PI. The timing of the switch was based on the participant's group: Groups PI/1K and NNRTI/1K switched to second-line treatment when viral load was 1,000 copies/ml or greater; Groups PI/30K and NNRTI/30K switched to second-line treatment when viral load was 30,000 copies/ml or greater. Participants who failed second-line therapy discontinued study treatment and were offered the best available therapy at the discretion of the clinician.

Participants had study visits at Weeks 2, 4, 8, 12, 16, 24, and every 12 weeks thereafter until the drug regimen was switched to second-line treatment. Participants then had a re-entry visit and the schedule of visits restarted. Participants were in the study between 4 and 7 years, depending on when they enrolled. All study visits included medical history, a physical exam, and blood collection. Urine collection occurred at most visits. Participants were asked to complete adherence questionnaires and PACTG participants underwent neuropsychological assessments at selected visits.

All participants in this study were encouraged to coenroll in PACTG 219C, Long-Term Effects of HIV Exposure and Infection in Children. Participants in the European portion of the study may be asked to enroll in a substudy to observe the development and progression of lipodystrophy syndrome in children.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
266
Inclusion Criteria
  • Older than 30 days and younger than 18 years of age (may enroll up to the day before their 18th birthday)
  • HIV infected
  • Not previously on HAART or received anti-HIV drugs for less than 56 consecutive days after birth to prevent mother-to-infant HIV transmission. Participants who have previously received nevirapine for the prevention of mother-to-infant HIV transmission are not eligible for this study.
  • Willing to use acceptable methods of contraception
Exclusion Criteria
  • Grade 3 or 4 clinical or laboratory toxicity. More information on this criterion can be found in the protocol.
  • Active opportunistic infection or a serious bacterial infection at the time of study entry
  • Pancreas, nervous system, blood, liver, or kidney problems that make it impossible to take study medications
  • Taking any medication that cannot be combined with the study medications in first-line therapy
  • Received therapy for cancer
  • Pregnant or breastfeeding

Study & Design

Study Type
INTERVENTIONAL
Study Design
FACTORIAL
Arm && Interventions
GroupInterventionDescription
PI/30KNRTIs (ABC, FTC, FTC/TDF, 3TC, 3TC/AZT, d4T, TDF, ddC, AZT)2 NRTIs plus 1 PI with a regimen change recommended when viral load reaches 30,000 copies/ml or higher
PI/30KPIs (AMP, IDV, LPV/r, NFV, SQV, RTV)2 NRTIs plus 1 PI with a regimen change recommended when viral load reaches 30,000 copies/ml or higher
NNRTI/30KNNRTIs (EFV, NVP)2 NRTIs plus an NNRTI with a regimen change recommended when viral load reaches 30,000 copies/ml or higher
PI/1KNRTIs (ABC, FTC, FTC/TDF, 3TC, 3TC/AZT, d4T, TDF, ddC, AZT)Two NRTIs plus a PI with a regimen change recommended at when viral load reaches 1000 copies/ml or higher
PI/1KPIs (AMP, IDV, LPV/r, NFV, SQV, RTV)Two NRTIs plus a PI with a regimen change recommended at when viral load reaches 1000 copies/ml or higher
NNRTI/30KNRTIs (ABC, FTC, FTC/TDF, 3TC, 3TC/AZT, d4T, TDF, ddC, AZT)2 NRTIs plus an NNRTI with a regimen change recommended when viral load reaches 30,000 copies/ml or higher
NNRTI/1KNRTIs (ABC, FTC, FTC/TDF, 3TC, 3TC/AZT, d4T, TDF, ddC, AZT)2 NRTIs plus an NNRTI with a regimen change recommended when viral load reaches 1,000 copies/ml or higher
NNRTI/1KNNRTIs (EFV, NVP)2 NRTIs plus an NNRTI with a regimen change recommended when viral load reaches 1,000 copies/ml or higher
Primary Outcome Measures
NameTimeMethod
Change in Viral Load Measured in log10 HIV-1 RNA Copies/mlBaseline visit and 4 years after Study Entry
Secondary Outcome Measures
NameTimeMethod
Rate of Grade 3 or Higher Signs, Symptoms, or Laboratory Abnormalities ExperiencedUp to 6 yrs. (average 4.85 yrs.)

Adverse events were graded according to the following guidelines:

PACTG: "The Manual for Expedited Reporting of Adverse Events to DAIDS" (DAIDS EAE Manual) dated May 6, 2004.

PENTA: International Conference for Harmonization (ICH) requirements and the EU Clinical Trials Directive 2001/20/EC (20).

A rating of Grade 3 is severe and Grade 4 is life-threatening. The rate of serious (Grade 3 or above)events is reported as the number of events per 100 child/years.

Participants With Significant HIV-related Clinical Events, Defined as CDC Category C (AIDS Defining) Diagnoses (Except for Recurrent Bacterial Infections)or DeathUp to 6 yrs. (average 4.85 yrs.)
Time to Switching to an Alternative Class ART Regimen (Based on Initial Randomized Regimen)Up to 6 yrs. (average 4.85 yrs.)

25th Percentiles in weeks from randomization to starting an alternative class ART regimen (based on initial randomized regimen)

Time to HIV-1 RNA of 400 Copies/ml or Greater During First-line Therapy or Permanent Discontinuation of First-line TherapyUp to 6 yrs. (average 4.85 yrs.)

25th Percentiles in weeks from randomization HIV-1 RNA of 400 copies/ml or greater during first-line therapy or permanent discontinuation of first-line therapy.

Time to HIV-1 RNA of 30,000 Copies/ml or Greater During Second-line Therapy or Permanent Discontinuation of Second-line TherapyUp to 6 yrs. (average 4.85 yrs.)

25th Percentiles in weeks from randomization to HIV-1 RNA of 30,000 copies/ml or greater during second-line therapy or permanent discontinuation of second-line therapy

Number of Children With an HIV-1 RNA Level Less Than 400 Copies/ml Regardless of Therapy at Week 204Week 204
Change in CD4% From Randomization to 4 YearsRandomization to 4 years
Number of Children With HIV-1 RNA Less Than 400 Copies/ml and on Original Randomized Therapy at 24 Weeks24 weeks

Trial Locations

Locations (31)

Rutgers - New Jersey Medical School CRS

🇺🇸

Newark, New Jersey, United States

UMDNJ - Robert Wood Johnson Med. School, Div. of Allergy, Immunology & Infectious Diseases

🇺🇸

New Brunswick, New Jersey, United States

South Florida CDTC Ft Lauderdale NICHD CRS

🇺🇸

Fort Lauderdale, Florida, United States

WNE Maternal Pediatric Adolescent AIDS CRS

🇺🇸

Worcester, Massachusetts, United States

St. Jude/UTHSC CRS

🇺🇸

Memphis, Tennessee, United States

Texas Children's Hospital CRS

🇺🇸

Houston, Texas, United States

Connecticut Children's Med. Ctr.

🇺🇸

Hartford, Connecticut, United States

Univ. of Miami Ped. Perinatal HIV/AIDS CRS

🇺🇸

Miami, Florida, United States

SUNY Stony Brook NICHD CRS

🇺🇸

Stony Brook, New York, United States

Univ. of Puerto Rico Ped. HIV/AIDS Research Program CRS

🇵🇷

San Juan, Puerto Rico

San Juan City Hosp. PR NICHD CRS

🇵🇷

San Juan, Puerto Rico

Children's Hosp. & Research Ctr. Oakland, Ped. Clinical Research Ctr. & Research Lab.

🇺🇸

Oakland, California, United States

Univ. of Chicago - Dept. of Peds., Div. of Infectious Disease

🇺🇸

Chicago, Illinois, United States

Washington University Therapeutics (WT) CRS

🇺🇸

Saint Louis, Missouri, United States

Jacobi Med. Ctr. Bronx NICHD CRS

🇺🇸

Bronx, New York, United States

Columbia IMPAACT CRS

🇺🇸

New York, New York, United States

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

🇺🇸

Syracuse, New York, United States

Seattle Children's Hospital CRS

🇺🇸

Seattle, Washington, United States

USF - Tampa NICHD CRS

🇺🇸

Tampa, Florida, United States

Oregon Health & Science Univ. - Dept. of Peds., Div. of Infectious Disease

🇺🇸

Portland, Oregon, United States

Usc La Nichd Crs

🇺🇸

Alhambra, California, United States

Miller Children's Hosp. Long Beach CA NICHD CRS

🇺🇸

Long Beach, California, United States

Nyu Ny Nichd Crs

🇺🇸

New York, New York, United States

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

🇺🇸

Los Angeles, California, United States

Chicago Children's CRS

🇺🇸

Chicago, Illinois, United States

Children's Hospital of Los Angeles NICHD CRS

🇺🇸

Los Angeles, California, United States

Howard Univ. Washington DC NICHD CRS

🇺🇸

Washington, District of Columbia, United States

Harlem Hosp. Ctr. NY NICHD CRS

🇺🇸

New York, New York, United States

Univ. of Florida College of Medicine-Dept of Peds, Div. of Immunology, Infectious Diseases & Allergy

🇺🇸

Gainesville, Florida, United States

Tulane Univ. New Orleans NICHD CRS

🇺🇸

New Orleans, Louisiana, United States

UNC at Chapel Hill School of Medicine - Dept. of Peds., Div. of Immunology & Infectious Diseases

🇺🇸

Chapel Hill, North Carolina, United States

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